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Trials With No Placebo
Antisense Oligonucleotide
Antisense Oligonucleotide Therapy for Autosomal Dominant Leukodystrophy
Recruiting1 awardPhase 1 & 2
Rochester, Minnesota
This trial involves giving a personalized drug to one person with a specific genetic condition called Autosomal Dominant Leukodystrophy (ADLD) caused by a LMNB1 gene mutation.
Gene Therapy
Gene Therapy for Krabbe Disease
Recruiting1 awardPhase 1 & 2
Ann Arbor, Michigan
This 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.
Gene Therapy
Gene Therapy with FBX-101 for Krabbe Disease
Recruiting1 awardPhase 1 & 2
Pittsburgh, Pennsylvania
This trial tests a gene therapy given through an IV after a stem cell transplant in patients with Krabbe disease. The therapy uses a virus to deliver healthy genes to help fix the genetic problem. Gene therapy using viral vectors has shown some success in extending survival in mouse models of Krabbe disease.
Enzyme Replacement Therapy
Intrathecal SHP611 for Metachromatic Leukodystrophy
Recruiting1 awardPhase 2
Torrance, California
This trial is testing if SHP611, injected into the fluid around the brain and spinal cord, can help children with Metachromatic Leukodystrophy (MLD) keep their ability to move and speak for a longer time. The study will also check how well children tolerate this treatment over a long period.
Stem Cell Therapy for Inherited Metabolic Brain Diseases
Recruiting1 awardPhase 1
Durham, North Carolina
This trial is testing a new treatment for inborn errors of metabolism that includes transplanting stem cells from umbilical cords and injecting them into the spinal cord. The goal is to see if this is a safe and effective treatment for early demyelinating disease in the central nervous system.
Frequently Asked Questions
Do I need insurance to participate in a trial?
Almost all clinical trials will cover the cost of the 'trial drug' — so no insurance is required for this. For trials where this trial drug is given alongside an already-approved medication, there may be a cost (which your insurance would normally cover).
Is there any support for travel costs?
Many of the teams running clinical trials will cover the cost of transportation to-and-from their care center.
Will I know what medication I am taking?
This depends on the specific study. If you're worried about receiving a placebo, you can actively filter out these trials using our search.
How long do clinical trials last?
Some trials will only require a single visit, while others will continue until your disease returns. It's fairly common for a trial to last somewhere between 1 and 6 months.
Do you verify all the trials on your website?
All of the trials listed on Power have been formally registered with the US Food and Drug Administration. Beyond this, some trials on Power have been formally 'verified' if the team behind the trial has completed an additional level of verification with our team.
How quickly will I hear back from a clinical trial?
Sadly, this response time can take anywhere from 6 hours to 2 weeks. We're working hard to speed up how quickly you hear back — in general, verified trials respond to patients within a few days.