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Transcatheter Mitral Valve Replacement with the Medtronic Intrepid™ TMVR System in patients with severe symptomatic mitral regurgitation – APOLLO Trial (APOLLO)
The purpose of this study is to determine if replacing the mitral valve without open-heart surgery is as safe and effective as standard mitral valve surgery in patients with similar medical conditions. This system allows a bioprosthetic mitral valve (investigational valve) to be implanted through a long, thin, flexible tube that is inserted through an incision in the side of the chest or through an incision made in the groin area and through a vein in the leg. Participation in the study is expected to last approximately 5 years from the day the valve is implanted.
• diagnosis of moderate or severe mitral value regurgitation with symptoms
• multidisciplinary heart team thinks patient is not able to have treatment an approved transcatheter repair or conventional mitral valve procedure
• prior transcatheter mitral valve procedure with device currently implanted
• left ventricular ejection fraction <30%
Prophylactic Antibiotic Use to Prevent Urinary Tract Infection Following Radical Cystectomy and Urinary Diversion: Randomized Clinical Trial
This research is being done to determine whether not taking oral prophylactic antibiotics after surgery is less effective compared to taking oral prophylactic antibiotics after surgery in preventing urinary tract infections (UTI) within 90 days after surgery. We will divide study participants randomly (similar to tossing a coin) into two groups; one group not receiving postoperative prophylactic antibiotics and the other group receiving prophylactic antibiotics postoperatively. Both groups will receive the exact same preparation before surgery, care during the day of surgery care, postoperative care, and care after hospital discharge.
• muscle invasive bladder cancer and planning to undergo radical cystectomy with urinary diversion
• at least 18 years old
• currently receiving antibiotics for an active infection
• poor renal function
• allergic to nitrofurantoin and unable to take an alternative antibiotic (cephalexin, trimethoprim-sulfamethoxazole, or ciprofloxacin)
• women who are pregnant
An Open-label, Single Arm, Multicenter, Phase III Study on the Efficacy, Safety,and Pharmacokinetics of FP-001 42 mg Controlled Release in Patients with Central (Gonadotropin-Dependent) Precocious Puberty
The study drug FP-001 (Leuprolide mesylate) is being developed for children that are suffering from central (gonadotropin-dependent) precocious puberty (CPP). Leuprolide has been approved in the United States (US) and the European Union (EU) as treatment for prostate cancer already, and other forms of Leuprolide from other companies have been approved for the treatment of CPP. In this clinical study, Leuprolide will be used in the form of a 6-month depot injection.
• females aged 2 to 8 years or males aged 2 to 9 years old
• diagnosis of Central Precocious Puberty (CPP) within 12 months
• additional inclusion criteria (study staff will review)
• major medical or psychiatric illness that could interfere with study visits
• history of seizures, epilepsy, and/or central nervous system disorders that may be associated with seizures or convulsions
• specific prior treatments (study staff will review)
MT2019-06: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.
The purpose of this study is to evaluate the safety and ability of a transplant with your own gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. The goal is to determine if a sufficient amount of hemoglobin that prevents red blood sickling can be produced after the gene modified stem cells are returned to your body. This study may provide information on the potential usefulness of bb1111 for treatment of sickle cell disease
• must be 2 to 50 years old
• diagnosis of Sickle Cell Disease
• weigh a minimum of 6 kg (13.2 pounds)
• treated and followed for at least the past 24 months
• experienced at least 4 protocol-defined VOEs in the past 24 months
• experienced HU failure at any point in the past or must have intolerance to HU
• female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from starting the study to at least 6 months after drug product infusion.
