NCT06892288
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
레지스트리 요약
- 진행 상태
- 진행중(모집 종료) (ACTIVE_NOT_RECRUITING)
- 질환/조건
- Phosphomannomutase 2 Deficiency, PMM2-CDG
- 시험 유형
- 중재 연구 (INTERVENTIONAL)
- 단계
- Phase 2, Phase 3
- 목적
- 치료
- 중재 유형
- 약물
- 개입·치료
- GLM101 (Part A, Double-blind), Placebo (Part A, Double-blind), GLM101 (Part B, Open-label)
- Lead Sponsor
- Glycomine, Inc. (산업계)
캐시 시각: 2026-08-15 00:58:58
요약
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults. Researchers will compare participants receiving GLM101 to those receiving a placebo to see if GLM101 improves symptoms of PMM2-CDG.
The study includes two treatment parts: a 24-week double blind placebo-controlled treatment period (Part A), and a 24-week open-label phase where every participant will receive GLM101(Part B).
The study includes two treatment parts: a 24-week double blind placebo-controlled treatment period (Part A), and a 24-week open-label phase where every participant will receive GLM101(Part B).
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