Long-Term Safety and Clinical Activity of mRNA-3705 in Methylmalonic acidemia (MMA) Patients

AddtoAny
Share:

WATCH THIS PAGE

Subscribe to be notified of changes or updates to this page.

4 + 8 =
Solve this simple math problem and enter the result. E.g. for 1+3, enter 4.

This extension study will determine the long-term safety and effectiveness of mRNA-3705, a treatment for Methylmalonic acidemia (MMA), in patients who previously enrolled in and completed a prior mRNA-3705 study.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at maddenr [at] chop.edu or 215-590-3376.

ClinicalTrials.gov Identifier

NCT05295433

View full trial information on ClinicalTrials.gov

Eligibility & Criteria

IRB #:
22-020354
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
Study Phase:
Phase I
Phase II
Eligible Age Range:
1 - 99 Years
Gender:
All
Study Categories:

Visit Criteria

This study will include the following procedures:

  • Review of Medical Records and contraception/a pregnancy test (if applicable)
  • Interviews
  • Questionnaires
  • Physical exams and assessment of vital signs
  • ECGs, ECHOs
  • Nutritional assessments
  • Blood and Urine collection in excess of Standard of Care
  • Neurocognitive and Motor assessments
  • Administration of study drug (mRNA-3705) via an intravenous infusion, pre-dose H2 blocker (such as famotidine), H1 blocker (cetirizine), and acetaminophen or ibuprofen
Leader
Can Ficicioglu

Can Ficicioglu, MD, PhD