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Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.

147 Study Matches

ALX-HPP-501: An Observational,Longitudinal Prospective, Long-term Registry of Patients with Hypophosphatasia

This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.

Principal Investigator: Kyriakie Sarafoglou
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00004936
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Inclusion Criteria:

• confirmed diagnosis of HPP.
• documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
• able to read and speak English
Exclusion Criteria:

• currently participating in an Alexion-sponsored clinical trial
Conditions: Rare Diseases
Keywords: HPP, Hypophosphatasia
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COG AALL2121: A Phase 2 study of SNDX-5613 in combination with chemotherapy for patients with relapsed or refractory KMT2A-rearranged infant leukemia

This phase II trial tests the safety and best dose of revumenib when given together with chemotherapy, and how well the treatment regimen works for infants and young children with leukemia that has come back (relapsed) or does not respond to treatment (refractory) and is associated with a KMT2A (MLL) gene rearrangement (KMT2A-R). Revumenib is an oral medicine that directly targets the changes that occur in a cell with a KMT2A rearrangement and has been shown to specifically kill these leukemia cells in test tubes and animals. Drugs used in chemotherapy, such as vincristine, prednisone, asparaginase, fludarabine and cytarabine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial is being done to find out if the combination of revumenib and chemotherapy may help to treat the cancer cells better than either treatment alone.

Principal Investigator: Peter Gordon
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
IRB Number: STUDY00021176
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Inclusion Criteria:

• Age: Patients must be 1 month to less than 6 years old at the time of study enrollment and must have had initial diagnosis of leukemia less than 2 years old.
• Diagnosis: Patients must have KMT2A-rearranged acute lymphoblastic leukemia (ALL), acute leukemia of ambiguous lineage (ALAL), or mixed phenotype acute leukemia (MPAL), which is determined to be refractory or in first marrow relapse.
• Disease status: First relapse, refractory or failure to achieve remission
• See link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:

• Patients with isolated extramedullary leukemia.
• Patients diagnosed with Down syndrome.
Conditions: Cancer
Keywords: ALAL, ALL, leukemia, MPAL, relapse
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Synergistic Enteral Regimen for Treatment of the Gangliosidoses (SYNER-G) (Syner-G)

The Syner-G regimen research study seeks to evaluate the use of a combination of a medication called miglustat and a ketogenic diet for treatment of the gangliosidoses to learn if this combination will provide improved clinical outcomes compared to what we currently know about the natural course of the disease.

Principal Investigator: Jeanine Jarnes
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
IRB Number: 1311M46101
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Inclusion Criteria:

• no more than 17 years old
• documented infantile or juvenile gangliosidosis disease
Exclusion Criteria:

• severe kidney disease
• females who are pregnant or breast feeding
• females who are post puberty who are unwilling to use highly effective birth control
Conditions: Rare Diseases
Keywords: GM1 Gangliosidoses, GM2 Gangliosidoses, Sandhoff Disease, Tay-Sachs Disease
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D2D - What's Bugging You? Assessing the public's knowledge, attitudes, and perceptions of tick and mosquito borne diseases.

You are invited to take part in a survey to help us learn more about what people know and think about diseases that are spread by ticks and mosquitoes. This study is being conducted by the researchers at the University of Minnesota Medical School on the Duluth Campus. The goal is to understand how people feel about these diseases and how much they know about how to protect themselves.

Principal Investigator: Clara Smoniewski
Age Group: Not specified
This study is also accepting healthy volunteers
IRB Number: STUDY00025228
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Inclusion Criteria:

• MN and WI residents
• age 16 and up
• English speaking
Conditions: Infectious Diseases, Prevention & Wellness, Community Health
Keywords: climate change, Lyme disease, vector-borne disease
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MT2013-09C : Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases

This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.

Principal Investigator: Margaret MacMillan, MD
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: 1305M34181
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Inclusion Criteria:

• up to 55 years old
• see link to clinicaltrials.gov for inclusion criteria specific to each type of leukemia
Exclusion Criteria:

• Radiation Oncology will evaluate all patients who have had previous radiation therapy
• pregnant or breastfeeding
• HIV positive
• study staff will review additional exclusion criteria
Conditions: Cancer
Keywords: Clinics and Surgery Center (CSC), Acute Lymphocytic Leukemia (ALL), Acute Myeloid Leukemia (AML), Chronic Myelogenous Leukemia
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A Randomized, Controlled, Multicenter, Phase 3 Clinical Study Comparing Vusolimogene Oderparepvec in Combination with Nivolumab Versus Treatment of Physician s Choice in Patients with Advanced Melanoma That Has Progressed on an Anti-PD-1 and an Anti-CTLA-4 Containing Treatment Regimen [IGNYTE-3]

The purpose of this research is to compare the effects of nivolumab with vusolimogene oderparepvec (VO) against standard of care treatment drug(s) currently available for patients with advanced melanoma. We expect that taking part in this research will last up to 60 months.

