GLYCOGEN STORAGE DISEASE TYPE IA
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE IA explained in plain language.
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Gene therapy breakthrough aims to control rare metabolic disease
Disease control Recruiting nowThis study tests a new gene therapy called BEAM-301 in 36 adults with glycogen storage disease type Ia (GSDIa) who have a specific gene change. The goal is to see if a single dose can safely improve blood sugar control and reduce the need for cornstarch supplements. Participants …
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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Can home tests replace lab draws for rare disease patients?
Diagnosis Recruiting nowThis study aims to see if a home lactate meter and a standard glucose meter give accurate readings compared to lab tests in people with glycogen storage disease types Ia, Ib, and XI. Ten participants will have an 8-hour hospital stay with hourly blood draws and finger-stick tests…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Diagnosis
Last updated Jun 27, 2026 13:01 UTC
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GSDIa patients monitored for 10 years after gene therapy
Knowledge-focused Recruiting nowThis observational study follows people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401. The goal is to track their health and safety for at least 10 years after treatment. No new drugs or treatments are given in this study. It will help re…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:00 UTC
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Can a simple Finger-Stick replace lab tests for rare metabolic disease?
Knowledge-focused Recruiting nowThis study looks at whether portable lactate monitors (like those used at home) give accurate results for people with glycogen storage disease type Ia. Ten participants will have their lactate and glucose levels measured by finger-stick and by standard lab tests. The goal is to s…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 UTC