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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

Methylmalonic Acidemia Clinical Trial

Official title:
A Phase 1/2, Global, Open-Label, 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

Clinical Trial Summary

The primary objective of this study is to evaluate the long-term safety of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in other clinical studies of mRNA-3705.

Clinical Trial Description

Participants with isolated MMA due to MUT deficiency who were previously enrolled in other clinical studies of mRNA-3705 will have the option to enroll into this extension study provided all eligibility criteria have been met. The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (up to 2 years after the last dose of study drug). Treatment Period will continue unless one of the following occurs: mRNA-3705 receives marketing approval and reimbursement in the country of origin of the participant (following market approval and access being in place, all participants who wish to continue treatment will be offered mRNA-3705 through market access, with the intent to prevent treatment interruption. Safety monitoring will be performed for all participants under treatment per market access requirements), the participant discontinues study drug, the participant is no longer receiving clinical benefit (in the opinion of the Investigator), or Sponsor discontinues the development of mRNA-3705. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05295433
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 March 8, 2022
Completion date April 2, 2034
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
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
Recruiting NCT04899310 - A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Phase 1/Phase 2
Terminated NCT05438485 - Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia