A Phase 1/2 interventional study of mRNA-3704 in Methylmalonic Acidemia (MMA) and Metabolism, Inborn Errors, sponsored by ModernaTX, Inc.. Withdrawn. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2020-11-13.
Sponsored by ModernaTX, Inc. · Phase 1/2, Interventional, and Treatment
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.
107 studies on the registry are indexed under Metabolism, Inborn Errors; 23 are open to participants now.
Browse Metabolism, Inborn Errors studies →ModernaTX, Inc. is the lead sponsor of 112 studies on the registry; 14 are open to participants now.
Of its 60 completed or terminated interventional studies of FDA-regulated products, 32 (53%) have results posted.
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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:
Exclusion Criteria:
Patients are excluded from the study if any of the following criteria apply:
mRNA-3704
Biological: mRNA-3704
mRNA-3704
Biological: mRNA-3704
mRNA-3704
Biological: mRNA-3704
mRNA-3704
Biological: mRNA-3704
Biological: mRNA-3704
mRNA-3704 dispersion for intravenous (IV) infusion
Incidence of treatment-emergent adverse events
Time frame: Day 1 (initial mRNA-3704 dose) through 52 weeks after final mRNA-3704 dose
Change in plasma methylmalonic acid levels
Baseline (pre-dose levels) to post-dose levels measured after single and after repeated administrations of mRNA-3704
Time frame: Week -4 through 36 weeks after initial mRNA-3704 dose
Maximum observed concentration (Cmax) after administration of mRNA-3704
Time frame: Baseline through 36 weeks after initial mRNA-3704 dose
Time of Cmax (Tmax)
Time frame: Baseline through 36 weeks after initial mRNA-3704 dose
Area under the plasma concentration-time curve (AUC)
Time frame: Baseline through 36 weeks after initial mRNA-3704 dose
Change in plasma 2-methylcitrate levels
Baseline (pre-dose levels) to levels measured after single and after repeated administrations of mRNA-3704
Time frame: Week -4 through 36 weeks after initial mRNA-3704 dose
Measurement of anti-PEG antibodies
Time frame: Pre-dose through up to 52 weeks after final mRNA-3704 dose
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is withdrawn, as verified in Nov 2020. You cannot join it, but the record below documents what was studied.
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ModernaTX, Inc.