A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Purpose
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.
Condition
- Glycogen Storage Disease
Eligibility
- Eligible Ages
- Over 6 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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 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.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
|
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. |
|
Recruiting Locations
The University of Texas Health Science Center at Houston
Houston, Texas 77030-1501
Houston, Texas 77030-1501
More Details
- Status
- Recruiting
- Sponsor
- ModernaTX, Inc.
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.