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Why We Started 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.

Bask
Bask GillCEO at Power
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      Why We Started 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.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      14 Fabry Disease Trials Near You

      Power is an online platform that helps thousands of Fabry Disease 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 Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      Venglustat for Fabry Disease

      Fairfax, Virginia
      This trial is testing a medication called venglustat to see if it can help reduce pain in people with Fabry disease. The medication works by targeting the root causes of nerve and abdominal pain.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:16+

      Key Eligibility Criteria

      Disqualifiers:Cardiovascular Disease, Hepatitis, HIV, Others
      Must Not Be Taking:Cytochrome P450 Drugs

      122 Participants Needed

      Migalastat for Fabry Disease

      Fairfax, Virginia
      An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:2 - 11

      Key Eligibility Criteria

      Disqualifiers:Renal Impairment, Kidney Disease, Others
      Must Not Be Taking:Miglitol, Miglustat

      8 Participants Needed

      Venglustat for Fabry Disease

      Fairfax, Virginia
      This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy. * Study visits will take place approximately every 3 to 6 months * Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:18 - 65

      Key Eligibility Criteria

      Disqualifiers:Stroke, Heart Failure, Seizures, Others
      Must Not Be Taking:CYP3A4 Inducers/inhibitors

      104 Participants Needed

      Migalastat for Fabry Disease

      Fairfax, Virginia
      An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Kidney Transplant, Peritoneal Dialysis, Others
      Must Not Be Taking:Miglitol, Agalsidase, Miglustat

      14 Participants Needed

      Long-term Safety of Lucerastat for Fabry Disease

      Fairfax, Virginia
      This trial is testing the safety and tolerability of a medication called lucerastat, which is taken by mouth. It is aimed at adults with Fabry disease, a condition where harmful fats build up in the body. Lucerastat helps to reduce these fat levels.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Acute Kidney Injury, Stroke, Others

      107 Participants Needed

      Pegunigalsidase Alfa for Fabry Disease

      Fairfax, Virginia
      The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Medical, Emotional, Behavioral, Psychological, Others

      29 Participants Needed

      Pegunigalsidase Alfa for Fabry Disease

      Fairfax, Virginia
      The objective of CLI-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:18 - 60

      Key Eligibility Criteria

      Disqualifiers:Medical, Emotional, Behavioral, Psychological, Others

      97 Participants Needed

      Gene Therapy for Fabry Disease

      Fairfax, Virginia
      This trial tests ST-920, a treatment using a virus to deliver a gene that helps produce an important enzyme in patients with Fabry disease. The goal is to help these patients by continuously making the enzyme to reduce harmful substances in their bodies. ST-920 is a gene therapy treatment for Fabry disease, which aims to deliver a gene to produce the enzyme alpha-galactosidase A, addressing the enzyme deficiency central to the disease.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      34 Participants Needed

      AMT-191 for Fabry Disease

      Fairfax, Virginia
      This is an open-label, multi-center study to evaluate safety, tolerability, and exploratory efficacy of a single dose of intravenously-administered AMT-191. The plan is to investigate 2 sequential dose cohorts with 3-6 Participants per cohort. Participants will continue receiving regularly scheduled enzyme replacement therapy (ERT) until they meet the criteria for withdrawal.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:18 - 50
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Allergy To ERT, Proteinuria, Malignancy, Others
      Must Be Taking:Enzyme Replacement Therapy

      12 Participants Needed

      Gene Therapy for Fabry Disease

      Fairfax, Virginia
      This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Kidney Transplant, HIV, Diabetes, Others
      Must Be Taking:ERT

      18 Participants Needed

      HM15421 for Fabry Disease

      Fairfax, Virginia
      This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patients with FD.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Dialysis, Stroke, HIV, Others
      Must Not Be Taking:ACE Inhibitors, ARBs

      18 Participants Needed

      Shorter Infusion of Fabrazyme for Fabry Disease

      Fairfax, Virginia
      This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Age:2 - 65

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      18 Participants Needed

      Multiparametric MRI for Fabry Cardiomyopathy

      Toronto, Ontario
      This study will evaluate whether cardiac MRI T1 and T2 mapping improves our ability to detect early abnormalities in the heart in patients with Fabry disease and identify patients at increase risk of adverse events.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Myocardial Infarction, MRI Contraindication

      250 Participants Needed

      PRX-102 for Fabry Disease

      Cincinnati, Ohio
      A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2, 3
      Age:2 - 17

      Key Eligibility Criteria

      Disqualifiers:Acute Kidney Injury, Dialysis, Malignancy, Cardiomyopathy, Others
      Must Be Taking:ERT Treatment

      22 Participants Needed

      Why Other Patients Applied

      "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."

      IZ
      Healthy Volunteer PatientAge: 38

      "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."

      WR
      Obesity PatientAge: 58

      "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."

      HZ
      Arthritis PatientAge: 78

      "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."

      AG
      Paralysis PatientAge: 50

      "I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

      ZS
      Depression PatientAge: 51
      Match to a Fabry Disease Trial

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      Why We Started 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.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?
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      Frequently Asked Questions

      How much do Fabry Disease 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 Fabry Disease 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 Fabry Disease 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 Fabry Disease 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 Fabry Disease 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 Fabry Disease clinical trials?

      Most recently, we added Migalastat for Fabry Disease, HM15421 for Fabry Disease and PRX-102 for Fabry Disease to the Power online platform.