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NCT04899310 · ClinicalTrials.gov registry record · Phase 1
A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
A Phase 1 study of Methylmalonic Acidemia, sponsored by ModernaTX.
- Active
- Registry status
- Phase 1
- Development phase
- 74
- Enrollment target
- 11
- Study locations
NCT04899310: Active Phase 1 study of Methylmalonic Acidemia, sponsored by ModernaTX.
NCT04899310 is a Phase 1 study of Methylmalonic Acidemia that is active but no longer recruiting, run by ModernaTX. The registered enrollment target is 74 participants, below the 415-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (82% lower). The trial reports 11 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04899310, a Phase 1 study of Methylmalonic Acidemia, is active but no longer recruiting, sponsored by ModernaTX.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 74 participants
- Enrollment target
- 11
- Study locations
Study Summary
This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenously (IV)-infused mRNA-3705.
Conditions Studied
Interventions
- BIOLOGICAL Placebo
- BIOLOGICAL mRNA-3705
Study Locations (11)
Other
- Hôpital Necker - Enfants Malades - Paris
- Erasmus MC - Rotterdam
- Universitair Medisch Centrum Utrecht - Utrecht
- Hospital Universitario Cruces - Barakaldo
- Hospital Universitario 12 de Octubre - Madrid
- Royal Manchester Children's Hospital - Manchester
California
- UCLA Medical Center - Los Angeles
- Lucile Packard Children's Hospital at Stanford - Palo Alto
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Alberta
- Stollery Children's Hospital University of Alberta - Edmonton
Ontario
- Hospital For Sick Children - Toronto
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 74 participants |
| Start Date | 2021-08-06 |
| Est. Completion | 2028-07-01 |
| Phase | Phase 1 |
What the registry record for NCT04899310 still lists
NCT04899310 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 74 participants, a relatively small participant target, below the 415-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (82% lower).
The record links to 1 condition, with Methylmalonic Acidemia appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04899310 lists 11 locations in 5 states (Other, California, Pennsylvania).
Frequently Asked Questions
What is clinical trial NCT04899310 about?
NCT04899310 is a clinical study titled "A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia". This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenously (IV)-infused mRNA-3705.
What is the current status of trial NCT04899310?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 74 participants. The study started on 2021-08-06. Estimated completion is 2028-07-01.
What conditions does trial NCT04899310 study?
This clinical trial studies the following conditions: Methylmalonic Acidemia.
What interventions are being tested in trial NCT04899310?
The interventions under investigation include: Placebo (BIOLOGICAL), mRNA-3705 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04899310?
This trial is sponsored by ModernaTX, which has 90 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04899310 being conducted?
This trial has 11 study locations across California, Pennsylvania, Alberta, Ontario. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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