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Sirolimus safety and tolerability study in primary immunodeficiency
jRCTs031230296Recruiting
- Sponsor
- Japan Agency for Medical Research and Development
- Start
- Sep 2023
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label trial of sirolimus in Immunogenetic disorder, Activated PI3Kδ syndrome (APDS), CTLA4 deficiency and 5 more (2 cohorts), sponsored by Japan Agency for Medical Research and Development. Primary endpoint: Frequency and rate of adverse events. Started Sep 2023; status: recruiting.
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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