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Multicenter study of calcifediol in symptomatic carriers of dysferlinopathy
2007-005808-41Phase 4
Key facts
- Status
- Active
- Start
- Jan 2008
- Countries
- 1
- Sponsor
- Hospital de la Santa Creu i Sant Pau Research Institute
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Drugs
- calcifediol
- Conditions
- dysferlinopathy
01
Overview
Phase 4, single-arm trial of calcifediol in dysferlinopathy, sponsored by Hospital de la Santa Creu i Sant Pau Research Institute. Primary endpoints: Clinical and analytical response to calcifediol and Change in dysferlin expression in monocytes. Started Jan 2008; status: active.
02
Design
1 arm- Study design
- Non-randomizedSingle group
Calcifediol monotherapy
Symptomatic carriers of dysferlin gene mutations receive calcifediol to evaluate clinical, analytical, and dysferlin expression responses.
03
Who it enrols
Adults aged 18 to 60 years who are symptomatic carriers of a mutation in the dysferlin gene (dysferlinopathy). Patients already treated with calcifediol are excluded.
04
Endpoints
Primary
Clinical and analytical response to calcifediol
Change in dysferlin expression in monocytes
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