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.

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2022-501997-20-00

Acronym

mRNA-3705-P101-EXT

Enrollment

Registered

2023-09-20

Start date

2023-12-01

Completion date

Unknown

Last updated

2025-12-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency

Brief summary

Incidence and severity of TEAEs, including study drug-related and not related TEAEs, AESIs, SAEs, and TEAEs leading to treatment discontinuation

Detailed description

Percentage change in plasma methylmalonic acid and 2-MC levels (primary biomarkers) from baseline, Predose and postdose hMUT mRNA and SM-86 levels, Changes in MDE frequency pretreatment and post-treatment: Annualized MDE rate, Frequency and duration of HCRU visits, Annualized frequency of MMA- related hospitalizations, Annualized frequency of MMA- related healthcare visits, Presence and titers of anti-PEG and anti-hMUT antibodies, Changes in PedsQL™ measurements, Changes in IGA-I, IGA-S, and EQ-5D-5L/Y

Interventions

DRUGPARACETAMOL

DRUGHYDROXYZINE HYDROCHLORIDE

DRUGFAMOTIDINE

DRUGCETIRIZINE DIHYDROCHLORIDE

DRUGIBUPROFEN

DRUGDEXAMETHASONE PHOSPHATE

DRUGSODIUM CHLORIDE SOLUTION 0.9%

DRUGDIPHENHYDRAMINE HYDROCHLORIDE

DRUGmRNA-3705

DRUGDEXAMETHASONE

DRUGFEXOFENADINE HYDROCHLORIDE

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Incidence and severity of TEAEs, including study drug-related and not related TEAEs, AESIs, SAEs, and TEAEs leading to treatment discontinuation	—

Secondary

Measure	Time frame
Percentage change in plasma methylmalonic acid and 2-MC levels (primary biomarkers) from baseline, Predose and postdose hMUT mRNA and SM-86 levels, Changes in MDE frequency pretreatment and post-treatment: Annualized MDE rate, Frequency and duration of HCRU visits, Annualized frequency of MMA- related hospitalizations, Annualized frequency of MMA- related healthcare visits, Presence and titers of anti-PEG and anti-hMUT antibodies, Changes in PedsQL™ measurements, Changes in IGA-I, IGA-S, and EQ-5D-5L/Y	—

Measure

Time frame

—

Countries

France, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026