
Search Results
A Multicenter, Randomized, Parallel-group, Double-blind, Two-arm, Phase III Study to Evaluate the Safety and Efficacy of Anifrolumab Compared with Placebo in Male and Female Participants 18 to 70 Years of Age Inclusive with Systemic Sclerosis (DAISY)
We are doing this study to learn more about anifrolumab (SAPHNELOTM) in patients with systemic sclerosis and to better understand the studied disease and associated health problems. Systemic sclerosis (scleroderma) affects the skin as well as other organs, such as blood vessels, muscles and joints, digestive tract, kidneys, lungs and heart.
• 18 to 70 years old
• diagnosis of systemic sclerosis within 6 years from first non-Raynaud's symptoms
• skin at injections sites is without symptoms
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• severe heart or lung disease
• history of any other inflammatory diseases
• history of hematopoietic stem cell transplantation or solid organ or limb transplantation
• current or a history of cancer within past 5 years
• active current or history of reoccurring infections
Continuation of a Home/Community-Based Anal Cancer Screening Unit and Protocol at LGBTQ+ Focused Community Events
This study will help to identify challenges and barriers to self-performing anal cancer screening tests, and may identify unique ways to make this form of screening easier, more cost-effective, and more frequently performed. We believe that it has the potential to minimize the frequency of both disease and death from anal cancer among high-risk patient groups.
• at least 35 years old
• assigned sex of “male” at birth
• engage in anoreceptive intercourse with male partners
• willing to provide reliable contact information
• in the case of a positive screen, willing to undergo a clinic visit and HRA
• fluent in English
• previous diagnosis of high-grade anal dysplasia or anal cancer
PEPN2011 - A Phase 1/2 Study of Tegavivint (IND#156033, NSC#826393) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors
This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating children, adolescents, and young adults with recurrent or refractory solid tumors, including lymphomas and desmoid tumors.
• 12 months to 30 years old
• patients with recurrent or refractory solid tumors including non-Hodgkin lymphoma and desmoid tumors are eligible
• patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• pregnant or breast-feeding women
• patients who are currently receiving other anti-cancer agents
• patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant
• patients with primary brain tumors
• patients who have received a solid organ transplant
COG AGCT1531 - A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors
This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
• newly diagnosed with a Stage I germ cell tumor or metastatic germ cell tumor
• see link to clinicaltrials.gov for detailed inclusion criteria
• patients must have had no prior systemic therapy for the current cancer diagnosis
• patients must have had no prior radiation therapy (exception of CNS irradiation of brain metastases for standard risk 1 patients)
• female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs
• lactating females who plan to breastfeed their infants
• there are additional exclusion criteria (study staff will review)
COG AALL1621 - A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518, IND#133494) in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients (≥1 year and < 22 years ) with CD22 positive B acute lymphoblastic leukemia that has come back or does not respond to treatment. Immunotoxins, such as inotuzumab ozogamicin, are antibodies linked to a toxic substance and may help find cancer cells that express CD22 and kill them without harming normal cells.
• 1 to 21 years old
• must have B Acute Lymphoblastic Leukemia (B-ALL), or previously diagnosed B lymphoblastic lymphoma (B-LL)
• Patients with one of the following: Second or greater relapse; Primary refractory disease with at least 2 prior induction attempts; First relapse refractory to at least one prior re-induction attempt; OR Any relapse after HSCT (Cohort 1 ONLY)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• currently receiving another investigational drug
• currently receiving or plan to receive other anti-cancer agents (except hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy, and intrathecal chemotherapy)
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease (Le-Na)
This is an observational study intended to generate preliminary data to understand how lysosomal dysfunction can affect the biogenesis of extracellular vesicles, its content and function. The study entails 2 visits over a 3-month period. On enrollment, participants will be scheduled for the 2 visits, during which fasting blood samples will be collected.
• ages 18 to 80
• healthy volunteers without any known diagnosis
• hematological cancer or other uncontrolled medical conditions
A PHASE II, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO ASSESS THE EFFICACY AND SAFETY OF RO7790121 IN PATIENTS WITH MODERATE TO SEVERE ATOPIC DERMATITIS
The purpose of this study is to learn about the efficacy and safety of RO7790121, which is a new medication being developed to treat people who have moderate to severe atopic dermatitis (AD). Participants will receive either the new medication or a placebo (doesn't contain any medicine) and the results will be compared. The total time of study treatment for an individual will be approximately 30 weeks.
