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Trials With No Placebo
RNA-based Therapy
mRNA-3745 for Glycogen Storage Disease
Recruiting1 awardPhase 1 & 2
Farmington, Connecticut
This trial is testing a new treatment called mRNA-3745, which uses messenger RNA to help the body produce a missing protein. It is aimed at adults and children with Glycogen Storage Disease Type Ia (GSD1a). The study will check if the treatment is safe and how well it works.
Enzyme Replacement Therapy
Cipaglucosidase Alfa + Miglustat for Pompe Disease
Recruiting2 awardsPhase 3
Gainesville, Florida
This trial will study the safety, how well it works, and side effects of an experimental drug called cipaglucosidase alfa/miglustat in children with a disease called classic infantile-onset Pompe disease.
Gene Therapy
Gene Transfer for Pompe Disease
Recruiting1 awardPhase 1 & 2
Phoenix, Arizona
This trial is testing a new treatment called SPK-3006 for adults with late-onset Pompe disease who are already on enzyme replacement therapy. The treatment involves an infusion that delivers a gene to help produce a missing enzyme. The goal is to see if this new approach is safe and effective.
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.