Status:
COMPLETED
Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome
Lead Sponsor:
Children's Hospital of Fudan University
Conditions:
Activated PI3K-delta Syndrome
Immunodeficiency Primary
Eligibility:
All Genders
Up to 18 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this proposed research is to evaluate the efficacy and safety of the rapamycin therapy in patients with activated phosphoinositide 3-kinase δ syndrome (APDS).
Detailed Description
Activated phosphoinositide 3-kinase δ syndrome (APDS) is a recently described autosomal dominant primary immunodeficiency (PID), caused by the mutations in PIK3CD gene. The manifestations of APDS main...
Eligibility Criteria
Inclusion
- Patients with activated phosphoinositide 3-kinase δ syndrome
- No more than 18 years old
Exclusion
- Patients with serious fungous infection
- Patients with serious complications
- Lack of parental consent
Key Trial Info
Start Date :
December 1 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 30 2023
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT03383380
Start Date
December 1 2017
End Date
November 30 2023
Last Update
July 9 2024
Active Locations (2)
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1
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China, 201102
2
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China