• Diagnosis of Atopic Dermatitis
• Prior or current treatment with any approved or investigational biologics
MT2024-08: Phase I open-label, dose escalation trial of BI 1831169 monotherapy and in combination with an anti-PD-1 mAb in patients with advanced or metastatic solid tumors.
This study tests the use of the oncolytic virus BI1831169 (VSV-GP) as an immunotherapy in patients with advanced solid tumors. This trial is the first-in-human trial to test the safety and early efficacy of BI1831169 by itself (Part 1) and in combination with the PD-1 inhibitor ezabenlimab (Part 2).
• confirmed diagnosis of an advanced, and/or metastatic or relapsed/refractory solid tumor that can not be surgically removed
• must have exhausted available treatment options or refused established treatment options
• restricted from physically strenuous activity but able to walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• see link to clinicaltrials.gov for additional Inclusion criteria
• major surgery or radiation therapy in the past 4 weeks
• active hepatitis B or C infection
• severe or serious, acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation (study staff will review)
• see link to clinicaltrials.gov for complete Exclusion criteria
A Phase 1, First in Human, Dose-Escalation Study of TORL-1-23 in Participants with Advanced Cancer (TRIO049)
This first-in-human study will evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of TORL-1-23 in patients with advanced cancer.
• advanced solid tumor
• restricted strenuous physical activity but can walk and able to carry light work e.g., light house work, office work
• progressive or symptomatic brain metastases
• serious, uncontrolled medical disorder or active, uncontrolled infection
• history of significant hear disease
• history of another cancer within 3 years
• women who are pregnant or breast feeding
• contact study staff for additional exclusion criteria
PEPN2111 - A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
A Phase I/II trial of single agent intravenous CBL0137 in pediatric patients (≥ 12 months and ≤ 30 years) with relapsed/refractory solid tumors, including CNS tumors and lymphoma.
• 12 months to 30 years old
• patients with relapsed or refractory solid tumors or lymphoma, including patients with CNS tumors or known CNS metastases, or patients with progressive or recurrent DIPG (diagnosed by biopsy or imaging characteristics) and other H3 K27M-mutant diffuse midline gliomas previously treated with radiation therapy, or patients with relapsed or refractory osteosarcoma
• patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment
• patients have consented to receive a central venous catheter prior to the administration of CBL0137
• see link to clnicaltrials.gov for complete inclusion and exclusion criteria
• pregnant or breast-feeding women
• patients who have an uncontrolled infection
• patients who have received a prior solid organ transplantation
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.
• 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)
• 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
AALL2131; An International Pilot Study of Chemotherapy and Tyrosine Kinase Inhibitors with Blinatumomab in Patients with Newly-Diagnosed Philadelphia Chromosome-Positive or ABL-class Philadelphia Chromosome-Like B-cell Acute Lymphoblastic Leukemia
This phase III trial compares the effect of the combination of blinatumomab with dasatinib and standard chemotherapy versus dasatinib and standard chemotherapy for treating patients with Philadelphia chromosome positive (PH+) or Philadelphia chromosome-like (Ph-Like) ABL-class B-Cell acute lymphoblastic leukemia (B-ALL). Blinatumomab is a bispecific antibody that binds to two different proteins-one on the surface of cancer cells and one on the surface of cells in the immune system. An antibody is a protein made by the immune system to help fight infections and other harmful processes/cells/molecules. Blinatumomab may bind to the cancer cell and a T cell (which plays a key role in the immune system's fighting response) at the same time. Blinatumomab may strengthen the immune system's ability to fight cancer cells by activating the body's own immune cells to destroy the tumor. Dasatinib is in a class of medications called tyrosine kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply, which may help keep cancer cells from growing. Giving blinatumomab and dasatinib in combination with standard chemotherapy may work better in treating patients with PH+ or Ph-Like ABL-class B-ALL compared to dasatinib and chemotherapy alone.
• Known history of chronic myeloid leukemia (CML)
• ABL-class Ph-like B-ALL who are CNS2 or CNS3 at end of Induction phase.
• ALL developing after a previous cancer treated with cytotoxic chemotherapy.
• Active, uncontrolled infection or active systemic illness that requires ongoing vasopressor support or mechanical ventilation
• Down syndrome (trisomy 21)
• Pregnancy and breast feeding.
