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

Phase 2Active Not RecruitingNCT05095727

ModernaTX, Inc.15 enrolled

Overview

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.

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

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Glycogen Storage Disease

Interventions

mRNA-3745DRUG

Sterile frozen liquid dispersion for injection

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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.

Locations (16)

University of Connecticut Health Center

Farmington, Connecticut, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

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

Secondary Outcomes

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.

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

Data from ClinicalTrials.gov

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