Filter Results
Conditions
Locations
Treatment Type
Trial Phase
Trial Status
Paid Participation
Clear All
14 Leukoencephalopathy Trials
Power is an online platform that helps thousands of Leukoencephalopathy patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Gene Therapy for Krabbe Disease
Ann Arbor, MichiganThis trial tests a single dose of a virus carrying a healthy gene in patients with severe forms of Krabbe disease. The virus helps by delivering the healthy gene to the patient's cells. Gene therapy has shown promise in extending survival in previous studies.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:< 18
Sex:All
9 Participants Needed
Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders
Pittsburgh, PennsylvaniaThe objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 55
Sex:All
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others
100 Participants Needed
Gene Therapy for Canavan Disease
Chicago, IllinoisThe main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:< 30
Sex:All
Key Eligibility Criteria
Disqualifiers:Anti-AAV9 Antibodies, Prior Gene Therapy, Others
Must Not Be Taking:Immunosuppressants, Anti-epileptics
26 Participants Needed
NT-I7 for Progressive Multifocal Leukoencephalopathy
Bethesda, MarylandBackground:
Progressive multifocal leukoencephalopathy (PML) is a brain infection. It is caused by a virus. PML can happen in people with a weakened immune system. PML is associated with cognitive and visual impairment as well as motor and speech disturbances. There is no treatment for PML. Researchers want to see if a new drug can help.
Objective:
To see if the drug NT-I7 can help increase lymphocyte numbers, which may help control PML infection.
Eligibility:
Adults ages 18 and older with PML who are enrolled in Protocol #13-N-0017.
Design:
Participants will be screened under Protocol #13-N-0017.
Participants will have a 7-day inpatient stay, outpatient visits, and follow-up phone calls.
Participants will have a medical history and physical exam. They will give urine samples. Blood will be drawn from an arm vein or through an intravenous (IV) catheter.
Participants will get up to 3 doses of NT-I7. It will be given by injection into the muscle.
Participants will have lumbar punctures ( spinal taps ). A thin needle will be inserted into the spinal canal in the lower back. Cerebrospinal fluid will be removed. X-ray may be used to guide the procedure.
Participants will have magnetic resonance imaging (MRI) of the brain. The MRI scanner is a metal cylinder surrounded by a magnetic field. During MRIs, participants will lie on a table that slides in and out of the scanner. Soft padding or a coil will be placed around their head. They will get gadolinium, a contrast agent, through an IV catheter.
Participation will last for 12 to 19 months.
...
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Autoimmune CNS, Immune-mediated Vital Organ, Others
Must Not Be Taking:Immune-suppressive Medications
12 Participants Needed
Radioactive Tracer for Multiple Sclerosis
Bethesda, MarylandBackground:
Multiple sclerosis (MS) and progressive multifocal leukoencephalopathy (PML) are disorders that affect the central nervous system (CNS). The CNS includes the brain, spinal cord, and optic nerves. Both diseases can cause muscle weakness and impair vision, speech, and coordination. Researchers are working to better understand how MS and PML affect the CNS.
Objective: To test whether an experimental radioactive tracer (minibody) can help positron emission tomography (PET) scans detect certain immune cells in the CNS of people with MS and PML.
Eligibility:
People aged 18 years and older with MS or PML.
Design:
Participants will come to the clinic for at least 3 visits over 4 to 6 weeks.
Participants will undergo testing. They will have a physical and neurological exam. They will have blood tests and tests of their heart function. They will have a magnetic resonance imaging (MRI) scan of the brain. They may have a spinal tap: Their lower back will be numbed, and a needle will be inserted between the bones of the spine to withdraw fluid from around the spinal cord.
Minibody is given through a tube with a needle placed in a vein in the arm. This takes 5 to 10 minutes. Participants will have heart function tests before and after receiving the minibody.
Participants will return the next day for the PET scan. They will lie on a table that moves through a doughnut-shaped machine. This scan will take about 1 hour.
