What side effects are associated with this type of intervention?

Common treatments for Methylmalonic Acidemia (MMA) include dietary management, vitamin B12 (hydroxocobalamin) injections, and in some cases, liver or combined liver-kidney transplantation. For mRNA-based therapies like mRNA-3705, which aim to produce functional methylmalonyl-CoA mutase enzyme, potential side effects may include immune reactions to the mRNA or the delivery system, injection site reactions, and flu-like symptoms. As with other gene therapies, there is also a risk of unintended immune responses or off-target effects. It is essential to monitor patients closely for any adverse reactions during treatment.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for mRNA-based therapies for Methylmalonic Acidemia (MMA) in the provided context. Traditional treatments for MMA have included vitamin B12 supplementation and dietary protein restriction, which have shown effectiveness in managing the condition. The mRNA-3705 trial represents a novel approach, focusing on mRNA-based therapy to produce functional methylmalonyl-CoA mutase enzyme, which is a new direction in the treatment of MMA.

What other types of research exist?

Promising alternatives to mRNA-3705 for Methylmalonic Acidemia patients may include gene therapy approaches that aim to correct the underlying genetic defect, enzyme replacement therapies to provide functional methylmalonyl-CoA mutase enzyme, and small molecule therapies that can enhance the residual activity of the defective enzyme or reduce the toxic metabolites. Consulting with a healthcare provider for the latest clinical trials and approved treatments is recommended.

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Nucleic Acid-Based Therapy

mRNA-3705 for Methylmalonic Acidemia

Phase 1 & 2

Recruiting

Research Sponsored by ModernaTX, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant has a body weight of ≥11.0 kilograms (kg) at the Screening Visit.

Participant has a diagnosis of isolated MMA due to MUT deficiency confirmed by molecular genetic testing.

Must not have

Participant has previously received gene therapy for the treatment of MMA.

Participant has a diagnosis of isolated MMA cb1A, cb1B, or cb1D enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combined MMA with homocystinuria.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing mRNA-3705, a treatment that helps the body produce a missing enzyme, in patients with high levels of MMA due to a genetic condition. The goal is to see if it is safe and effective in reducing harmful substance buildup.

Who is the study for?

This trial is for individuals with isolated Methylmalonic Acidemia (MMA) due to MUT deficiency, confirmed by genetic testing. Participants must have normal or supplemented vitamin B12 levels, weigh at least 11 kg, and agree to use effective contraception. Excluded are those with organ transplants, other MMA types, prior gene therapy for MMA, significant unrelated medical conditions, or certain infections.

What is being tested?

The study tests mRNA-3705 in patients with elevated methylmalonic acid from MUT deficiency. It aims to evaluate the safety of the drug as well as how it's processed in the body (pharmacokinetics) and its effect on the disease (pharmacodynamics).

What are the potential side effects?

While specific side effects of mRNA-3705 aren't detailed here, common reactions may include injection site discomfort, potential allergic responses, flu-like symptoms such as fever and chills, fatigue and possible unknown risks given it's a new treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I weigh at least 11 kilograms.

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I have been diagnosed with MMA due to MUT deficiency through genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had gene therapy for MMA before.

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I have a specific type of metabolic disorder related to MMA or combined MMA with homocystinuria.

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I have had an organ transplant or will have one during the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: mRNA-3705Experimental Treatment1 Intervention

Participants in Part 1 will receive a weight based dose of mRNA-3705, administered intravenously (IV), once every 2 weeks (Q2W) or once every 3 weeks (Q3W) for up to 10 doses over approximately 40 weeks. Participants in Part 2 will receive mRNA 3705 at the selected dose level and frequency for up to 12 months.

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Methylmalonic Acidemia (MMA) is commonly treated with dietary management, vitamin B12 supplementation, and in some cases, liver or kidney transplantation. These treatments aim to reduce the accumulation of methylmalonic acid and manage symptoms. mRNA-based therapies, such as mRNA-3705, work by delivering synthetic mRNA to cells, instructing them to produce functional methylmalonyl-CoA mutase enzyme, which is deficient in MMA patients. This approach directly addresses the underlying enzyme deficiency, potentially reducing methylmalonic acid levels more effectively and improving metabolic stability. This is crucial for MMA patients as it can lead to better management of the disease and improved quality of life.