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

Part of paid clinical trials in Los Angeles, California.

Sponsor
ModernaTX, Inc.
Study ID
NCT04159103
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Propionic Acidemia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • mRNA-3927 — BIOLOGICAL
    mRNA-3927 dispersion for IV infusion

Study Details

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination 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).

Key Dates

Start date
Apr 15, 2021
Status verified
Jan 2026
Primary completion
Aug 31, 2027
Completion
Aug 31, 2027

Study Design

Enrollment
77 participants (estimated)
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1 (Dose Optimization), Part 2 (Pivotal Study), and Part 3 (Infants)
    Part 1 (Dose Optimization): Participants (≥1 year of age) will receive single dose of mRNA-3927 by intravenous (IV) infusion every 2 weeks (Q2W) or every 3 weeks (Q3W) for up to 10 doses. Part 2 (Pivotal Study): Participants (≥1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months. Part 3: Participants (\<1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months.

Primary Outcome Measure

Part 1: Number of Participants with Treatment-emergent Adverse Event (TEAE), Serious Adverse Events (SAE) and TEAEs Leading to Discontinuation [ Time Frame: Day 1 (initial mRNA-3927 dose) up to Week 150 (End of Study) ]

Central Contacts

Locations (15)

FacilityCityStateZIPSite coordinators
Ronald Reagan UCLA Medical CenterLos AngelesCalifornia90095
Rosemary Silva-Garcia
[email protected]
UCSD Altman Clinical and Transalational Research Institute BuildingLos AngelesCalifornia90027-
Lucile Packard Children's Hospital StanfordStanfordCalifornia94304
[email protected]
650-497-0454
Nicklaus Children's HospitalMiamiFlorida33155-
University of South Florida - 12901 Bruce B DownsTampaFlorida33606-3603-
Ann and Robert H Lurie Childrens Hospital of ChicagoChicagoIllinois60611-
Johns Hopkins Hospital, Adult Outpatient Clinical Research UnitBaltimoreMaryland21287-
Boston Children's HospitalBostonMassachusetts02115-
University of Michigan HospitalsAnn ArborMichigan48109-
Icahn School of Medicine at Mount Sinai - Clinical Research UnitNew YorkNew York10029-
Duke University Medical System (Duke Health)DurhamNorth Carolina27710
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
University Hospitals Cleveland Medical Center - 11100 Euclid AveClevelandOhio44106-2624-
Children's Hospital of Philadelphia (CHOP)PhiladelphiaPennsylvania19104
Rebecca Madden
[email protected]
Texas Children's HospitalHoustonTexas77030

Find similar trials in Los Angeles, CA

By research site
Ronald Reagan UCLA Medical Center· Los Angeles, CAUCSD Altman Clinical and Transalational Research Institute Building· Los Angeles, CALucile Packard Children's Hospital Stanford· Stanford, CANicklaus Children's Hospital· Miami, FLUniversity of South Florida - 12901 Bruce B Downs· Tampa, FLAnn and Robert H Lurie Childrens Hospital of Chicago· Chicago, IL

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