ACTIVE_NOT_RECRUITING

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

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Conditions

Methylmalonic AcidemiaIsolated Methylmalonic AcidemiaIsolated Methylmalonic AciduriaElevated Methylmalonic Acid (MMA)Metabolism, Inborn ErrorsGenetic DiseasesModernamRNAmRNA-3705

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2021-08-06
Study Completion:2028-07-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04899310

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Year
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * (Part 1 only) Participant has a body weight of ≥11.0 kilograms at the screening visit.
  2. * Participant has a diagnosis of isolated MMA due to MUT deficiency confirmed by molecular genetic testing.
  3. * Participant has a blood vitamin B12 level equal to or above the lower limit of normal (based on laboratory reference range) confirmed in the screening period.
  4. * Participant or their legally authorized representative is willing and able to provide informed consent and/or assent as mandated by local regulations and is willing and able to comply with study-related assessments.
  5. * Sexually active participants of childbearing or reproductive potential agree to use a highly effective method of contraception, consistent with local regulations, during the study and for 3 months after the last administration of study drug.
  6. * (Part 2 only) Participants with 2 screening MMA levels ≥400 micromolar.
  7. * (Parts 2 and 3 only) Participant is ≥5 years of age at the time of informed consent/assent.
  1. * Participant has a diagnosis of isolated MMA cofactor adenosyl-cobalamin (cb1A, cb1B, or cb1D) enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combined MMA with homocystinuria.
  2. * Participant has previously received gene therapy for the treatment of MMA.
  3. * Participant has a history of organ transplantation or planned organ transplantation during the period of study participation.
  4. * Participant has an active, unstable, or clinically significant medical condition not related to MMA or history of noncompliance that, in the investigator's opinion, could potentiate the risk while participating in this study, interfere with the interpretation of study results, or limit the participant's participation in the study. This may include, but is not limited to, history of relevant food or drug allergies; history of cardiovascular, central nervous, gastrointestinal, or infectious disease; history of clinically significant pathology; and/or history of cancer.
  5. * (Part 2 only) Participant has the partial MUT deficiency disease phenotype, as assessed by genotyping, clinical phenotype/presentation, or vitamin B12-responsive MMA.

Contacts and Locations

Study Locations (Sites)

UCLA Medical Center
Los Angeles, California, 90095
United States
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, 94304
United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: ModernaTX, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-08-06
Study Completion Date2028-07-01

Study Record Updates

Study Start Date2021-08-06
Study Completion Date2028-07-01

Terms related to this study

Keywords Provided by Researchers

  • Isolated Methylmalonic Acidemia
  • Isolated Methylmalonic Aciduria
  • Elevated Methylmalonic Acid (MMA)
  • Metabolism, Inborn Errors
  • Genetic Diseases
  • Moderna
  • mRNA
  • mRNA-3705

Additional Relevant MeSH Terms

  • Methylmalonic Acidemia