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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-502963-39-00 | Registry Identifier | Clinical Trials Information System (CTIS) |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mRNA-3745 | Drug | Sterile frozen liquid dispersion for injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation | Day 1 up to approximately 3.5 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline through up to Week 32 |
| Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges |
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Inclusion Criteria:
Exclusion Criteria:
Note: Additional inclusion/exclusion criteria may apply, per protocol.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Connecticut Health Center | Farmington | Connecticut | 06030-0001 | United States | ||
| Boston Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34035281 | Background | 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. |
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| Baseline through up to Week 32 |
| Change From Baseline in Time to Hypoglycemia During Fasting Challenges | Baseline through up to Week 32 |
| Change From Baseline in Maximum Effect (Emax) During Fasting Challenges | Baseline through up to Week 32 |
| SAD only: Maximum Observed Concentration (Cmax) of Messenger Ribonucleic Acid (mRNA) and Lipid Nanoparticle (LNP) | 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 | Pre-infusion, during infusion, at EOI and post-infusion on Day 1 up to Week 52 |
| Change From Baseline in Metabolic Biomarkers of GSD1a | Baseline through up to approximately 6.5 years |
| MAD only: Maximum Observed Concentration at Steady State (Cmax,ss) of mRNA and LNP | Pre-infusion, during infusion, at the EOI and post-infusion on Day 1 up to Week 52 |
| Boston |
| Massachusetts |
| 02115 |
| United States |
| Duke University Medical Center | Durham | North Carolina | 27713 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| The University of Texas Health Science Center at Houston | Houston | Texas | 77030-1501 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| University of Utah | Salt Lake City | Utah | 84132-0001 | United States |
| Stollery Children's Hospital University of Alberta | Edmonton | Alberta | T6G 2R7 | Canada |
| The Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| AP-HP - Hôpital Antoine Béclère | Clamart | 92140 | France |
| CHRU Tours - Hopital Clocheville | Tours | 37000 | France |
| Universitair Medisch Centrum Groningen | Groningen | 9713 GZ | Netherlands |
| Instytut Pomnik Centrum Zdrowia Dziecka | Warsaw | 04-730 | Poland |
| Hospital Universitario 12 de Octubre | Madrid | 28026 | Spain |
| Hospital Regional Universitario de Malaga | Málaga | 29011 | Spain |
| ID | Term |
|---|---|
| D006008 | Glycogen Storage Disease |
| D005953 | Glycogen Storage Disease Type I |
| D044882 | Glucose Metabolism Disorders |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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