A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
NCT ID: NCT04899310
Last Updated: 2025-12-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
74 participants
INTERVENTIONAL
2021-08-06
2028-07-01
Brief Summary
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Detailed Description
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Parts 1 and 3 are designed to characterize the safety, tolerability, and pharmacological activity of mRNA-3705 administered via intravenous infusion to participants with isolated MMA due to MUT deficiency. Part 2 will evaluate the efficacy of mRNA-3705 as assessed by the change in plasma methylmalonic acid levels.
Participants who complete the treatment period in any part of the study, including the end of treatment (EOT) visit, will be offered participation in the mRNA-extension study (mRNA-3705-P101-EXT; NCT05295433) or may transition to the follow-up period of the study. All participants, including those randomized to placebo in Part 2, will receive mRNA-3705 in the extension study.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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mRNA-3705
Participants in Part 1 will receive a weight-based dose of mRNA-3705, administered IV, once every 2 weeks or once every 3 weeks for up to 10 doses over approximately 40 weeks. Participants in Part 2 and Part 3 will receive mRNA-3705 at the selected dose level and frequency for 3 months.
mRNA-3705
Sterile liquid for infusion
Placebo
Participants only in Part 2 will receive placebo at the selected frequency for 3 months.
Placebo
Sterile liquid for infusion
Interventions
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mRNA-3705
Sterile liquid for infusion
Placebo
Sterile liquid for infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participant has a diagnosis of isolated MMA due to MUT deficiency confirmed by molecular genetic testing.
* 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.
* 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.
* 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.
* (Part 2 only) Participants with 2 screening MMA levels ≥400 micromolar.
* (Parts 2 and 3 only) Participant is ≥5 years of age at the time of informed consent/assent.
Exclusion Criteria
* Participant has previously received gene therapy for the treatment of MMA.
* Participant has a history of organ transplantation or planned organ transplantation during the period of study participation.
* 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.
* (Part 2 only) Participant has the partial MUT deficiency disease phenotype, as assessed by genotyping, clinical phenotype/presentation, or vitamin B12-responsive MMA.
1 Year
ALL
No
Sponsors
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ModernaTX, Inc.
INDUSTRY
Responsible Party
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Locations
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UCLA Medical Center
Los Angeles, California, United States
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital at Westmead
Westmead, New South Wales, Australia
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada
Hospital For Sick Children
Toronto, Ontario, Canada
Hôpital Necker - Enfants Malades
Paris, , France
Erasmus MC
Rotterdam, , Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, , Netherlands
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Cruces
Barakaldo, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
CHUS - H. Clinico U. de Santiago
Santiago de Compostela, , Spain
Hospital Universitario Virgen del Rocio - PPDS
Seville, , Spain
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Campos Monserrath
Role: primary
Thu Quan
Role: primary
Rebecca Madden
Role: primary
Cheri Copithorne
Role: primary
Dhillon Preet
Role: primary
Michaela Semeraro
Role: primary
Role: primary
Role: primary
Isabel Maria Miquel Aymar
Role: primary
Martin Rodriguez Ohiane
Role: primary
Ianire Gallego Amaro
Role: primary
Laura Lopez Valverde
Role: primary
Ana Muñoz
Role: primary
Laura Crowther
Role: primary
Other Identifiers
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2022-502492-32-00
Identifier Type: OTHER
Identifier Source: secondary_id
mRNA-3705-P101
Identifier Type: -
Identifier Source: org_study_id