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A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

Methylmalonic Acidemia Clinical Trial

Official title:
A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Clinical Trial 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, pharmacokinetics, and pharmacodynamics of mRNA-3705.

Clinical Trial Description

This study comprises 2 parts: Dose Optimization part (Part 1) followed by a Dose Expansion part (Part 2). The study is designed to evaluate multiple doses and dosing intervals of mRNA-3705. In both parts, after confirmation of eligibility, participants will enter an Observation Period (48 to 72 hours pre-dose) in Part 1 and 24 hours before dose 1 in Part 2), followed by the Treatment Period. Participants who complete the Treatment Period, including the End of Treatment (EOT) Visit, are offered participation in the mRNA-3705 extension study. If the participant chooses to participate and meets eligibility criteria, they will be enrolled in the extension study; otherwise, they will transition to the follow-up part of the study (approximately 2-year follow-up in Part 1 and 6-months follow-up in Part 2). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04899310
Study type Interventional
Source ModernaTX, Inc.
Contact Moderna Clinical Trials Support Center
Phone 1-877-777-7187
Email clinicaltrials@modernatx.com
Status Recruiting
Phase Phase 1/Phase 2
Start date August 6, 2021
Completion date August 1, 2028
View Details
See also
  Status Clinical Trial Phase
Enrolling by invitation NCT05842837 - A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA
Completed NCT01599286 - Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Phase 2
Completed NCT02426775 - Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia Phase 3
Terminated NCT04581785 - Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia Phase 1/Phase 2
Recruiting NCT05295433 - An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 Phase 1/Phase 2
Terminated NCT04836494 - A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia Phase 1
Completed NCT03484767 - "The MaP Study": Mapping the Patient Journey in MMA and PA
Terminated NCT04732429 - Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Phase 2
Recruiting NCT00078078 - Clinical and Laboratory Study of Methylmalonic Acidemia
Recruiting NCT05040178 - An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Active, not recruiting NCT05506254 - Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
Recruiting NCT04176523 - Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Recruiting NCT01289158 - Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study N/A
Withdrawn NCT01341379 - Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate Phase 2
Terminated NCT05438485 - Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia