63 Participants Needed

Long-Term Safety of mRNA-3705 for Methylmalonic Acidemia

Recruiting at 8 trial locations
MC
MW
Overseen ByModerna WeCare Team
Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: ModernaTX, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

What data supports the effectiveness of the drug mRNA-3705 for treating methylmalonic acidemia?

Research on a similar mRNA treatment showed that it significantly reduced harmful substances in the blood and improved survival and weight gain in mice with methylmalonic acidemia, without causing liver damage or inflammation.12345

What safety data exists for mRNA-3705 treatment in humans?

In studies with mice, the mRNA-3705 treatment showed no increase in liver toxicity or inflammation with repeated doses, suggesting it may be safe. However, human safety data is not provided in the available research.12456

How is the drug mRNA-3705 different from other treatments for methylmalonic acidemia?

mRNA-3705 is unique because it uses a synthetic messenger RNA (mRNA) to produce the enzyme methylmalonyl-CoA mutase, which is often missing or defective in people with methylmalonic acidemia. Unlike traditional treatments, it is delivered intravenously (through a vein) using lipid nanoparticles, which helps the body produce the necessary enzyme without the risks associated with gene therapy, such as immune reactions or genetic damage.12457

What is the purpose of this trial?

The primary objective of this study is to evaluate the long-term safety of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in other clinical studies of mRNA-3705.

Eligibility Criteria

This trial is for people with Methylmalonic Acidemia who were in a previous study (mRNA-3705-P101). They must have finished the earlier trial or moved to this one because they missed doses due to COVID-19 vaccination.

Inclusion Criteria

I missed doses in a previous study due to COVID-19 vaccination.
Completed the End of treatment (EOT) Visit in Study mRNA-3705-P101 within 10 days of first dose of mRNA-3705 in the current study.

Exclusion Criteria

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
I have had a liver or kidney transplant.
Not expected to receive clinical benefit from continued mRNA-3705 administration, in the opinion of the Investigator

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive mRNA-3705 at the same dose levels and intervals as in previous studies, unless modified by the Sponsor

Indefinite, until specific conditions are met
Every 2 or 3 weeks, depending on previous study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2 years after the last dose

Open-label extension

Participants may continue treatment long-term if mRNA-3705 receives market approval and reimbursement

Treatment Details

Interventions

  • mRNA-3705
Trial Overview The trial is testing the long-term safety and effectiveness of mRNA-3705, a treatment for those with isolated methylmalonic acidemia caused by MUT deficiency, based on their prior participation in an initial study.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: mRNA-3705Experimental Treatment1 Intervention
Participants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks \[Q2W\], or every 3 weeks \[Q3W\]) last received in the clinical study of mRNA-3705 in which they initially participated, unless the Sponsor recommends modification.

mRNA-3705 is already approved in United States for the following indications:

🇺🇸
Approved in United States as mRNA-3705 for:

    Find a Clinic Near You

    Who Is Running the Clinical Trial?

    ModernaTX, Inc.

    Lead Sponsor

    Trials
    127
    Recruited
    66,790,000+

    Dr. Stephen Hoge

    ModernaTX, Inc.

    Chief Medical Officer

    MD from Harvard Medical School

    Stéphane Bancel profile image

    Stéphane Bancel

    ModernaTX, Inc.

    Chief Executive Officer since 2011

    MBA from Harvard Business School, MSc in Engineering from École Centrale Paris

    Findings from Research

    Patients with methylmalonic acidaemia who are cobalamin responsive have a significantly better long-term outcome, with milder disease and no reported deaths, compared to the non-responsive group.
    The non-responsive patients, especially those with early onset, face a poor prognosis with higher mortality rates and cognitive challenges, indicating a need for alternative treatments for this group.
    Neurological outcome of methylmalonic acidaemia.Nicolaides, P., Leonard, J., Surtees, R.[2019]
    In a study of 12 patients with cblA-type methylmalonic acidemia (MMA), the main symptoms included vomiting, dyspnea, and drowsiness, with 11 patients showing a positive response to vitamin B12 treatment.
    After treatment, significant reductions in harmful metabolites were observed, and 66.7% of patients achieved normal development, indicating that early intervention can lead to better clinical outcomes.
    [Analysis of 12 cases with methylmalonicacidemia cblA type].E, H., Han, L., Ye, J., et al.[2020]
    High-performance liquid chromatography-tandem mass spectrometry (LC-MS/MS) effectively measures methylmalonic acid (MMA), 2-methylcitric acid (MCA), and homocysteine (Hcy) levels in children with methylmalonic aciduria, providing a reliable method for treatment follow-up.
    The study established significant correlations between MMA and MCA levels in dried blood spots and urine, indicating that LC-MS/MS can be a valuable tool for monitoring treatment efficacy, although local reference intervals need to be defined.
    Clinical Application of LC-MS/MS in the Follow-Up for Treatment of Children with Methylmalonic Aciduria.Wang, Y., Sun, Y., Jiang, T.[2020]

    References

    Neurological outcome of methylmalonic acidaemia. [2019]
    [Analysis of 12 cases with methylmalonicacidemia cblA type]. [2020]
    Clinical Application of LC-MS/MS in the Follow-Up for Treatment of Children with Methylmalonic Aciduria. [2020]
    Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia. [2022]
    Treatment of methylmalonic acidemia by liver or combined liver-kidney transplantation. [2019]
    Neurologic outcome following liver transplantation for methylmalonic aciduria. [2023]
    Clinical and Molecular Spectrum of Patients with Methylmalonic Acidemia. [2023]
    Unbiased ResultsWe believe in providing patients with all the options.
    Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
    Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
    Back to top
    Terms of Service·Privacy Policy·Cookies·Security