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Phase 2 sirolimus study in primary immunodeficiency with mTOR pathway overactivation
jRCT2031210309Phase 2Completed
- Sponsor
- Japan Agency for Medical Research and Development
- Start
- Dec 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sirolimus in Immunogenetic disorder, Activated PI3Kδ syndrome (APDS), CTLA4 deficiency and 4 more, sponsored by Japan Agency for Medical Research and Development. Primary endpoint: Primary efficacy endpoint at Week 12. Started Dec 2022; status: completed.
02
Drugs
sirolimus
03
Conditions
Immunogenetic disorderActivated PI3Kδ syndrome (APDS)CTLA4 deficiencyLRBA deficiency (LRBA)Immunodysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome (IPEX)
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