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.
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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

MavriX Bio, LLC

978-538-8554

info@mvxbio.com

CONTACT