Principal Investigator: Benjamin Manning
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00023233
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Inclusion Criteria:

• at least 12 years old
• confirmed metastatic Stage IIIb through IV/M1a through M1d cutaneous melanoma that cannot be surgically removed
• disease progression (PD) on an approved anti-PD-1 and an anti-CTLA-4 treatment, administered either as a combination regimen (eg, nivolumab + ipilimumab) or in sequence for at least 8 weeks
• documented BRAF V600 mutation status
• see link to clinicaltrials.gov for complete inclusion criteria
Exclusion Criteria:

• more than 2 lines of systemic therapy for advanced melanoma
• known acute or chronic hepatitis
• known human immunodeficiency virus (HIV) infection
• prior cancer in the previous 3 years, except for locally curable cancers that have apparently been cured
• see link to clinicaltrials.gov for complete exclusion criteria
Conditions: Cancer
Keywords: Clinics and Surgery Center (CSC), Advanced Melanoma, Melanoma
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Transcranial Magnetic Stimulation to Augment Exposure and Response Prevention for Pediatric OCD (NExT)

We are doing this study to see if we can improve the standard treatment for OCD, Exposure with Response Prevention, by pairing it with Transcranial Magnetic Stimulation to the parts of the brain that cause OCD symptoms.

Principal Investigator: Christine Conelea
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00020646
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Inclusion Criteria:

• 12 to 21 years old
• right-handed
• currently have OCD symptoms
Exclusion Criteria:

• inability to have a MRI
• left-handed
• study staff will review additional exclusion criteria
Conditions: Brain & Nervous System, Children's Health, Mental Health & Addiction
Keywords: Obsessive-compulsive disorder, OCD, psychology, therapy, TMS, transcranial magnetic stimulation
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A Phase 1/2a Open-Label Dose-Ranging and Observer-Blind Placebo-Controlled, Safety and Immunogenicity Study of mRNA-1647 Cytomegalovirus Vaccine in Female and Male Participants 9 to 15 Years of Age; mRNA-1647-P104

This study it to test an investigational vaccine called mRNA-1647 that is being developed for preventing cytomegalovirus (CMV) infection in people. CMV is a common virus that can spread easily through an infected person’s saliva or other body fluids such as blood, urine, and breast milk. We want see if the trial vaccine can prevent CMV infection in participants who have not been previously infected, to understand the safety (how many side effects you may have) of the trial vaccine, and to see if the trial vaccine results in participants making antibodies to CMV.

Principal Investigator: Mark Schleiss
Age Group: Up to 18 years old
This study is also accepting healthy volunteers
IRB Number: SITE00001871
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Inclusion Criteria:

• female or male 9 to 15 years of age
• in good general health
• BMI requirements ( study staff will review)
• female participants of childbearing potential: negative pregnancy test and adequate contraception for at least 28 days prior to receiving vaccine through 3 months following vaccine administration
Exclusion Criteria:

• received, or plans to receive, any nonstudy vaccine less than 28 days prior to or after any study medication
• any diagnosis or condition requiring significant changes in management or medication within the 2 months before starting the study
• contact study staff for review of additional exclusion criteria
Conditions: Children's Health, Infectious Diseases
Keywords: CMV, cytomegalovirus, vaccine
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Longitudinal Study of Porphyrias

The objective is to conduct a longitudinal investigation of the natural history, complications, and therapeutic outcomes in people with acute and cutaneous porphyria.

Principal Investigator: Marshall Mazepa, MD
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: SITE00000892
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Inclusion Criteria:

• patient of any age
• diagnosis of a porphyria
• biochemical findings, as documented by laboratory reports of porphyria-specific testing performed after 1980
Exclusion Criteria:

• elevations of porphyrins in urine, plasma or erythrocytes due to other diseases
Conditions: Rare Diseases
Keywords: Acute Porphyrias, Cutaneous Porphyrias
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A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature (ISS)

This research is being done to learn if a study drug called vosoritide can help children who are shorter than should be for their age to grow.

