Trials / Withdrawn
WithdrawnNCT03810690
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia
A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
- Status
- Withdrawn
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- ModernaTX, Inc. · Industry
- Sex
- All
- Age
- 1 Year
- Healthy volunteers
- Not accepted
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 of age with elevated plasma methylmalonic acid. The study is designed to characterize baseline biomarker levels followed by assessment of safety, pharmacokinetics, and pharmacodynamics of different doses of mRNA-3704 in patients affected by MMA as part of the Dose Escalation phase. During the Dose Escalation phase, three dose levels of mRNA-3704 are planned to be investigated in this study among patients with MMA due to MUT deficiency: low dose, mid dose, and high dose. An additional cohort to evaluate a fourth dose level may be considered jointly by the independent SMC and the Sponsor. Upon establishment of a dose with acceptable safety and pharmacodynamic activity, additional patients will be enrolled in a Dose Expansion phase to allow for further characterization of the safety and pharmacodynamics of mRNA-3704. Patients in both phases of study will participate in a pre-dosing observational period, followed by a treatment period, and then a follow-up period after withdrawal of treatment.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | mRNA-3704 | mRNA-3704 dispersion for intravenous (IV) infusion |
Timeline
- Start date
- 2019-05-28
- Primary completion
- 2020-08-18
- Completion
- 2020-08-18
- First posted
- 2019-01-22
- Last updated
- 2020-11-13
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03810690. Inclusion in this directory is not an endorsement.