- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05095727
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
February 16, 2024 updated by: ModernaTX, Inc.
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.
Study Overview
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 Type
Interventional
Enrollment (Estimated)
45
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Moderna Clinical Trials Support Center
- Phone Number: 1-877-777-7187
- Email: clinicaltrials@modernatx.com
Study Locations
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Alberta
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Edmonton, Alberta, Canada, T6G 2R7
- Recruiting
- Stollery Children's Hospital University of Alberta
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Contact:
- Phone Number: (780) 248-5611
- Email: dory.sample@albertahealthservices.ca
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Connecticut
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Farmington, Connecticut, United States, 06030-0001
- Recruiting
- University of Connecticut Health Center
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Contact:
- Phone Number: 860-679-6584
- Email: shking@uchc.edu
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Recruiting
- Boston Children's Hospital
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Contact:
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North Carolina
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Durham, North Carolina, United States, 27713
- Recruiting
- Duke University Medical Center
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Contact:
- Phone Number: 919-681-4026
- Email: gretchen.nichting@duke.edu
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Ohio
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Cincinnati, Ohio, United States, 45229
- Recruiting
- Cincinnati Children's Hospital Medical Center
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Contact:
- Phone Number: 513-636-4507
- Email: laurie.bailey@cchmc.org
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Texas
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Houston, Texas, United States, 77030
- Recruiting
- Baylor College of Medicine
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Houston, Texas, United States, 77030-1501
- Recruiting
- The University of Texas Health Science Center at Houston
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Contact:
- Phone Number: 713-500-7098
- Email: heather.saavedra@uth.tmc.edu
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Utah
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Salt Lake City, Utah, United States, 84132-0001
- Recruiting
- University of Utah
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Documented GSD1a with confirmation by genetic testing
- Documented history of a symptomatic hypoglycemic event with blood glucose <60 mg/dL (<3.3 mmol/L)
- Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening
Exclusion Criteria:
- 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
- Requirement for continuous feeds via gastrostomy or nasogastric tubes
Note: Additional inclusion/exclusion criteria may apply, per protocol.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: SAD: mRNA-3745
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.
|
Sterile frozen liquid dispersion for injection
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Experimental: MAD: mRNA-3745
Participants will receive multiple IV doses of mRNA-3745 in an inpatient setting.
Participants will have the option to continue treatment in the OLE.
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Sterile frozen liquid dispersion for injection
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
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Day 1 up to approximately 3.5 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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Baseline through up to Week 32
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Change From Baseline in Time to Hypoglycemia During Fasting Challenges
Time Frame: Baseline through up to Week 32
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Baseline through up to Week 32
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Change From Baseline in Maximum Effect (Emax) During Fasting Challenges
Time Frame: Baseline through up to Week 32
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Baseline through up to Week 32
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Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges
Time Frame: Baseline through up to Week 32
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Hypoglycemia is defined as blood glucose <60 milligrams (mg)/deciliter (dL) (3.3 millimoles [mmol]/liter [L]) and/or symptoms of hypoglycemia.
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Baseline through up to Week 32
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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
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Pre-infusion, during infusion, at the end of infusion (EOI) and post-infusion on Day 1 up to Week 52
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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
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Pre-infusion, during infusion, at EOI and post-infusion on Day 1 up to Week 52
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Change From Baseline in Metabolic Biomarkers of GSD1a
Time Frame: Baseline through up to approximately 6.5 years
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Baseline through up to approximately 6.5 years
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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
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Pre-infusion, during infusion, at the EOI and post-infusion on Day 1 up to Week 52
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 1, 2022
Primary Completion (Estimated)
January 7, 2026
Study Completion (Estimated)
December 26, 2028
Study Registration Dates
First Submitted
October 4, 2021
First Submitted That Met QC Criteria
October 14, 2021
First Posted (Actual)
October 27, 2021
Study Record Updates
Last Update Posted (Actual)
February 20, 2024
Last Update Submitted That Met QC Criteria
February 16, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- mRNA-3745-P102
- 2022-502963-39-00 (Registry Identifier: Clinical Trials Information System (CTIS))
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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