HM2023-21: A Phase 3 Randomized Study Comparing Talquetamab in Combination with Pomalidomide (Tal-P), Talquetamab in Combination with Teclistamab (Tal-Tec), and Investigator s Choice of Either Elotuzumab, Pomalidomide, and Dexamethasone (EPd) or Pomalidomide, Bortezomib, and Dexamethasone (PVd) in Participants with Relapsed or Refractory Myeloma who Have Received 1 to 4 Prior Lines of Therapy Including an Anti-CD38 Antibody and Lenalidomide (MonumenTAL-6)
The purpose of this study is to compare the effects of talquetamab in combination with teclistamab (Tal-Tec), the effects of talquetamab in combination with pomalidomide (Tal-P), and the effects of either the combination of elotuzumab, pomalidomide, and dexamethasone (EPd) or pomalidomide, bortezomib, and dexamethasone (PVd) in treating patients with multiple myeloma, who have not responded to previous treatment.
• diagnosis of multiple myeloma
• cancer that has recurred or has not improved with treatment
• previously treated 1 to 4 times (lines of therapy)
• able to walk and complete all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• agree not to be pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or within 6 months after the last dose of study treatment
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• stroke, transient ischemic attack, or seizure in the past 6 months
• active central nervous system (CNS) involvement or exhibits clinical signs of meningeal involvement of multiple myeloma
A Pivotal Study Evaluating Safety and Efficacy of the ShiraTronics Migraine Therapy System in RELIEVing, Interrupting, and Preventing Chronic Migraine (RELIEV-CM2)
This research study is testing a potential new treatment for refractory chronic migraine (RCM). The potential new treatment is called ShiraTronics Migraine Therapy. The purpose of this study is to demonstrate the safety and effectiveness of the ShiraTronics Migraine Therapy System. The ShiraTronics System delivers mild electrical pulses to nerves associated with migraine pain around the back and front of your head. These electrical pulses interrupt or change the transmission of pain signals to the brain, which can potentially relieve your chronic migraine pain and symptoms. The ShiraTronics System is approved by the United States Food and Drug Administration for investigational (under research) use, and not approved for sale.
• at least 22 years old
• migraines started before 50 years old
• migraines occurring for at least 12 months before starting the study
• 15 - 26 headache days/month, among which ≥ 8 days has the features of probable migraine, and minimum of 2 headache-free days/month
• use of preventive(s) migraine medication for at least 3 months before starting the study
• headache or migraine other than refractory chronic migraine (RCM)
• previously implanted neurostimulator
• received botulinumtoxinA (Botox) for any other medical or cosmetic reasons requiring injections in the head, face, or neck within the past 3 months
• cervical radiofrequency ablation within 12 months
• other implanted electrical stimulation device or any metallic implant located in the head including CSF shunt and surgical clip above the shoulder line (excluding dental implants)
• women who are pregnant or breastfeeding or planning a pregnancy during participation in the study
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of ARD-101 for the Treatment of Hyperphagia in Patients with Prader-Willi Syndrome (HERO)
This study is for people who feel very hungry all the time, have trouble controlling eating (hyperphagia) and have Prader-Willi Syndrome (PWS). ARD-101 is being studied to see if it can help the body release certain gut hormones that may help reduce excessive hunger and food-seeking behaviors in people with PWS. The investigational treatment is a tablet taken by mouth and swallowed whole. The study will continue for up to 20 weeks (about 5 months).
• at least 13 years of age
• confirmed diagnosis of Prader-Willi Syndrome (PWS)
• living in a stable care setting with the same caregiver(s) for at least 6 months and one designated caregiver is willing and able to adhere to study-related procedures and is willing to participate in all study visits and complete study-related questionnaires
• females must not be pregnant when starting the study and willing to use effective birth control for 90 days after the last dose of study drug
• males engaged in sexual relations with a female of childbearing potential must utilize a highly effective method of contraception until 90 days after the last dose of study drug
• see link to clinicaltrials.gov for complete Inclusion criteria
• women who are pregnant or breastfeeding
• difficulty swallowing or inability to swallow oral medication
• significant medical or mental health diagnosis
• see link to clinicaltrials.gov for complete Exclusion criteria
MT2022-27: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
People planning to undergo a bone marrow transplant and are at risk for developing problems with the lungs related to this planned therapy. The types and seriousness of lung problems that may develop after transplant can be very different and currently, we don’t exactly know what risk factors influence who develops them or how they may respond to therapy. Also, we do not know what the best test is to monitor lung function after transplant, especially in children and young adults. The purpose of this study is to help investigators learn more about lung problems after bone marrow transplant including what is the best method for diagnosing lung problems and following how well the lungs are working. In this study, clinical information, laboratory results and imaging findings will be collected from medical records to assist researchers in learning more about lung complications after bone marrow transplant.
