A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

Moderna

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Glycogen Storage Disease

Treatments: Drug: mRNA-3745

Study type: Interventional

Funder types: Industry

Identifiers

NCT05095727

mRNA-3745-P102

2022-502963-39-00 (Registry Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 45 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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45 participants in 2 patient groups

SAD: mRNA-3745

Experimental group

Description:

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.

Treatment:

Drug: mRNA-3745

MAD: mRNA-3745

Experimental group

Description:

Participants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE.

Treatment:

Drug: mRNA-3745