Principal Investigator: Brad Miller, MD, PhD
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
IRB Number: STUDY00023763
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Inclusion Criteria:

• 3 to 10 years old if a girl
• 3 to 11 years old if a boy
• have short stature compared to others of the same gender and age with no known cause
• able to move and stand without help
Exclusion Criteria:

• known chromosomal imbalance or genetic variant causing short stature syndrome
• previous treatment with a growth promoting agent
Conditions: Diabetes & Endocrine, Rare Diseases
Keywords: idiopathic short stature, ISS, short
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MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders

The primary research element is to determine whether a graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide, tacrolimus and MMF will reduce the likelihood of chronic GVHD in patients receiving a standard hematopoietic myeloablative stem cell transplant. The treatment related components of this protocol are established clinical practices. We are looking at cumulative incidence of chronic GVHD requiring systemic immunosuppressive treatment at 1 year post-transplant.

Principal Investigator: Punita Grover
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00001087
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Inclusion Criteria:

• no more than 60 years old
• may be unable to work; able to live at home and care for self
• women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria (differ by diagnosis)
Exclusion Criteria:

• if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
• active central nervous system cancer
• active HIV infection or known HIV positive serology
• active uncontrolled infection
• women who are pregnant or breast feeding
Conditions: Cancer
Keywords: Clinics and Surgery Center (CSC), Acute Leukemia, Acute Myeloid Leukemia, ALL, AML, HSCT
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Tissue biopsies for the study of FSHD

A single visit study with muscle and/or skin biopsy / blood draw, performed to determine whether a molecular or cellular defect can be attributed to cells of Fascioscapulohumeral Muscular Dystrophy (FSHD) muscle. This study is recruiting both individuals with genetically confirmed FSHD as well as unaffected healthy (control) individuals.

Principal Investigator: Michael Kyba
Age Group: Not specified
This study is also accepting healthy volunteers
IRB Number: STUDY00000409
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Inclusion Criteria:

• Genetic confirmation of Fascioscapulohumeral Muscular Dystrophy (FSHD)
• at least 4 years old
• Healthy Family Members: parent or sibling of someone who has FSHD
Exclusion Criteria:

• heart failure, respiratory insufficiency that requires respiratory support
• taking anticoagulants or anti platelet medications (aspirin or NSAIDs)
Conditions: Rare Diseases, Heart & Vascular
Keywords: Fascioscapulohumeral Muscular Dystrophy (FSHD), Clinics and Surgery Center (CSC)
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PEPN2312; A Phase 1 study of GRN163L (Imetelstat, IND# 170891, NSC# 754228) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia that is in second or greater relapse or that is refractory to relapse therapy; myelodysplastic syndrome or juvenile myelomonocytic leukemia in first or greater relapse or is refractory to relapse therapy

This phase I trial tests the safety, side effects, and best dose of imetelstat in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) that has not responded to previous treatment (refractory) or that has come back after a period of improvement (recurrent). Imetelstat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as fludarabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving imetelstat in combination with fludarabine and cytarabine may work better in treating patients with refractory or recurrent AML, MDS, and JMML.

Principal Investigator: Robin Williams
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
IRB Number: STUDY00022789
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Inclusion Criteria:

• Between 1 year and less than or equal to 18 years of age at enrollment
• Patients, with or without Down syndrome (DS), and with de novo acute myeloid leukemia, therapy-related AML, MDS or JMML.
• In second or greater relapse or refractory AML or First or greater relapse of MDS, or First or greater relapse of JMML
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:

• Pregnant or breast-feeding
• Currently receiving investigational drugs or other anti-cancer agents
Conditions: Cancer
Keywords: acute myeloid leukemia, AML, JMML, juvenile myelomonocytic leukemia, MDS, myelodysplastic syndrome
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A Phase 2, Open-Label, Basket Study of Atrasentan in
Patients with Proteinuric Glomerular Diseases (AFFINITY)

The purpose of the research is to find out if atrasentan delays worsening of kidney function in IgAN, FSGS, and Alport Syndrome.

