A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Recruiting
Phase 1
Phase 2
Interventional Study
Angelman Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 4 – 50
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 12 (estimated)
- Sponsor
- MavriX Bio, LLC · Industry
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About This Trial
The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
1. The participant's parent/legal guardian must provide written informed consent.
2. Symptoms consistent with AS and documented genetic confirmation of one of the following genotypes resulting in a diagnosis of AS:
1. Full maternal UBE3A gene deletion causing AS in the r…
Contacts