Status:
WITHDRAWN
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia
Lead Sponsor:
ModernaTX, Inc.
Conditions:
Methylmalonic Acidemia (MMA)
Metabolism, Inborn Errors
Eligibility:
All Genders
1+ years
Phase:
PHASE1
PHASE2
Brief Summary
This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3704 in patients with methylmalonic acidemia/aciduria (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency between 1 to 18 years o...
Eligibility Criteria
Inclusion
- Patients are eligible to be included in the study only if all of the following criteria apply:
- Confirmed diagnosis of isolated MMA due to MUT deficiency based on the following criteria:
- Elevated plasma methylmalonic acid concentrations (≥ 100 µmol/L)
- Presence of normal serum/plasma Vitamin B12 and plasma homocysteine levels
- Confirmed diagnosis by molecular genetic testing
- Patient must be ≥ 1 year of age at the time of consent/assent (Inclusion of the first three patients will be restricted to individuals age ≥ 8 years)
Exclusion
- Patients are excluded from the study if any of the following criteria apply:
- Diagnosis of isolated MMA cblA, cblB, or cblD enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combined MMA with homocystinuria
- History of organ transplantation
- Previously received gene therapy for the treatment of MMA.
- Estimated glomerular filtration rate (GFR) \< 30 mL/min/1.73 m2; or patients who receive chronic dialysis
Key Trial Info
Start Date :
May 28 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 18 2020
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT03810690
Start Date
May 28 2019
End Date
August 18 2020
Last Update
November 13 2020
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