Principal Investigator: Michelle Rheault
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00012146
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Inclusion Criteria:

• Age 18 years and older for patients in the IgAN, FSGS, and Alport Syndrome cohorts
• age 18-70 years for patients in the DKD cohort
• receiving a maximally tolerated dose of RAS inhibitor therapy (ACEi or ARB) that has been stable for at least 12 weeks
• there are different requirements for each diagnosis category & study staff will review these
Exclusion Criteria:

• current diagnosis of another cause of chronic kidney disease or another primary glomerulopathy
• history of kidney transplantation or other organ transplantation
• except for FSGS patients, use of systemic immunosuppressant medications, such as steroids, for more than 2 weeks in the past 3 months
• blood pressure above 150 mmHg systolic or 95 mmHg diastolic
• history of heart failure or a previous hospital admission for fluid overload.
• history of liver disease
• hemoglobin below 9 g/dL or blood transfusion for anemia within the past 3 months.
• cancer in the past 5 years (except nonmelanoma skin cancer and curatively treated cervical carcinoma in situ)
• women who are pregnant, breastfeeding, or intend become pregnant during the study
• recently received an investigational agent -clinically significant unstable or uncontrolled medical condition (study staff will review)
Conditions: Kidney, Prostate & Urinary
Keywords: Glomerular Disease, Alport Syndrome, IgAN, FSGS, Proteinuric Glomerular Diseases
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A Window of Opportunity Trial of Mirdametinib plus Vorinostat for NF1 Associated, Malignant Peripheral Nerve Sheath Tumor; MPNST (Le-Na)

This is a small, Phase 0, window of opportunity study to provide human experience to support our pre-clinical data and gain preliminary information regarding the safety and tolerability of mirdametinib and vorinostat when given in combination.

Principal Investigator: Christopher Moertel, MD
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
IRB Number: STUDY00022372
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Inclusion Criteria:

• Known Neurofibromatosis type 1 (NF-1) syndrome based on current diagnostic criteria
• Diagnosis of suspected MPNST by PET or MRI imaging
• Confirmation of histone H3 lysine 27 trimethylation-negative MPSNT by immunohistochemistry
• Twelve years of age or older - Complete blood count (CBC), platelet, liver and kidney function within institutional normal limits performed within 14 days of 1st dose of study drug
• Must be able to swallow capsules
• Females of childbearing potential must use highly effective contraception (see inclusion criteria section) from the time of study enrollment through 6 months after the last dose of vorinostat and mirdametinib
• Males with partners of childbearing potential must use highly effective contraception from the time of study enrollment through 3 months after the last dose of vorinostat
• Provides voluntary written consent prior to any study related activities, with parental/guardian consent and assent for those 12 to 17 years of age at enrollment
Exclusion Criteria:

• Pregnant or breastfeeding – females of childbearing potential must have a negative pregnancy test (serum and urine) within 7 days prior to the 1st dose of the study drugs
• Significant cardiac disease
• Ophthalmologic conditions
• Radiation therapy or chemotherapy in the past year
• Participants receiving systemic or ocular glucocorticoid therapy within 14 days prior to the first dose of study treatment
Conditions: Brain & Nervous System
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Udall P1A4

Through this research, the study staff hopes to better understand how DBS works and to define the optimal site in the brain for DBS treatment for Parkinson’s Disease. You will be asked to come for one study visit where you will perform some physical and mental tasks while on and temporarily off your medications and DBS treatment. Participation in this research study will take 7-8 hours.

Principal Investigator: Jerrold Vitek
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00019735
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Inclusion Criteria:

• at least 10 years old
• diagnosis or suspected diagnosis of Parkinson's disease, Essential Tremor, or Dystonia
• implanted Deep Brain Stimulator (DBS)
• have a 7T MRI
Exclusion Criteria:

• history of dementia
• women who are pregnant or breastfeeding
• other exclusion criteria (study staff will review)
Conditions: Brain & Nervous System
Keywords: DBS, Dystonia, Essential Tremor, ET, Movement Disorders, Parkinson's, Parkinson's Disease, PD
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COG ACNS1833 - A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)

The overall goal of this phase 3 non-inferiority study is to assess if selumetinib works as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with low-grade glioma (LGG).

Principal Investigator: Christopher Moertel, MD
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: SITE00000799
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Inclusion Criteria:

• 2 to 21 years old
• all tumors considered low-grade glioma or low-grade astrocytoma
• patients with metastatic disease or multiple independent primary LGG are eligible
• patients must have the ability to swallow whole capsules
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:

• must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
Conditions: Cancer
Keywords: Brain Cancer, Low Grade Astrocytoma, Low Grade Glioma
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Can spectral power and coherence reflect the integrity of the efferent cerebellar cortical pathway in cerebellar mutism syndrome?