• up to 24 years old
• undergoing allogeneic or autologous HSCT
Phase 1b/3 global, randomized, controlled, open-label trial comparing treatment with RYZ101 to standard of care (SoC) therapy in subjects with inoperable, advanced, somatostatin receptor expressing (SSTR+), well-differentiated gastro-enteropancreatic neuroendocrine tumors (GEP-NETs) that have progressed following prior 177Lu-labelled somatostatin analogue (177Lu-SSA) therapy (ACTION-1) (ACTION-1)
The main purpose of this the study is to find out if a new investigational drug RYZ101 more effectively treats your cancer than the standard therapy, and to see if it is safe, tolerable, and to learn the pharmacokinetics (PK).
• diagnosis of inoperable, advanced, well-differentiated, somatostatin receptor expressing (SSTR+) gastroenteropancreatic neuroendocrine cancer
• able to walk and complete all self care but unable to carry out any work activities; up and about more than 50% of waking hours
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• significant cardiovascular disease such as heart failure
• hypertension that isn't controlled by medication
• uncontrolled diabetes
• history of liver cirrhosis
MT2023-51 A Phase 2 Multicenter Study of Autologous Tumor Infiltrating Lymphocytes (LN-145) in Patients with Metastatic Non-Small-Cell Lung Cancer
This study is being done to learn more about the efficacy and safety of LN-145 in participants with metastatic stage IV non-small cell lung cancer.
• confirmed diagnosis of metastatic Stage IV NSCLC without specific genomic alterations
• if the tumor has a treatable mutation(s) (other than EGFR, ALK, or ROS1 genomic alterations), 1 additional line of therapy with the appropriate targeted therapy is required
• may be restricted from strenuous activity but walking and able to carry out work of a light or sedentary nature, e.g., light house work, office work
• patients of childbearing potential or those with partners of childbearing potential must be willing to practice an approved method of highly effective birth control during treatment and up to 12 months after all protocol-related therapy
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• on systemic steroid therapy ≥ 10 mg/day of prednisone or equivalent
• have any form of primary immunodeficiency
• had another primary cancer within the previous 3 years
A Phase 3, Open-label, Multicenter, Randomized Study of Xaluritamig vs Cabazitaxel or Second Androgen Receptor-Directed Therapy in Subjects With Metastatic Castration- Resistant Prostate Cancer Previously Treated With Chemotherapy
This is a research study designed to test how well a new medication (xaluritamig) works compared to other treatments for people with metastatic castration-resistant prostate cancer. These patients have already been treated with a certain chemotherapy. Participants will be randomly assigned to one of two groups: xaluritamig or either cabazitaxel (existing cancer treatment) or another treatment chosen by the doctor. The goal of the study is to find out which treatment is more effective and safer for patients.
• diagnosis of adenocarcinoma of the prostate
• evidence of progressive disease
• completed requirements for previous treatment
• may not be able to do strenuous activity but able to walk and able to carry out work of a light or sedentary nature, e.g., light house work, office work
• see link to clinicaltrials.gov for complete inclusion criteria
• history of central nervous system (CNS) metastasis
• significant side effects from previous treatment that haven't resolved
• see link to clinicaltrials.gov for complete exclusion criteria
JAK Inhibitors to Preserve C-Peptide Production in New Onset T1D: A Phase 2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subtype-Selective JAK Inhibitors for Preservation of Pancreatic Cell Function in Newly Diagnosed Type 1 Diabetes Mellitus
This is a new type 1 diabetes onset study for ages 12-35 years old. We are looking at JAK inhibitor drugs to see if they can preserve beta cell function.
• age 12-35 years (inclusive)
• diagnosis of T1D within 100 days of first study visit
• positive for at least one islet cell autoantibody
• HbA1c no more than 10 %
• body weight at least 35kg (77 pounds)
• willing to comply with intensive diabetes management and wear a Continuous Glucose Monitoring Device (CGM)
• up to date on recommended immunizations (including flu and COVID-19)
• willing to use highly effective contraception for 3 months after the last dose of study medication
• current use of a medication that affects glucose control
• treatment with other immunosuppressive agents (including biologics or steroids), other than inhaled or topical glucocorticoids
• current history of infection (HIV, Hepatitis B, TB, herpes etc.)
