Status:
RECRUITING
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Lead Sponsor:
MavriX Bio, LLC
Conditions:
Angelman Syndrome
Eligibility:
All Genders
4-50 years
Phase:
PHASE1
PHASE2
Brief Summary
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 cente...
Detailed Description
MVX-220 is an investigational gene replacement therapy intended to provide a functional copy of the UBE3A gene to individuals with Angelman syndrome. This study is designed to evaluate the safety, tol...
Eligibility Criteria
Inclusion
- Key
- The participant's parent/legal guardian must provide written informed consent.
- Symptoms consistent with AS and documented genetic confirmation of one of the following genotypes resulting in a diagnosis of AS:
- Full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13
- Uniparental disomy
- Imprinting center defect
- The participant must be 18 to 50 years of age, inclusive (for adult participants), or 4 to 8 years of age, inclusive (for pediatric participants), at Screening.
- The participant must have the ability to ambulate independently.
- The participant must be on stable antiepileptic medications (with no changes within 1 month prior to the Screening visit, except for weight associated dose adjustments).
- Key
Exclusion
- Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for participation.
- Laboratory abnormalities including but not limited to:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN)
- Total and/or fractionated bilirubin (direct and/or indirect) \> ULN
- Gamma-glutamyl transferase (GGT) \> ULN
- Estimated glomerular filtration rate (eGFR) below the lower limit of normal (LLN) for age
- Hemoglobin \< 8 g/dL
- White blood cell (WBC) count outside the normal range for age
- Platelet count \< LLN
- Partial thromboplastin time (PTT) outside the reference range
- PT/International normalized ratio (INR) outside the reference range
- Any known history and/or family history of hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) or multisystem inflammatory syndrome (MIS).
- Any known history and/or family history of disordered complement function and/or complement gene mutation(s).
- History of systemic lupus erythematous, Still's disease, rheumatoid arthritis, and/or other severe autoimmune conditions per judgment of the Investigator.
- Any known history of thrombotic microangiopathy (TMA)/microangiopathic hemolytic anemia, or hypercoagulable conditions including, but not limited to, disseminated intravascular coagulation (DIC), deep venous thrombosis, and pulmonary embolism.
- Current therapy with high dose immunosuppressants.
- Prior or current treatment with an investigational drug within 6 months or 5-half-lives of the hospital admission whichever is longer.
- Prior treatment with an antisense oligonucleotide within 1 year of hospital admission.
- A history of gene therapy administration.
- Any contraindication to ICM administration procedure, including contraindications to imaging, contrast use, anesthesia, or any condition that would increase the risk of adverse outcomes from the ICM procedure.
- Any contraindication to glucocorticoid use
Key Trial Info
Start Date :
October 29 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 31 2031
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT07181837
Start Date
October 29 2025
End Date
May 31 2031
Last Update
November 13 2025
Active Locations (2)
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1
Rush University Medical Center
Chicago, Illinois, United States, 60612
2
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104