Glycogen Storage DiseasePhase 1/2NCT05095727
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
A Phase 1/2 study testing mRNA-3745 in people with Glycogen Storage Disease, run by ModernaTX, Inc. at 16 sites in 6 countries.
Trial summary
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Glycogen Storage Disease
- What is being tested?
- mRNA-3745
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 16 sitesin 6 countries
- Study dates
- 1 Jun 2022to 30 Nov 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Solid organ transplant
- Received gene therapy for GSD1a
- Presence of liver adenoma >5 centimeters (cm) in size
- Diagnosis of type 1 or type 2 diabetes mellitus
- Presence of liver adenoma with growth of >2 cm or >5 newly diagnosed liver adenomas, in the previous 2 years
- Note: Additional inclusion/exclusion criteria may apply, per protocol.
Trial locations and contacts
16 sites in 6 countries.
16 sites in 6 countries
United States8 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Duke University Medical Center
Durham, North Carolina, 27713
University of Connecticut Health Center
Farmington, Connecticut, 06030-0001
The University of Texas Health Science Center at Houston
Houston, Texas, 77030-1501
Baylor College of Medicine
Houston, Texas, 77030
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Utah
Salt Lake City, Utah, 84132-0001
Canada2 sites
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
France2 sites
AP-HP - Hôpital Antoine Béclère
Clamart, 92140
CHRU Tours - Hopital Clocheville
Tours, 37000
Spain2 sites
Hospital Universitario 12 de Octubre
Madrid, 28026
Hospital Regional Universitario de Malaga
Málaga, 29011
Netherlands1 site
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ
About this study
Official title: A Phase 1/2, Adaptive, Open-label, Single Ascending Dose to Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a), Followed by an Open-label Extension
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.
Read the detailed description
The study includes a single ascending dose (SAD) stage and a multiple ascending dose (MAD) stage. Participants enrolled in the MAD stage have the option to continue treatment in an open-label extension (OLE) period that will assess long-term safety and clinical activity of mRNA-3745.
- Conditions
- Glycogen Storage Disease
- Being tested
- mRNA-3745
- Keywords
- Glycogen storage disease type 1a, GSD1a, Von Gierke disease, Glucose metabolism disorder, Genetic disorder, Autosomal recessive disorder, messenger RNA, mRNA, Pediatric
- Medical subject headings
- Glycogen Storage Disease, Glycogen Storage Disease Type I, Glucose Metabolism Disorders, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mRNA-3745Drug
Sterile frozen liquid dispersion for injection
| Study group | Role | What they receive |
|---|---|---|
| SAD: mRNA-3745 | Experimental | Participants 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. Receives: Drug: mRNA-3745 |
| MAD: mRNA-3745 | Experimental | Participants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE. Receives: Drug: mRNA-3745 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation
Measured: Day 1 up to approximately 3.5 years
Secondary outcomes (8)
Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges
Hypoglycemia is defined as blood glucose <60 milligrams (mg)/deciliter (dL) (3.3 millimoles [mmol]/liter [L]) and/or symptoms of hypoglycemia.
Measured: Baseline through up to Week 32
Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges
Measured: Baseline through up to Week 32
Change From Baseline in Time to Hypoglycemia During Fasting Challenges
Measured: Baseline through up to Week 32
Change From Baseline in Maximum Effect (Emax) During Fasting Challenges
Measured: Baseline through up to Week 32
SAD only: Maximum Observed Concentration (Cmax) of Messenger Ribonucleic Acid (mRNA) and Lipid Nanoparticle (LNP)
Measured: Pre-infusion, during infusion, at the end of infusion (EOI) and post-infusion on Day 1 up to Week 52
SAD only: Area Under the Concentration-Time Curve From Time 0 to the Time of the Last Measurable Concentration (AUC0-t) of mRNA and LNP
Measured: Pre-infusion, during infusion, at EOI and post-infusion on Day 1 up to Week 52
Change From Baseline in Metabolic Biomarkers of GSD1a
Measured: Baseline through up to approximately 6.5 years
MAD only: Maximum Observed Concentration at Steady State (Cmax,ss) of mRNA and LNP
Measured: Pre-infusion, during infusion, at the EOI and post-infusion on Day 1 up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2021
- Study start1 Jun 2022
- Primary completion30 Nov 2026 (estimated)
- Study completion30 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Glycogen Storage DiseaseStudies of the same condition, open and under way.
