Glycogen Storage Disease Type IA Clinical Trials

21 clinical trials for Glycogen Storage Disease Type IA across international registries, including 5 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program — RECRUITING
• Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease — RECRUITING
• Accuracy of Lactate Meter in GSDIa — RECRUITING
• A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) — RECRUITING · PHASE1
• AN OPEN-LABEL DOSE-ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MRNA-3745 IN PARTICIPANTS WITH GLYCOGEN STORAGE DISEASE TYPE 1A (GSD1A) — RECRUITING
• Clinical trial for the research into changes in renal function of adolescent and adult patients with Glycogen storage disease type Ia. — ACTIVE_NOT_RECRUITING · PHASE2
• A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) — COMPLETED · PHASE3
• Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia — COMPLETED
• GSDIa Disease Monitoring Program — TERMINATED · PHASE3
• A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adenoassociated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia. — OTHER · PHASE3
• A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa) — OTHER · PHASE2
• Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa — COMPLETED
• Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease — TERMINATED
• A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia — OTHER
• Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia — COMPLETED · PHASE1
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF ADENOASSOCIATED VIRUS SEROTYPE 8-MEDIATED GENE TRANSFER OF GLUCOSE-6- PHOSPHATASE IN PATIENTS WITH GLYCOGEN STORAGE DISEASE TYPE IA — COMPLETED · PHASE3
• A PHASE 1/2 FIRST-IN-HUMAN, 2-PART STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS OF SINGLE ASCENDING DOSES (PART 1: OPEN-LABEL) AND REPEAT DOSES (PART 2: RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED) OF UX053 IN PATIENTS WITH GSD III — COMPLETED · PHASE1
• Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease — COMPLETED
• A LONG-TERM FOLLOW-UP STUDY TO EVALUATE THE SAFETY AND EFFICACY OF ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE 8 (AAV8)-MEDIATED GENE TRANSFER OF GLUCOSE-6-PHOSPHATASE (G6PASE) IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA (GSDIA) — COMPLETED
• A PHASE 1/2, OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE 8 (AAV8)-MEDIATED GENE TRANSFER OF GLUCOSE-6-PHOSPHATASE (G6PASE) IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA (GSDIA) — COMPLETED · PHASE1
• Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch — COMPLETED

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