- Anxiety
- Depression
- Alzheimer's Disease
- Weight Loss
- Heart Disease
- Cancer
- Asthma
Trial Phase
Trial Status
Paid Participation
Clear All
10 Lysosomal Storage Disorder Trials Near You
Power is an online platform that helps thousands of Lysosomal Storage Disorder 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.
Learn More About PowerGene Therapy with FBX-101 for Krabbe Disease
Trial Details
Key Eligibility Criteria
6 Participants Needed
RGX-121 Gene Therapy for Hunter Syndrome
Trial Details
Key Eligibility Criteria
48 Participants Needed
Gene Therapy for Fabry Disease
Trial Details
Key Eligibility Criteria
18 Participants Needed
Gene Therapy for Krabbe Disease
Trial Details
Key Eligibility Criteria
9 Participants Needed
AAV9 Vector Gene Therapy for GM1 Gangliosidosis
Trial Details
Key Eligibility Criteria
54 Participants Needed
IMT Therapy for Pompe Disease
Trial Details
Key Eligibility Criteria
29 Participants Needed
Gene Therapy for Danon Disease
Trial Details
Key Eligibility Criteria
12 Participants Needed
DFT383 for Cystinosis
Trial Details
Key Eligibility Criteria
30 Participants Needed
Gene Therapy for Tay-Sachs and Sandhoff Diseases
Trial Details
Key Eligibility Criteria
11 Participants Needed
Stem Cell Transplantation for Osteopetrosis
Trial Details
Key Eligibility Criteria
100 Participants Needed
Why Other Patients Applied
"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."
"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."
"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."
Know someone looking for new options?
Spread the word
Learn More About Power
We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.
Frequently Asked Questions
How much do Lysosomal Storage Disorder 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 Lysosomal Storage Disorder 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 Lysosomal Storage Disorder 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 Lysosomal Storage Disorder 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 Lysosomal Storage Disorder 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 Lysosomal Storage Disorder clinical trials?
Most recently, we added DFT383 for Cystinosis, Gene Therapy for Danon Disease and Gene Therapy for Krabbe Disease to the Power online platform.