Condition
Location

7 Angelman Syndrome Trials

Power is an online platform that helps thousands of Angelman Syndrome 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.

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:All
Sex:All
75 Participants Needed
The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:4 - 17
Sex:All
120 Participants Needed
This trial is testing a drug called ION582, given directly into the spinal fluid, to see if it is safe for people with Angelman syndrome. The study will check how well patients tolerate different doses of the drug.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:2 - 50
Sex:All
51 Participants Needed
This trial tests Alogabat, a medication, to see if it can help children aged 5-17 with deletion Angelman Syndrome. Researchers will give the medication daily and check for improvements in brain activity over a few months.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:5 - 17
Sex:All
56 Participants Needed
This is a phase I, multicenter, non-randomized, adaptive, open-label, multiple ascending, intra-participant, dose-escalation study with a long-term extension (LTE) part and an optional open-label extension (OOE) part. The objective of the study is to investigate the safety, tolerability, PK and PD of RO7248824 administered intrathecally (IT) in participants with AS. Two linked sets of dose escalation cohorts are planned based on two different age groups, namely participants with AS aged ≥ 5 to ≤ 12 years in cohorts A1 to A5 (with at least 2 participants ≤ 8 years old in each cohort) and AS participants aged ≥ 1 to ≤ 4 years in cohorts B1 to B5. The two sets of cohorts will be run in parallel, with each cohort A1 to A5 preceding and gating the linked cohort B1 to B5 (e.g., A1 precedes B1).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:1 - 12
Sex:All
74 Participants Needed
The purpose of this study is to evaluate the effectiveness of an adapted, telehealth functional behavioral therapy (FBTsIDD) specifically focused on promoting appropriate communication and behavioral strategies in individuals with syndromic intellectual and developmental disorders. Participants will be asked to complete virtual study assessments at intake and then on a monthly basis for the duration of 3-6 months. In addition, participants will attend weekly or biweekly virtual intervention visits with a study therapist.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:2 - 12
Sex:All
92 Participants Needed
Image of trial facility.

PIXI for Neurogenetic Disorders

Research Triangle Park, North Carolina
This trial tests a program called PIXI, which helps parents of infants with genetic disorders. The program educates parents about their baby's condition, coaches them on how to interact with their baby, and provides family support. The goal is to improve the baby's development and family well-being.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Unphased
Age:< 99
Sex:All
120 Participants Needed
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Frequently Asked Questions

How much do Angelman Syndrome 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 Angelman Syndrome 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 Angelman Syndrome 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 Angelman Syndrome 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 Angelman Syndrome 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 Angelman Syndrome clinical trials ?

Most recently, we added GTX-102 for Angelman Syndrome, GTX-102 for Angelman Syndrome and Virtual Therapy for Developmental Disabilities to the Power online platform.