• current or past use of tobacco or nicotine containing products more than the equivalent of 5 cigarettes per day
• current or past medical or mental health conditions (study staff will review)
• women who are pregnant, breast feeding, or planning to become pregnant
MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
The primary purpose of this study is to record outcomes and patient characteristics in the Cancer Center’s and BMT databases for patients who are undergoing an allogeneic (donor) hematopoietic stem cell transplant. The data will be analyzed for transplant “milestones” such as time to blood count recovery (engraftment) and how patients are doing at 3 months and 6 months after the transplant. Participation in this study will not alter treatment or medical care. All information for this study will be collected from medical records.
• up to 50 years old
• diagnosis of immunodeficiency or histiocytic disorder
• see link to clinicaltrials.gov for complete inclusion criteria
• pregnant or breastfeeding
• active, uncontrolled infection and/or HIV positive
• acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
ASSESS ALL ALS Study
We are doing this research to collect a wide range of samples, clinical information, and measurements that will be used for future research into ALS and related neurological diseases. Participants will be asked to complete 7 in-person study visits and monthly remote self-assessment activities. Access to a personal device (computer and/or smartphone or tablet) that is connected to the internet is needed to complete the monthly remote activities.
• diagnosis of Amyotrophic Lateral Sclerosis (ALS) by a physician
• access to a smartphone, computer or tablet, and internet (need not be in the home - access to a public library or other available computer with internet connection is sufficient
• for HEALTHY participants: no diagnosis of ALS , Progressive Muscular Atrophy (PMA) or Primary Lateral Sclerosis (PLS), no family history ALS/Frontotemporal Dementia (FTD) in a close family member** unless the participant has previously tested negative for the known causative ALS genes, and access to a smartphone, computer or tablet, and internet (need not be in the home - access to a public library or other available computer with internet connection is sufficient
• see link to clinicaltrials.gov
• cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, active suicidal ideation, suicide attempt, or untreated major depression <= 90 days of starting the study,
• clinically significant unstable medical condition
Brain vascular and neural function linked to balance across the adult lifespan
We are studying the relationship between cerebrovascular health and balance control associated with aging. Cerebrovascular function will be assessed with an ultrasound of cerebral blood flow and EKG done while participants exercise. We will also do an EEG with balance testing. Participants will complete two sessions, each 1.5 -2.5 hours and 2.5-3 hours in duration, 1 to 14 days apart. We will compare the results of younger, middle-aged and older adult participants.
• Age within the following 3 group age ranges, 21-30yo; middle-aged: 40-55yo; older: 65-95yo adults
• no major orthopedic disability
• vision that is 20/40 or better with or without corrective lenses
• English speaking
• able to stand for 3 minutes and walk 10 meters (33 feet) with or without an assistive device and without the assistance of another person
• see link to clinicaltrials.gov for complete Inclusion criteria
• insulin-dependent diabetes
• peripheral neuropathy
• myocardial infarction (heart attack) or symptoms of coronary artery disease within 2 years
• congestive heart failure or class IV heart failure
• any impairment affecting balance or thinking
Reducing Childhood Obesity Using Ecological Momentary Intervention (EMI) and Video Feedback at Family Meals
The proposed study is an individual three-arm randomized controlled tiled aimed at utilizing state-of-the-art intervention methods to examine whether increasing the quality and the quantity of family meals reduces childhood obesity.
• child 5-10 years old
• have a sibling who lives in the home with the child
• live in the Twin Cities Metro area
• speak English or Spanish
• child with medically necessary dietary restrictions (e.g., feeding tube)
• child is developmentally unable to participate in the study
ELEVATE, a global observational longitudinal prospective registry of patients with acute hepatic porphyria (AHP) (ELEVATE)
This is a global, multicenter, prospective, observational, longitudinal registry conducted to characterize the natural history and real-world clinical management of patients diagnosed with AHP. This protocol will not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of registry participation.