- More trials run by ModernaTX, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- ModernaTX, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05095727
- Sponsor's protocol ID
- mRNA-3745-P102
- Other IDs
- 2022-502963-39-00
- Status last verified
- Dec 2025
- First submitted
- 4 Oct 2021
- Last updated
- 15 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH. mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2.
PubMed 34035281
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Adaptive, Open-label, Single Ascending Dose to Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a), Followed by an Open-label Extension
- ClinicalTrials.gov ID
- NCT05095727
- Acronym / sponsor ID
- mRNA-3745-P102
- Conditions
- Glycogen Storage Disease
- Keywords
- Glycogen storage disease type 1a, GSD1a, Von Gierke disease, Glucose metabolism disorder, Genetic disorder, Autosomal recessive disorder, messenger RNA, mRNA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 15 participants (actual)
- Lead sponsor
- ModernaTX, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Jun 2022 (actual)
- Primary completion
- 30 Nov 2026 (estimated)
- Study completion
- 30 Nov 2026 (estimated)
- First posted
- 27 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.
Detailed description
The study includes a single ascending dose (SAD) stage and a multiple ascending dose (MAD) stage. Participants enrolled in the MAD stage have the option to continue treatment in an open-label extension (OLE) period that will assess long-term safety and clinical activity of mRNA-3745.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SAD: mRNA-3745 | Experimental | Drug: mRNA-3745Participants 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-3745 | Experimental | Drug: mRNA-3745Participants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (6 listed)
- Solid organ transplant
- Received gene therapy for GSD1a
- Presence of liver adenoma >5 centimeters (cm) in size
- Diagnosis of type 1 or type 2 diabetes mellitus
- Presence of liver adenoma with growth of >2 cm or >5 newly diagnosed liver adenomas, in the previous 2 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mRNA-3745Sterile frozen liquid dispersion for injection | Drug | MAD: mRNA-3745, SAD: mRNA-3745 |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation
Time frame: Day 1 up to approximately 3.5 years
8 secondary outcomes
Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges
Time frame: Baseline through up to Week 32
Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges
Time frame: Baseline through up to Week 32
Change From Baseline in Time to Hypoglycemia During Fasting Challenges
Time frame: Baseline through up to Week 32
Change From Baseline in Maximum Effect (Emax) During Fasting Challenges
Time frame: Baseline through up to Week 32
SAD only: Maximum Observed Concentration (Cmax) of Messenger Ribonucleic Acid (mRNA) and Lipid Nanoparticle (LNP)
Time frame: Pre-infusion, during infusion, at the end of infusion (EOI) and post-infusion on Day 1 up to Week 52
SAD only: Area Under the Concentration-Time Curve From Time 0 to the Time of the Last Measurable Concentration (AUC0-t) of mRNA and LNP
Time frame: Pre-infusion, during infusion, at EOI and post-infusion on Day 1 up to Week 52
Change From Baseline in Metabolic Biomarkers of GSD1a
Time frame: Baseline through up to approximately 6.5 years
MAD only: Maximum Observed Concentration at Steady State (Cmax,ss) of mRNA and LNP
Time frame: Pre-infusion, during infusion, at the EOI and post-infusion on Day 1 up to Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Dec 2025.
- Background: PubMed 34035281Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH. mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
16 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing mRNA-3745 in people with Glycogen Storage Disease, run by ModernaTX, Inc. at 16 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 15 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada, France, Spain, Netherlands and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation (time frame: Day 1 up to approximately 3.5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05095727, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Dec 2025.