Actively Recruiting
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Led by MavriX Bio, LLC · Updated on 2026-03-16
12
Participants Needed
3
Research Sites
291 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parent or legal guardian provides written informed consent
- Diagnosis of Angelman syndrome with documented genetic confirmation of either full maternal UBE3A gene deletion, uniparental disomy, or imprinting center defect
- Age 18 to 50 years for adults or 4 to 8 years for children at screening
- Ability to walk independently
- Stable antiepileptic medication regimen with no changes within 1 month prior to screening except for weight-based dose adjustments
You will not qualify if you...
- Presence of any clinically significant medical condition other than Angelman syndrome that would make participation unsuitable
- Abnormal laboratory values including elevated liver enzymes (ALT or AST), elevated bilirubin, elevated gamma-glutamyl transferase, reduced kidney function, low hemoglobin (<8 g/dL), abnormal white blood cell count, low platelet count, abnormal coagulation times (PTT, PT/INR)
- Personal or family history of hemophagocytic lymphohistiocytosis, macrophage activation syndrome, or multisystem inflammatory syndrome
- Personal or family history of complement system disorders or complement gene mutations
- History of severe autoimmune diseases such as systemic lupus erythematosus, Still's disease, or rheumatoid arthritis
- History of thrombotic microangiopathy, microangiopathic hemolytic anemia, or hypercoagulable conditions including disseminated intravascular coagulation, deep vein thrombosis, or pulmonary embolism
- Current treatment with high dose immunosuppressants
- Treatment with an investigational drug within the past 6 months or 5 half-lives of hospital admission, whichever is longer
- Treatment with antisense oligonucleotides within 1 year of hospital admission
- Prior gene therapy administration
- Contraindications to intra-cisterna magna injection or related procedures, including imaging, contrast use, anesthesia, or conditions increasing procedural risk
- Contraindications to glucocorticoid use
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
Actively Recruiting
2
Rush University Medical Center
Chicago, Illinois, United States, 60612
Actively Recruiting
3
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
Research Team
M
MavriX Bio, LLC
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
3
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