A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

Part of paid clinical trials in Los Angeles, California.

Sponsor
MavriX Bio, LLC
Study ID
NCT07181837
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Angelman Syndrome

Eligibility Criteria

Sex
ALL
Age
4 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • MVX-220 — GENETIC
    AAVhu68 viral vector

Study Details

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.

Key Dates

Start date
Oct 29, 2025
Status verified
Mar 2026
Primary completion
Mar 31, 2028
Completion
May 31, 2031

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1: Adults ages 18-50
    MVX-220, single dose intra cisterna magna injection
  • Experimental: Cohort 2: Children ages 4-8
    MVX-220, single dose intra cisterna magna injection
  • Experimental: Cohort 3: Optional cohort, adults and children ages 4-50
    MVX-220, single dose intra cisterna magna injection

Primary Outcome Measure

Incidence of Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest as assessed through clinical safety, laboratory tests, ECG, vital sign measurements, and physical examinations [ Time Frame: Up to Week 104 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Cedars-Sinai Medical CenterLos AngelesCalifornia90048
Alejandra Gonzalez
[email protected]
310-423-7779
Rush University Medical CenterChicagoIllinois60612
Milana Milic
[email protected]
888-352-7874
Boston Children's HospitalBostonMassachusetts02115
Abigail Ryan
[email protected]
617-355-6000

Find similar trials in Los Angeles, CA

By research site
Cedars-Sinai Medical Center· Los Angeles, CARush University Medical Center· Chicago, ILBoston Children's Hospital· Boston, MA

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