A Phase 1/2 Study of FOG-001 in Participants with Locally Advanced or Metastatic Solid Tumors (FOG-001-101)
This study is for people who have been diagnosed with cancer that is advanced (means disease has spread from the original site or has come back) or is metastatic (means the disease has spread to other parts of the body). FOG-001 is currently being developed to treat people with cancer. We want see how safe and tolerable FOG-001 is when given alone and together with other anticancer drugs at different dose levels in participants. We also want to see how FOG-001 affects the body and the cancer
• locally advanced or metastatic solid tumor
• may not be able to do physically strenuous activity but walking and able to carry out work of a light or sedentary nature, e.g., light house work, office work
• see link to clinicaltrials.gov for specific requirements by type of tumor
• history of bone metastasis except patients with mCRPC
• Osteoporosis
• uncontrolled inflammatory bowel disease (i.e., ulcerative colitis or Crohn's disease)
• women who are pregnant, breastfeeding, or planning to be come pregnant
• see link to clinicaltrials.gov for complete Exclusion criteria
MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.
• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant
Vestibular and Cortical Contributions to Transitions in Freezing of Gait and Parkinson's Disease
This study will examine the brain activation associated with the transition between movements in Parkinson’s disease, and if these changes are related to the development of problems with moving (like freezing of gait) and thinking. We will look at brain activity associated with the vestibular (inner ear) and cortical (surface of the brain) systems. People with Parkinson’s disease (especially people with freezing of gait) sometimes have difficulty initiating changes in movement types, such as going from standing to walking, turning, or switching between slow and fast repetitive movements. The difficulty in movement transitions may be associated with an overactive vestibular system (inner ear) or cortical system. In each experiment in this study, we expect about 20-25 participants with Parkinson’s disease and freezing of gait, 20-25 participants with Parkinson’s without freezing of gait, and 20-25 control participants
• 40 to 80 years old
• diagnosis of idiopathic PD with and without freezing gait
• able to walk independently without the use of an assistive device (e.g. cane) for 50 meters (about 160 feet)
• for HEALTHY OLDER ADULTS: 40 to 80 years old, able to walk independently without a cane or walker and able to perform complex activities of daily living independently
• any musculoskeletal disorder that affects the ability to stand or walk
• history of musculoskeletal disorders that significantly affect movement of lower limbs
• uncorrected visual impairment
• history of visual and/or vestibular conditions
• moderate to severe hearing impairment
• women who are pregnant
• study staff will discuss additional exclusion criteria
Imaging Core Aim 2, and Udall Project 2 Aim 2
We are conducting this research to try to find a better way of treating Parkinson's disease, specifically postural instability and gait disturbances (PIGD) by looking at certain brain characteristics using 3T magnetic resonance imaging (MRI.) We think we could discover how to make DBS more effective for such things as postural gait instability and other symptoms of PD. Participants will be asked to come to our research location, walk on a treadmill, and have a 3T MRI.
• at least 21 years old
• already have a MRI-compatible DBS device (Medtronic Percept/Percept RC DBS System) for treatment of Parkinson’s disease
• able to speak English
• women who are pregnant
• extreme claustrophobia
• unable to have a MRI
A Phase 3 Randomized, Open-label Study of MK-5684 Versus Alternative Abiraterone Acetate or Enzalutamide in Participants With Metastatic Castration-resistant Prostate Cancer (mCRPC) Previously Treated With Next-generation Hormonal Agent (NHA) and Taxane-based Chemotherapy (OMAHA-003) (OMAHA-003)
This trial is testing MK-5684 in men with metastatic castration-resistant prostate cancer (mCRPC) previously treated with next-generation Hormonal Agent (NHA) and Taxane-based chemotherapy. This trial will compare MK-5684 to abiraterone acetate and enzalutamide. Abiraterone acetate and enzalutamide are standard treatments for mCRPC. The purpose of this trial is to test the safety of the trial drug, MK-5684, see how well the drug works, and see how the body handles the drug. We also want to see if participants who get MK-5684 live longer compared to those who get abiraterone acetate or enzalutamide.
• diagnosis of adenocarcinoma of the prostate
• cancer progression while on androgen deprivation therapy with metastasis to bone or other areas of the body
• received 1 but no more than 2 taxane-based chemotherapy regimens
• unable to do strenuous activity but walking and able to carry out work of a light or sedentary nature, e.g., light house work, office work
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• unable to swallow capsules/tablets
• gastrointestinal disorder that might affect absorption
• poorly controlled diabetes mellitus
• history of clinically significant ventricular arrhythmias
• known additional cancer that is progressing or has required active treatment within the past 3 years