This study will be measuring brain activity using EEG and assessing motor skills and speech in children following cancerous brain tumor resection. No direct cancer treatments or objectives are being targeted.

Principal Investigator: Sharyl Samagia-Grivette
Age Group: Not specified
This study is also accepting healthy volunteers
IRB Number: STUDY00019602
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Inclusion Criteria:

• Cerebellar Mutism Syndrome (CMS) & Comparison (without CMS) Groups: ages 10 years 0 months to 25 years 11 months of age & fluent in English (parents/guardian do not need to be fluent in English)
• For those with Cerebellar Mutism Syndrome (CMS): history of resection of posterior fossa tumor at least 2 years before starting the study and at least 3 months post chemotherapy and radiation treatment
Exclusion Criteria:

• Comparison group without CMS: any developmental conditions including ADD/ADHD, learning disabilities, speech/language delay or disorder, motor delay/disorder, cognitive delay and/or diagnosis of autism spectrum disorder
• any genetic condition
• any neurologic condition including history of stroke, seizure disorder, or brain injury
• history of brain tumor or other cancer diagnosis
• CMS Group: any developmental conditions including ADD/ADHD, learning disabilities, speech/language delay or disorder, motor delay/disorder, cognitive delay and/or diagnosis of autism spectrum disorder prior to brain tumor diagnosis
• any genetic condition prior to brain tumor diagnosis
• any neurologic condition including history of stroke, seizure disorder, or brain injury disorder prior to brain tumor diagnosis
Conditions: Brain & Nervous System, Cancer, Children's Health
Keywords: brain tumor, cerebellar mutism syndrome (CMS)
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MT2022-45 Primary Immune Regulatory Disorders (PIRD): Longitudinal Study of Clinical Presentation, Treatment and Outcomes

Primary Immune Regulatory Disorders (PIRD disorders) are a group of diseases that cause the immune system to function abnormally and cause infections, autoimmunity or inflammation that can begin early in life. PIRD is usually caused by changes in genes in DNA. Researchers are trying to learn what types of medical problems patients with PIRD have and how these respond to treatment. Researchers also want to learn which genes cause PIRD and how it can cause the medical problems of PIRD.

Principal Investigator: Christen Ebens
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: SITE00001766
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Inclusion Criteria:

• age 0 to 99 years
• diagnosis of immune-mediated bowel disease affecting at least one segment of the bowel
• evidence of interstitial lung disease (ground-glass opacities) or pulmonary nodules/cysts
• decreased lung function
• additional inclusion and exclusion criteria apply (study staff will review)
• also enrolling parent, sibling, or child of eligible participants
Exclusion Criteria:

• documented HIV infection
Conditions: Rare Diseases
Keywords: Clinics and Surgery Center (CSC)
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ITCC-101/APAL2020D - A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML (A subtrial of the PedAL/EuPAL relapsed acute leukemia master protocol)

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse.

Principal Investigator: Peter Gordon
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: SITE00001628
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Inclusion Criteria:

• participants must be at least 29 days of age and less than 21 years of age at enrollment
• participants must have enrolled on APAL2020SC, NCT Number: NCT04726241
• children, adolescents, and young adults with acute myeloid leukemia without FLT3/internal tandem duplication (ITD) mutation
• second relapse who are sufficiently fit to undergo another round of intensive chemotherapy
• first relapse who per investigator discretion cannot tolerate additional anthracycline containing chemotherapy
• see link to clinicaltrials.gov for complete criteria
Exclusion Criteria:

• participants with Down syndrome
• participants with Acute promyelocytic leukemia (APL) or Juvenile myelomonocytic leukemia (JMML)
• study staff will review additional exclusion criteria
Conditions: Cancer, Cancer
Keywords: Acute Myeloid Leukemia, AML
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MT2019-09: A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCR alpha beta +/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC CSIDE Protocol (CSIDE)

We want to study if lower doses of a chemotherapy drug called busulfan will help babies with SCID achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants.

