Long-term evaluation of mRNA-3705 for treating methylmalonic acidemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the long-term safety and clinical effectiveness of mRNA-3705 in patients with isolated methylmalonic acidemia (MMA) who have previously participated in other studies involving this treatment. Participants will undergo a Treatment Period followed by a Follow-up Period lasting up to two years after their last dose. The study will monitor safety and clinical outcomes, with the possibility of transitioning to market access if mRNA-3705 receives approval. The goal is to ensure continuous treatment without interruption for eligible participants.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Completed the assigned dose regimen treatment time period in other clinical studies of mRNA-3705 or is eligible for early transition to this study because they missed more than 3 consecutive doses of study drug due to coronavirus disease 2019 (COVID-19) vaccination during Study mRNA-3705-P101 Part 1.
* Completed the End of treatment (EOT) Visit (or End of Study Visit in the case of unscheduled dosing) in Study mRNA-3705-P101 within 10 days of their first dose of mRNA-3705 in this extension study.

Exclusion Criteria:

* Not expected to receive clinical benefit from continued mRNA-3705 administration, in the opinion of the Investigator.
* Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
* History of liver and/or kidney transplant.

NOTE: Other inclusion and exclusion criteria may apply.

Where it's running

• UCLA Medical Center
  Los Angeles, California, United States Recruiting
• Lucile Packard Children's Hospital at Stanford
  Palo Alto, California, United States Recruiting
• Altman Clinical and Translational Research Institution
  San Diego, California, United States Recruiting
• Stollery Children's Hospital University of Alberta
  Edmonton, Alberta, Canada Recruiting
• Hospital For Sick Children
  Toronto, Ontario, Canada Recruiting
• Hôpital Necker - Enfants Malades APHP
  Paris, France Recruiting

Study contacts

• Study coordinator: Moderna WeCare Team
• Email: WeCareClinicalTrials@modernatx.com
• Phone: 1-866-663-3762

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Observing the effects of Carbaglu® in treating MMA and PA in patients

  This study is testing how safe Carbaglu® is for treating high ammonia levels in people with Methylmalonic Acidemia and Propionic Acidemia, both kids and adults.

  Recruiting at 5 sites Recordati Group · Washington D.C., District of Columbia and 4 other locations · NCT05040178

• Long-term management of organic acidemia with CARBAGLU®

  This study looks at how well carglumic acid helps people with propionic acidemia and methylmalonic acidemia over a long time, while also talking to patients and caregivers about their experiences.

  Recruiting at 31 sites Recordati Group · Bordeaux and 31 other locations · NCT04176523

• Observational study of Methylmalonic Acidemia

  This study looks at how often complications like metabolic stroke and developmental delays happen in people of all ages with Methylmalonic Acidemia to help improve future treatments.

  Recruiting at 2 sites National Institutes of Health Clinical Center (CC) · Washington D.C., District of Columbia and 2 other locations · NCT00078078

• MMA in kids up to 16 with MMUT mutations (MMUT-related isolated MMA) observational

  This observational study tests how outcomes vary in children with isolated MMA caused by MMUT mutations, including those who have had liver or liver+kidney transplants.

  Recruiting Genespire Srl · Philadelphia, Pennsylvania and 7 other locations · NCT07432880

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.