• if allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available
• unable to receive a transfusion
• prior allogeneic transplant or gene therapy
• prior or current malignancy or immunodeficiency disorder, except cured tumors such as squamous cell carcinoma of the skin
• women who are pregnant or breast feeding
• additional exclusion criteria (study staff will review)
A Phase 1b/2, Double-Blind, Placebo-Controlled, Randomized, Parallel-Arm Study to Explore the Safety, Pharmacokinetics, and Proof of Biological Activity of DS-7011a in Patients with Systemic Lupus Erythematosus and Active Cutaneous Lupus Erythematosus
The main purpose of this study is to gather information about DS-7011a, an investigational drug that may help to treat systemic lupus erythematosus (SLE), including cutaneous lupus erythematosus (CLE). Overall study duration for a participant is expected to be approximately 28 weeks.
• Diagnosed with Lupus at least 6 months ago
• active CLE (acute, subacute, and chronic cutaneous lupus), with active skin involvement
• Body mass index (BMI) at least 18 kg/m and body weight at least 45 kg (99lbs.)
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• history of cancer in the past 2 years
• history of chronic, recurrent (3 or more of the same type of infection in 1 year) or recent serious infection including HIV, viral hepatitis, or shingles
Gender Identity Measurement In Early School Aged Children
The goal of this study is to learn more about how children (ages 5-8 years) describe their gender identities. We are also interested in learning more about the ways children think and develop. We will be giving children several tasks designed as games to assess flexibility in thinking, creativity, and the ability to take another person’s perspective.
• Ages 5 to 8
• English speaking
• unable to speak English
A Phase 1b Open-Label Multicenter Study of OP-1250 (Palazestrant) in Combination with the CDK4/6 Inhibitor Ribociclib, with the PI3K Inhibitor Alpelisib, or with the mTOR inhibitor Everolimus in Adult Subjects with Advanced and/or Metastatic ER Positive, HER2 Negative Breast Cancer
The main purpose of this study is to look at how safe and well tolerated the study drug is in combination with ribociclib (Group 1) or alpelisib (Group 2), the levels of the study drug and ribociclib or alpelisib in your blood, and how your body and your cancer respond.
• at least 18 years old
• diagnosis of advanced and/or Metastatic HR Positive, HER2 Negative Breast Cancer
• received no more than 2 prior hormonal regimens for advanced or metastatic disease
• received no more than 1 prior chemotherapy for locally advanced or metastatic breast cancer
• significant heart disease
• cerebral vascular disease within 6 months
• pulmonary embolism, or deep venous thrombosis within the last 6 months
• pneumonitis or interstitial lung disease
• history or ongoing gastrointestinal disorders that result in poor absorption of medications
• history of significant liver disease
• study staff will review medical history
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
Rituximab-PVVR followed by Abatacept Versus Rituximab-PVVR Alone In New Onset Type 1 Diabetes
This study is to see if giving the medications rituximab-pvvr (Ruxience®) followed by abatacept can help preserve remaining insulin secretion if given within three months of being diagnosed with type 1 diabetes (T1D).
• 8 to 45 years old
• diagnosis of Type 1 Diabetes within 100 days of starting the study
• positive for at least one islet cell autoantibody
• willing to comply with intensive diabetes management
• willing to wear a continuous glucose monitoring device for a minimum of 10 days every 6 months
• weigh at least 20kg (44 pounds)
• up to date on all recommended vaccinations based on age
• willing to practice public health prevention measures such as social distancing, masking, and good hand hygiene, and/or receive therapeutics such as monoclonal antibodies and antivirals as directed by the study and recommended by local health authorities to prevent SARS-Cov-2 infection
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• history of immune deficiency
• chronic active infection other than localized skin infections
• current or past HIV or Hepatitis B or active Hepatitis C infection
• women who are pregnant, breast feeding, or anticipate pregnancy
• use of oral or inhaled steroids or other immunosuppressive agents
• history of cancer
ANBL2131 - A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma
This phase III trial tests how well adding dinutuximab to induction chemotherapy along with standard of care surgery radiation and stem cell transplantation works for treating children with newly diagnosed high risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found in greater than normal amounts on some types of cancer cells. This helps cells of the immune system kill the cancer cells. Chemotherapy drugs such as cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, dexrazoxane, doxorubicin, temozolomide, irinotecan and isotretinoin, 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. During induction, chemotherapy and surgery are used to kill and remove as much tumor as possible. During consolidation, very high doses of chemotherapy are given to kill any remaining cancer cells. This chemotherapy also destroys healthy bone marrow, where blood cells are made. A stem cell transplant is a procedure that helps the body make new healthy blood cells to replace the blood cells that may have been harmed by the cancer and/or chemotherapy. Radiation therapy is also given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of induction.