Principal Investigator: Christen Ebens
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
IRB Number: SITE00000541
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Inclusion Criteria:

• 0 to 2 years old
• infants with SCID, either typical or leaky or Omenn syndrome
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:

• any serious life-threatening or opportunistic infection at time of enrollment
• HIV or HTLV I/II infection
Conditions: Cancer, Cancer
Keywords: SCID, Severe Combined Immunodeficiency
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A Natural History Study of the Gangliosidoses

This is a research study documenting the natural history of disease in patients with GM1 or GM2 gangliosidosis. The information collected will be a way to evaluate disease progression and create a disease stage and severity index. Our goal is to use the data collected to measure the effectiveness of any treatments that are developed in the future.

Principal Investigator: Jeanine Jarnes
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: 1007M85712
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Inclusion Criteria:

• documented gangliosidosis disease
• able to complete neuropsychological and neurobehavioral assessments
• Late-onset gangliosidosis subjects must be able to tolerate MRI of the head
Exclusion Criteria:

• none
Conditions: Rare Diseases
Keywords: GM1 Gangliosidosis, GM2 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
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HM2024-28: A Pilot Study to Investigate the Clinical Utility of Tremoflo Airway Oscillometry in the Pediatric and Adolescent Population with Pulmonary Pathology or at Risk of Lung Injury

The purpose of this study is to help investigators learn more about what are the best methods for diagnosing and monitoring lung problems.

Principal Investigator: Samuel Goldfarb
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00023347
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Inclusion Criteria:

• age 0 to 24
• physician thinks patient might benefit from early and more frequent pulmonary function monitoring
• at risk for developing lung disease
Exclusion Criteria:

• unlikely to complete required study components
Conditions: Cancer, Respiratory System
Keywords: child cancer, lungs, oxygen level, asthma, BPD, Cystic Fibrosis, interstitial lung disease
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AOST2031: A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma

This phase III trial compares the effect of open thoracic surgery (thoracotomy) to thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery is a type of surgery done through a single larger incision (like a large cut) that goes between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest surgery where the doctor makes several small incisions and uses a small camera to help with removing the cancer. This trial is being done evaluate the two different surgery methods for patients with osteosarcoma that has spread to the lung to find out which is better.

Principal Investigator: Robin Williams
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: SITE00001674
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Inclusion Criteria:

• 50 years of age or younger
• have 4 or less nodules in the lung due to metastases or suspected metastases
• diagnosis of osteosarcoma
• contact study team for more detailed criteria
Exclusion Criteria:

• pleural or mediastinal based metastatic lesions, or with pleural effusion
• large, or central tumors that require a lobectomy or pneumonectomy
Conditions: Cancer
Keywords: lung cancer, osteosarcoma, pulmonary metastases, thoracotomy
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Cortical Inhibitory Biomarkers of Acute Suicidal States in Adolescents

Risk for suicidal behavior in young people may include particular types of brain functions. The goal of this research is to learn more about the biology of the brain as it relates to suicidal thoughts and behaviors.

Principal Investigator: Charles Lewis
Age Group: Up to 18 years old
This study is also accepting healthy volunteers
IRB Number: STUDY00011433
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Inclusion Criteria:

• ages 13-21
• current diagnosis of depression
• prior history of either thoughts about suicide, or suicidal behaviors (a suicide attempt or a planned attempt that was stopped before it began)
• must be able to communicate verbally and in writing in English
• parental permission is needed for participants aged 17 or younger
• must have reliable internet connection
Healthy volunteers
also needed
Exclusion Criteria:

• Active substance use in the past month
• Neurological disorders such as seizures, head injury
Conditions: Children's Health, Mental Health & Addiction
Keywords: Depression, Suicide, Adolescent
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A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. We will also check if the study drug works to improve your kidney function, if has an impact on your daily life and the amount of the study drug in your blood over a period of time (called pharmacokinetics)

Principal Investigator: Michelle Rheault
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00015869
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Inclusion Criteria:

• at least 12 years of age
• for people with Alport Syndrome: confirmed diagnosis by genetic testing and /or kidney biopsy
• for primary Focal Segmental Glomerulosclerosis (FSGS), (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein
• female patients, as well as, female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (90 days after the last dose of study medication)
• males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug
• contact study staff for additional criteria
Exclusion Criteria:

