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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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 0 (predose) to 336 hours postdose

Awards & highlights

Study Summary

This trial is studying a potential new treatment for people with a specific type of mutation that causes elevated levels of methylmalonic acid. The goal is to assess safety, how the body processes the treatment, and how well it works.

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.Check my eligibility

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).See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 0 (predose) to 336 hours postdose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~0 (predose) to 336 hours postdose

This trial's timeline: 3 weeks for screening, Varies for treatment, and 0 (predose) to 336 hours postdose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part 1: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Study Drug-related TEAES, Serious Adverse Events (SAEs), and TEAEs Leading to Treatment Discontinuation

Part 2: Annualized Frequency of Metabolic Decompensation Events (MDEs)

Secondary outcome measures

Area Under the Concentration-Time Curve (AUC) of hMUT mRNA-3705

Area Under the Effect Curve (AUEC) for Plasma Methylmalonic Acid Measurement after Single and Repeated Administrations of mRNA-3705

Change in Blood 2-Methylcitric Acid (2-MC ) Levels

+5 more

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 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

ModernaTX, Inc.Lead Sponsor

108 Previous Clinical Trials

61,379,254 Total Patients Enrolled

Media Library

mRNA-3705 (Nucleic Acid-Based Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04899310 — Phase 1 & 2

Methylmalonic Acidemia Research Study Groups: mRNA-3705

Methylmalonic Acidemia Clinical Trial 2023: mRNA-3705 Highlights & Side Effects. Trial Name: NCT04899310 — Phase 1 & 2

mRNA-3705 (Nucleic Acid-Based Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04899310 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the highest capacity of participants involved in this clinical trial?

"Indeed, the information accessible on clinicaltrials.gov confirms that this research is actively seeking candidates to join. It was initially announced on August 6th 2021 and has recently been updated on June 16th 2022. The study requires 33 participants from two locations in order to continue."

Answered by AI

What other explorations have been made regarding the efficacy of mRNA-3705?

"Currently, mRNA-3705 is the subject of 1 active clinical trial. This experiment has yet to reach Phase 3, though there are 4 sites across Toronto conducting research into this medicine's efficacy."

Answered by AI

Is this experiment currently taking on new participants?

"Indeed, the clinicaltrials.gov confirms that this medical study is actively recruiting and has been since August 6th 2021. 33 potential participants are sought between 2 different sites, and the research was last adjusted on June 16th 2022."

Answered by AI

Is this a groundbreaking research endeavor?

"Since 2021, mRNA-3705 has been under scientific evaluation. The initial investigation was conducted and funded by ModernaTX, Inc., with 33 participants in the trial cohort. Subsequently, the drug successfully completed Phases 1 & 2 of approval process. Currently there is one ongoing study sponsored by ModernaTX, Inc.."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2021. "A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04899310.