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mRNA-3745

Phase 1

Glycogen Storage Disease | Small molecule | Metabolic |Moderna, Inc.|Last Updated: Dec 15, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment15

FDA Designations

No designations recorded

Clinical trial landscape

mRNA-3745 · 1 trial · 1 indication

Phase 1 1
NCT05095727A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)Glycogen Storage Disease
ACTIVE NOT_RECRUITING15 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Glycogen Storage Disease
NCT0509572715
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Study Endpoints

Primary Endpoints

Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation
Day 1 up to approximately 3.5 years

Secondary Endpoints

Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges
Baseline through up to Week 32
Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges
Baseline through up to Week 32
Change From Baseline in Time to Hypoglycemia During Fasting Challenges
Baseline through up to Week 32
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SAD: mRNA-3745EXPERIMENTALParticipants will receive a single intravenous (IV) dose of mRNA-3745 on Day 1 in an inpatient setting. Participants that are/have been enrolled in the study and receive an administration of mRNA-3745 may also enroll in one of the MAD cohorts. The first MAD dose must occur at least 21 days after the SAD dose.
MAD: mRNA-3745EXPERIMENTALParticipants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE.

Interventions

NameTypeDescription
mRNA-3745DRUGSterile frozen liquid dispersion for injection
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Eligibility Criteria

Age Range6 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (G6PC) mutations by genetic testing. * Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening Exclusion Criteria: * Solid organ transplant * Received gene therapy for...

Countries:United StatesCanadaFranceNetherlandsPolandSpain
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Frequently asked questions about mRNA-3745

What is mRNA-3745 used for?

mRNA-3745 is an investigational therapy being developed for the treatment of Glycogen Storage Disease, specifically Glycogen Storage Disease Type 1a (GSD1a). It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who is developing mRNA-3745?

mRNA-3745 is being developed by Moderna, Inc., a biotechnology company publicly traded under the ticker symbol MRNA on the NASDAQ stock exchange. The company is conducting clinical research to evaluate the safety and efficacy of this investigational therapy.

What phase is mRNA-3745 in?

mRNA-3745 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet received regulatory approval and is still undergoing clinical trials to assess its safety and potential effectiveness in patients.

What clinical trials is mRNA-3745 in?

mRNA-3745 is being studied in a Phase 1 clinical trial with the identifier NCT05095727. This trial is titled 'A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)' and is currently active but not recruiting participants.

What is the enrollment and design of the mRNA-3745 clinical trial?

The Phase 1 clinical trial of mRNA-3745 (NCT05095727) is a controlled, open-label study with a planned enrollment of 15 participants. The trial is not randomized or double-blinded, and it includes both adult and pediatric participants aged 6 years and older.

Where is the mRNA-3745 clinical trial being conducted?

The Phase 1 clinical trial of mRNA-3745 is being conducted in multiple countries, including the United States, Canada, France, Netherlands, Poland, and Spain. The study is open to all sexes and does not include healthy volunteers.