• uncontrolled diabetes mellitus as evidenced by an HbA1c greater or equal to 11%
• uncontrolled high blood pressure
• moderate or severe liver impairment
• BMI greater than 40
• women who are pregnant or breast feeding
• additional exclusion criteria apply (study staff will review)
Conditions: Kidney, Prostate & Urinary
Keywords: Clinics and Surgery Center (CSC), Alport Syndrome, Focal Segmental Glomerulosclerosis
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Stability 2: ACL Reconstruction +/- Lateral Tenodesis with Patellar vs Quad Tendon (Protocol # PRO19020231) (STABILITY 2)

The purpose of this multicenter study is to compare outcomes between patients who will undergo different types of ACL reconstruction. All patients will have a tendon from their own knee used to reconstruct the ACL. Prior to knee surgery, researchers will randomize (i.e. a coin toss) to determine if ACL reconstruction will be done with patellar or quadriceps tendon and if the lateral extra-articular tenodesis will or will not be added to the ACL surgery. We will follow-up with participants as they undergo treatment and recovery after surgery for 2 years.

Principal Investigator: Jeffrey Macalena
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: SITE00000971
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Inclusion Criteria:

• age 14-25
• ACL deficient knee
• at least two of the following: participate in a competitive pivoting sport; have a pivot shift of grade 2 or greater; have generalized ligamentous laxity
Exclusion Criteria:

• previous ACL repair on either knee
• partial ACL tear
• multiple ligament injury (two or more ligaments requiring surgery)
• pregnancy
Conditions: Bone, Joint & Muscle
Keywords: Clinics and Surgery Center (CSC), ACL, Anterior cruciate ligament reconstruction, joint instability
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The TrialNet Natural History Study of the Development of Type 1 Diabetes

TrialNet is an international research group dedicated to the study, prevention, and early treatment of Type 1 diabetes. Type 1 diabetes is now understood as a disease that develops over time in stages. Stage 1 starts with the appearance of having two or more autoantibodies. This is followed by Stage 2, which is the development of abnormal blood glucose levels. Stage 3 is the clinical diagnosis of Type 1 diabetes. This study will help us learn more about how Type 1 diabetes occurs through the screening of diabetes-related autoantibodies and monitoring individuals who have tested positive for these autoantibodies.

Principal Investigator: Antoinette Moran
Age Group: Not specified
This study is also accepting healthy volunteers
IRB Number: SITE00000016
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Inclusion Criteria:

• 2.5 to 45 years old and have an immediate family member with type 1 diabetes (child, parent, or sibling)
• 2.5 to 20 years old who have an extended family member with type 1 diabetes (cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
Exclusion Criteria:

• already have diabetes
• history of being treated with insulin or oral diabetes medications
• using systemic immunosuppressive agents (topical and inhaled agents are acceptable)
• have any known serious diseases
Conditions: Diabetes & Endocrine
Keywords: T1DM, Type 1 Diabetes

NEA Research Lab

This keystone study will invite adolescents into a 2-semester creative arts / science program, with arts activities taking place at the Masonic Institute of the Developing Brain (MIDB).

Principal Investigator: Kathryn Cullen
Age Group: Up to 18 years old
This study is also accepting healthy volunteers
IRB Number: STUDY00015930
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Inclusion Criteria:

• age 13-17
• adolescents and at least one parent / guardian must be able to speak English
Exclusion Criteria:

• unable to have a MRI (e.g. claustrophobia, braces)
• neurodevelopmental disorder (e.g. intellectual disability, severe autism)
• major medical and/or neurological illness
• pregnancy when starting the study
Conditions: Children's Health, Mental Health & Addiction
Keywords: Adolescents, Arts, Creativity
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Testing ACL Reconstruction Outcome PredicTions (TAROT)

The purpose of this study is to see how accurately orthopaedic surgeons predict outcomes for people who have an anterior cruciate ligament (ACL) reconstruction. Patients will be asked to complete post-surgery surveys about occurrence of side effects, return to activity, reoperation, and satisfaction with the surgery at 4 months, 2 years, and 5 years.

Principal Investigator: Richard Kyle Martin
Age Group: Not specified
This study is NOT accepting healthy volunteers
IRB Number: STUDY00021757
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Inclusion Criteria:

• patients undergoing primary ACLR (anterior cruciate ligament reconstruction) at the University of Minnesota, Tria and CentraCare.
Conditions: Bone, Joint & Muscle
Keywords: ACL, anterior cruciate ligament, anterior cruciate ligament reconstruction, Clinics and Surgery Center (CSC), ortho surgeon prediction
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