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Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

NCT ID: NCT03810690

Last Updated: 2020-11-13

Study Results

Results pending

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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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1/PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2019-05-28

Study Completion Date

2020-08-18

Brief Summary

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This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3704 in patients with methylmalonic acidemia/aciduria (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency between 1 to 18 years of age with elevated plasma methylmalonic acid. The study is designed to characterize baseline biomarker levels followed by assessment of safety, pharmacokinetics, and pharmacodynamics of different doses of mRNA-3704 in patients affected by MMA as part of the Dose Escalation phase.

During the Dose Escalation phase, three dose levels of mRNA-3704 are planned to be investigated in this study among patients with MMA due to MUT deficiency: low dose, mid dose, and high dose. An additional cohort to evaluate a fourth dose level may be considered jointly by the independent SMC and the Sponsor.

Upon establishment of a dose with acceptable safety and pharmacodynamic activity, additional patients will be enrolled in a Dose Expansion phase to allow for further characterization of the safety and pharmacodynamics of mRNA-3704.

Patients in both phases of study will participate in a pre-dosing observational period, followed by a treatment period, and then a follow-up period after withdrawal of treatment.

Detailed Description

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Conditions

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Methylmalonic Acidemia (MMA) Metabolism, Inborn Errors

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose Escalation Phase: Dose Level 1

mRNA-3704

Group Type EXPERIMENTAL

mRNA-3704

Intervention Type BIOLOGICAL

mRNA-3704 dispersion for intravenous (IV) infusion

Dose Escalation Phase: Dose Level 2

mRNA-3704

Group Type EXPERIMENTAL

mRNA-3704

Intervention Type BIOLOGICAL

mRNA-3704 dispersion for intravenous (IV) infusion

Dose Escalation Phase: Dose Level 3

mRNA-3704

Group Type EXPERIMENTAL

mRNA-3704

Intervention Type BIOLOGICAL

mRNA-3704 dispersion for intravenous (IV) infusion

Dose Escalation Phase: Dose Level 4 (optional)

mRNA-3704

Group Type EXPERIMENTAL

mRNA-3704

Intervention Type BIOLOGICAL

mRNA-3704 dispersion for intravenous (IV) infusion

Dose Expansion Phase: mRNA-3704

Group Type EXPERIMENTAL

mRNA-3704

Intervention Type BIOLOGICAL

mRNA-3704 dispersion for intravenous (IV) infusion

Interventions

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mRNA-3704

mRNA-3704 dispersion for intravenous (IV) infusion

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

Patients are eligible to be included in the study only if all of the following criteria apply:

* Confirmed diagnosis of isolated MMA due to MUT deficiency based on the following criteria:

* Elevated plasma methylmalonic acid concentrations (≥ 100 µmol/L)
* Presence of normal serum/plasma Vitamin B12 and plasma homocysteine levels
* Confirmed diagnosis by molecular genetic testing
* Patient must be ≥ 1 year of age at the time of consent/assent (Inclusion of the first three patients will be restricted to individuals age ≥ 8 years)

Exclusion Criteria

Patients are excluded from the study if any of the following criteria apply:

* Diagnosis of isolated MMA cblA, cblB, or cblD enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combined MMA with homocystinuria
* History of organ transplantation
* Previously received gene therapy for the treatment of MMA.
* Estimated glomerular filtration rate (GFR) \< 30 mL/min/1.73 m2; or patients who receive chronic dialysis
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ModernaTX, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Countries

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United States

Other Identifiers

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mRNA-3704-P101

Identifier Type: -

Identifier Source: org_study_id