• Must have a diagnosis of NBL or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines
• Newly diagnosed, HRNBL defined as one of the following: a. Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification b. Age:: greater than or equal to 547 days and INRG Stage M regardless of biologic features c. Any; age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to Stage M without systemic chemotherapy d. Age: greater than or equal to 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to Stage M without systemic chemotherapy BSA: Patients must have a BSA greater than or equal to 0.25 m2
• Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features.
• Patients ?547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features.
• Patients with known bone marrow failure syndromes.
• Patients on chronic immunosuppressive medications
• Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy.
• Female patients who are pregnant or breastfeeding their infant.
Understanding modifiable barriers to treatment adherence in youth with type 2 diabetes to inform intervention development
This study will seek to learn about barriers to management of Type 2 diabetes for children ages 10-17. This study is recruiting children, and their parent/guardian, who are managing with oral medication or insulin.
• 10 to 17 years old
• confirmed diagnosis of type 2 diabetes
• prescribed pharmacologic therapy for treatment of type 2 diabetes (oral medication or insulin)
• live with a parent/caregiver at least 50% of the time who is willing to participate in the study
• able to read and speak English
• the parent/caregiver must be the adult who spends the largest proportion of time caring for the child (or at least 50%) & able to speak and read English
• diagnosis of type 1 or monogenic diabetes
• evidence of significant cognitive deficits or a severe, persistent psychiatric disorder
Syn-Sleep Study
The purpose of the study is to evaluate the use of the Syn-One Test for identifying patients with idiopathic rapid eye movement sleep disorder (iRBD) that will progress to synucleinopathies, such as Parkinson’s disease, dementia with Lewy bodies, pure autonomic failure and multiple system atrophy. Your participation will last approximately 2 years.
• age 18 to 65
• diagnosis of Rapid Eye Movement (RBD) sleep disorder
• have repeated episodes of sleep related vocalizations and/or complex motor behaviors
• documented by sleep study to occur during REM sleep
• diagnosis of Parkinson's disease, dementia, multiple system atrophy or any other cause
• unable to have a skin biopsy because of poor wound healing, use of anticoagulants other than aspirin or Plavix, or severe vascular disease
Atherosclerosis Risk in Communities (ARIC) Study - Visits Component
The purpose of the Atherosclerosis Risk in Communities (ARIC) Study - ARIC Generation 2 research study is to evaluate the link between glucose and heart problems in adults with type 2 diabetes. Heart problems can be common in people with type 2 diabetes. We are interested in measuring your blood sugar (glucose) using a continuous glucose monitor and monitoring your heart rhythm at the same time.
• Have a diagnosis of Type II Diabetes
• Age 50-80 years old
• Be willing to wear a continuous glucose monitor and heart rhythm monitor for two weeks
• Fluent English speaker
• Pacemaker
• Allergy to adhesive tape
• Planning to move away from the Minneapolis area in the next four years
• Live in nursing home/long-term care facility
• diagnosed with dementia or unable to consent for self
Biomechanical Mechanisms of Soft Tissue Deformation during the Volleyball Spike
No current studies have examined in vivo glenohumeral kinematics during the overhead volleyball spike despite the high prevalence of spike-related shoulder pain. The extreme shoulder positions achieved by volleyball players during the spike motion may contribute to unique deformations that could result in structural change or pathology of the rotator cuff and long head biceps tendons. The purpose of this study is to determine the extent to which GH kinematics differ between SAB and a simulated volleyball spike at a self-selected position of ball contact.
• 18 to 35 years old
• played at least 2 years of high school varsity-level volleyball and/or play competitive volleyball at the club level or NCAA D3/NAIA level or above
• have no shoulder pain that has resulted in removal from, or cessation of volleyball participation in the past 6 months
• have a negative clinical screening exam
• fluent in English
• any injuries, impairments, or pain of the hitting arm that limits participation in volleyball
• shoulder pain related to the cervical spine
• history of trauma and/or surgery of the hitting shoulder
• currently pregnant.
Development of objective measures for multisensory effects on hearing
Paired sound and electrical stimulation can alter how the brain encodes sounds and the perception of sounds, as shown in emerging evidence from animal studies. The aim of this study is to develop measures for paired stimulation that can be used reliably. These measures assess the following aspects of hearing: basic hearing sensitivity and cochlear health, perception of loudness and pitch, and perception of speech and music/complex sounds. This will lead to a greater understanding in the hearing science community about how multisensory stimulation can alter activity in the auditory system and sound perception, which may lead to new techniques for improving hearing disorders.
• not users of hearing aids or implanted devices
• normal hearing or hearing-impaired, with or without tinnitus
• users of hearing aids or implanted devices
MT2023-28: A Phase 1 Basket Study Evaluating the Safety and Feasibility of T-Plex, Autologous Customized T Cell Receptor-Engineered T Cells Targeting Multiple Peptide/HLA Antigens in Participants with Antigen-positive Locally Advanced (Unresectable) or Metastatic Solid Tumors
This study aims to find out if investigational new drugs, TSC-204-A0201, TSC-204- A0702 and TSC-200-A0201, can help your cancer better than the standard of care (SOC) that are currently available and accepted by medical experts as a proper treatment. T-cells are part of the immune system and develop from stem cells in the bone marrow. They help protect the body from infection and fight cancer. For this study, T-cells will be collected through a process called leukapheresis. T-cells from your leukapheresis will be used to make the study drugs specifically tailored for you and your immune system. The purpose of the study is to learn if the study drugs are safe and effective in treating your type of cancer.
• previously received at least one line of standard systemic therapy for advanced or metastatic cancer and have either progressed, recurred, or were intolerant to the previous treatment
• unable to do physically strenuous activity but able to walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• women must not be pregnant or breastfeeding
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• known active CNS metastases
• systemic steroid therapy
• history of a bleeding disorder
• active, uncontrolled bacterial, fungal, or viral infection
• prior history or have another cancer
FAST, RELIABLE, ELECTRICAL UNCONSCIOUS DETECTION
We are interested in learning more about how preconscious thinking works. Preconscious thinking are thoughts you aren't actively thinking about but can be thought of quickly with the right trigger. It is similar to a waiting room for your thoughts. We believe that preconscious thoughts are important for learning more about symptoms related to suicide and psychosis. This research will help us better understand how the preconscious relates to these mental health outcomes. Participants will be asked to complete a research visit where they will complete computer tasks while being monitored with EEG and video recording. We'll also ask participants to complete pre-visit mood check-ins which can be done remotely.
• age 18 to 65
• Must be UMN student
• reads/understands English
• have a smartphone compatible with EMA app or willing to use study loaner phone for duration of EMA collection period (optional for initial group)
• ability to travel to research site for in-person visit at M Health Fairview University of Minnesota Medical Center - West Bank
• active military member or DoD personnel
• pregnant or possibility of being pregnant
• diagnosed with a mental illness (e.g. depression, bipolar, schizophrenia, etc.)
Brain Mechanisms of Qigong for Neuropathic Pain Relief in Adults with Spinal Cord Injury
The researchers aim to prove that Qigong practice can result in reduced or relieved neuropathic pain, improved mood, life satisfaction, self-efficacy, enjoyment to move, and community integration; and decreased fear of movement, use of medication or health care services for adults with spinal cord injury.
