Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

Status Recruiting

Clinical Trial Summary

This First-in-Human (FIH) Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the optimal dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with acceptable safety and pharmacodynamic (PD) response in a Dose Optimization Group (Part 1) in participants ≥1 year of age, additional participants will be enrolled into the study in a Dose Expansion Group (Part 2) to allow for further characterization of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (<1 year of age).

Clinical Trial Description

During the Dose Optimization Stage, after each dose cohort is fully enrolled (≥1 year of age), , and the dose-limiting toxicity (DLT) observation window of at least 14 days is complete for the final participant in that cohort, the Sponsor will review the totality of available safety data in conjunction with all available PK/PD data. Based on this review, the Sponsor will recommend a revised dose and/or dosing interval. The Sponsor will abide by predefined constraints as to the maximum percentage change in dose and dose interval. A maximum of 9 cohorts will be enrolled in Part 1 (Dose Optimization). Upon establishment of a dose with acceptable safety and PD activity in a Dose Optimization Part (Part 1), additional participants (≥1 year of age) will be enrolled into the study in a Dose Expansion Part (Part 2) to allow for further characterization of the safety, efficacy, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response in infants (<1 year of age). Participants in all the phases will participate in a predosing observational period, followed by a treatment period, and then a follow-up period after withdrawal of treatment. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04159103
Study type	Interventional
Source	ModernaTX, Inc.
Contact	Moderna Clinical Trials Support Center
Phone	1-877-777-7187
Email	clinicaltrials@modernatx.com
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	April 15, 2021
Completion date	January 6, 2027

View Details

See also
Status	Clinical Trial	Phase
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Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Recruiting	`NCT05130437` - A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927	Phase 1/Phase 2
Recruiting	`NCT02890342` - Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Completed	`NCT02426775` - Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia	Phase 3
Terminated	`NCT04836494` - A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia	Phase 1
Completed	`NCT03484767` - "The MaP Study": Mapping the Patient Journey in MMA and PA
Terminated	`NCT04732429` - Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia	Phase 2
Recruiting	`NCT05040178` - An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Recruiting	`NCT05769621` - A Retrospective Study to Characterize Participants With Propionic Acidemia
Completed	`NCT00645879` - Anaplerotic Therapy in Propionic Acidemia	Phase 1
Recruiting	`NCT04176523` - Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Withdrawn	`NCT01341379` - Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate	Phase 2
Terminated	`NCT05438485` - Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
Completed	`NCT03159026` - Review of Charts From Amish/Mennonite Variant PA Patients

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.