Participants with PML may opt to repeat the minibody infusion and the PET scan within 6 months.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnant, Lactating, Severe Claustrophobia, Others
10 Participants Needed
Home Exercise for Neurodegenerative Disease
Baltimore, MarylandThe primary goal of this study is to address the need for targeted therapeutic interventions for impairments that impact walking in related neurodegenerative diseases.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:5 - 75
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Hypertension, Diabetes, Dementia, Others
30 Participants Needed
Crizanlizumab for RVCL
Saint Louis, MissouriThis trial tests crizanlizumab, a medication that prevents blood cells from sticking to vessel walls, in patients with a rare and fatal condition called RVCL. The goal is to see if it can reduce brain and eye damage by preventing small blood vessel blockages. Crizanlizumab is a monoclonal antibody developed by Novartis Pharmaceuticals for the prevention of vaso-occlusive crises in patients with sickle cell disease.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:25 - 75
Sex:All
20 Participants Needed
ABBV-CLS-7262 for Vanishing White Matter Disease
Philadelphia, PennsylvaniaThis trial is testing a new drug called ABBV-CLS-7262 in adults and children aged 6 years or older who have Vanishing White Matter disease. The study will last for almost two years and will involve frequent medical check-ups to see if the drug helps improve their condition.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:6+
Sex:All
Key Eligibility Criteria
Disqualifiers:Respiratory Support, Uncontrolled Seizures, Pregnancy, Others
50 Participants Needed
VGL101 for Leukoencephalopathy
Philadelphia, PennsylvaniaThis trial is testing iluzanebart, a medication given through infusions, in adults with a specific genetic mutation causing ALSP, a rare brain disease. The goal is to see if the medication is safe and can slow down the disease by looking at brain scans and other health markers. Participants will receive the treatment regularly for a year, with an option to continue for additional years.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
20 Participants Needed
SBT101 Gene Therapy for Adrenomyeloneuropathy
Worcester, MassachusettsThis is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years.
Patients will receive a single dose of SBT101 via IT route (or an imitation procedure) and will be followed for safety and efficacy for 2 years. Patients receiving SBT101 will be followed for an additional 3 years (5 total) for Safety. Patients receiving an imitation procedure will be offered the opportunity to receive SBT101 after 2 years, as data indicate.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 65
Sex:Male
Key Eligibility Criteria
Disqualifiers:Inflammatory Cerebral Disease, Diabetes, Others
Must Not Be Taking:Steroids, Sirolimus, Tacrolimus, Anesthetics
16 Participants Needed
Stem Cell Transplantation for Osteopetrosis
Minneapolis, MinnesotaThis trial tests a treatment using busulfan and fludarabine to prepare patients with certain genetic disorders for a bone marrow transplant. The goal is to safely replace their bone marrow with healthy donor cells by carefully monitoring drug levels. Busulfan is used to clear out bone marrow cells before the transplant, and fludarabine is being tested as a less harmful alternative to another drug.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 55
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Myeloablative Chemotherapy, Infections, Others
100 Participants Needed
Cilostazol for Cerebral Small Vessel Disease
Jacksonville, FloridaThis trial aims to test cilostazol, a medication that prevents blood clots and improves blood flow, in older adults with cerebral small vessel disease (SVD). The study will evaluate if cilostazol can slow the progression of SVD by improving blood flow and reducing inflammation. Participants will undergo various tests to measure changes in their condition over time.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnant, Breastfeeding, MRI Intolerance, Others
100 Participants Needed
This phase I trial tests the feasibility and safety of genetically modified cytotoxic T-lymphocytes in controlling infections caused by adenovirus (ADV), BK virus (BKV), cytomegalovirus (CMV), JC virus (JCV), or COVID-19 in immunocompromised patients with cancer. Viral infections are a leading cause of morbidity and mortality after hematopoietic stem cell transplantation, and therapeutic options for these infections are often complicated by associated toxicities. Genetically modified cytotoxic T-lymphocytes (CTLs) are designed to kill a specific virus that can cause infections. Depending on which virus a patient is infected with (ADV, BKV, CMV, JCV, or COVID-19), the CTLs will be designed to specifically attack that virus. Giving genetically modified CTLs may help to control the infection.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
30 Participants Needed
Cytotoxic T Lymphocytes for Virus-Related Cancer
Houston, TexasThis phase II trial studies how well donor cytotoxic T lymphocytes work in treating patients with malignancies with BK and/or JC virus. Cytotoxic T lymphocytes are made from donated blood cells that are grown in the laboratory and are designed to kill viruses that can cause infections in transplant patients and may be an effective treatment in patients with malignancies with BK and/or JC virus.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:All
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infections, Active Acute GVHD, Others
Must Be Taking:Antivirals
100 Participants Needed
Page 1 of 1+
Frequently Asked Questions
How much do Leukoencephalopathy clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Leukoencephalopathy clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Leukoencephalopathy trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Leukoencephalopathy is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Leukoencephalopathy medical study ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Leukoencephalopathy clinical trials ?
Most recently, we added Radioactive Tracer for Multiple Sclerosis, ABBV-CLS-7262 for Vanishing White Matter Disease and Home Exercise for Neurodegenerative Disease to the Power online platform.