• 18 to 75 years old
• spinal cord injury (SCI) at least 3 months ago
• medically stable with paraplegia (T1 and below) or tetraplegia (C4 and below)
• highest level of below-level SCI-related neuropathic pain >3 on the numeric pain rating scale.
• unable to have a MRI (stabilizing hardware is typically MRI safe)
• uncontrolled seizure disorder; cognitive impairment and/or communicative disability (e.g., due to brain injury) that prevent the participant from following directions or from learning
• ventilator dependent
• pregnant or plans to become pregnant during study
• inability to perform kinesthetic imagery
Neural underpinnings of understanding speech in different listening contexts
This study evaluates the neural underpinnings of understanding speech in different listening contexts, such as listening to two talkers simultaneously or speech in background noise.
• Group 1: Adults aged 18-70 years, normal hearing, proficient in spoken English
• Group 2: Adults aged 18-70 years, have hearing loss, proficient in spoken English
• Neurological problems
• auditory neuropathy spectrum disorder (ANSD)
• Wear a cochlear implant
The Parallel Auditory Brainstem Response
This study aims to test the accuracy and speed of the pABR for future clinical use by recruiting adults with a range of hearing loss profiles from normal hearing to severe loss.
• age 18-65 years
• hearing loss (can be from none to severe)
• no history of auditory neuropathy spectrum disorder or cochlear implant use
• can remain still or sleep for the test duration
• profound hearing loss (thresholds > 80 dB HL)
• cochlear implant use
• auditory neuropathy spectrum disorder
• abnormal tympanogram
• inability to sleep or remain very still for the duration of the test
A 52-week, Double-blind, Placebo-controlled, Randomized, Phase 3 study Intended to Determine the Effects of seladelpar on normalization of Alkaline phosphatase Levels in Subjects with Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)
The purpose of this research study is to determine if seladelpar (study drug) is effective at normalizing the disease marker alkaline phosphatase in subjects with PBC who are currently on ursodeoxycholic acid (UDCA) treatment or intolerant to UDCA. This will be done by looking at changes in blood tests. This research study is also measuring whether seladelpar makes itching (pruritus) better. This will be done using information about itching that subjects enter on a device or app on their phone.
• 18 to 75 years old
• taking Ursodeoxycholic Acid (UDCA) for 12 months prior to screening (with stable dose for more than 3 months before starting the study) OR unable to take UDCA (last dose of UDCA greater than 3 months before starting the study)
• study staff will review additional inclusion and exclusion criteria
• other chronic liver diseases
• drinking more than 2 drinks per day in women and 3 drinks per day in men, or unable to quantify alcohol intake reliably
• history of cancer diagnosed or treated, active or within 2 years, or ongoing evaluation for cancer; squamous or noninvasive basal cell skin cancers and cervical carcinoma in situ are allowed (if treated)
• women who are pregnant or plan to become pregnant, or are breastfeeding
Time Restricted Eating as a Viable Alternative to Caloric Restriction for Treating Hyperglycemia in a Population with Type 2 (T2DM) diabetes (SFS3)
Feasibility study to test our overall hypothesis that time restricted eating (TRE) presents a viable alternative to caloric restriction for improving glycemic measures and reducing weight in overweight/obese patients with metformin-only treated Type 2 diabetes (T2DM).
• adults who are overweight/obese and have type 2 diabetes treated only with metformin
• 18-65 years old
• BMI between 25-40 kg/m2
• HbA1c between 6.5-8.5%
• self reported weight must be stable (+/- 5 pounds) for at least 3 months prior to the study
• own a smartphone
• women who are pregnant or are planning to become pregnant
• eating disorders
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
MT2023-20: Hematopoietic cell transplant with reduced intensity conditioning and post-transplant cyclophosphamide for severe aplastic anemia and other forms of acquired bone marrow failure.
Although allogeneic hematopoietic cell transplant (HCT) is standard treatment for severe aplastic anemia, the use of the lower intensity conditioning drugs with a personalized dosing strategy, low dose total body irradiation (TBI) with dosing based on age and bone marrow abnormalities, and use of the drug cyclophosphamide early after transplant is a newer approach. We are studying whether this new approach is safer and more effective than our previous approach.
• 0 to 75 years old
• diagnosis of Idiopathic Severe Aplastic Anemia (SAA)
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• women who are pregnant, breastfeeding or intending to become pregnant during the study
• uncontrolled infection
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.
• 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
• documented HIV infection
MT2022-44 Analysis of Autoinflammation in Chronic Granulomatous Disease Patients Undergoing Hematopoietic Cell Transplantation or Gene Therapy (PIDTC 6908)
CGD disorders are a group of diseases that cause the immune system to function abnormally, leading to infections, autoimmunity and/or inflammation that can begin early in life. CGD is usually caused by changes in certain genes in DNA. Researchers are trying to learn what types of medical problems patients with CGD have and how these respond to treatment, including bone marrow transplantation and gene therapy. Researchers also want to learn which genes cause CGD and how specific gene abnormalities lead to medical problems.
• confirmed diagnosis of Chronic Granulomatous Disease (CGD)
• to be scheduled to undergo HCT or GT within one year of enrollment
• additional inclusion criteria (study staff will review)
• .presence of other primary immunodeficiency syndromes that do not meet the clinical and laboratory criteria for CGD
• HIV infection
• past or current medical problems or findings from physical examination or laboratory testing that are not listed above which, in the opinion of the investigator, may pose additional risks from participation
MT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH
In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.
• between 0 to 3 years of age
• meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
• planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course
• patient's parent/ legal guardians are unable to provide informed consent.
Concurrent Aerobic Exercise and Cognitive Training to Prevent Alzheimer's in at-risk Older Adults (The Exergames Telerehabilitation Study)
The purpose of this study is to test the effects of an in person or home-based exergame intervention on improving cognition and fitness in community-dwelling older adults with memory complaints. The exergame refers to a new low-cost virtual reality cognitive game with cycling. We expect that participants will be in this research study for 4 months. Intervention sessions will require approximately 1 hour, 3 days per week for 3 months.
• at least 65 years old
• experiencing a memory or cognitive problem
• speak English
• no physical problems that prevent exercise
• diagnosis of dementia or mild cognitive impairment
• neurological or major psychiatric disorder, alcohol/chemical dependency or recent medical condition (anesthesia or COVID-19 brain fog) likely causing cognitive impairment
• currently enrolled in another clinical study
Humanitarian Use Device: Medtronic DBS Therapy for Dystonia (HDE #H020007) (Dystonia IRB)
Humanitarian use devices are medical devices approved by the FDA for the treatment of medical conditions affecting fewer than 4,000 patients per year. The FDA reviewed the safety of the device and determined that the probable health benefits outweigh the risks of injury or illness from its use. Effective treatment of symptoms, however, has not been studied in formal clinical trials. Medtronic DBS Therapy delivers electrical stimulation to areas in the brain to help control symptoms of various movement disorders. You may be a candidate for Medtronic DBS Therapy for Dystonia if you have been diagnosed with chronic, intractable (drug refractory) primary dystonia. You or your third party payer (health insurance, Medicare, Medicaid or other) must provide payment for hospital, office and other medical costs related to this therapy.
• 7 to 99 years old
• diagnosed with chronic, intractable (drug refractory) primary dystonia determined by a neurologist or neurosurgeon
• patients who are at significant surgical risk as determined by the neurosurgeon and/or anesthesiologist
• patients who have not had an adequate trial of medical or non-surgical treatment
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.
• 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
• history of dementia
• women who are pregnant or breastfeeding
• other exclusion criteria (study staff will review)