Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome

• Status: Recruiting
• Trial end date: 2022-11-30
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Fudan University
• Treatments: Sirolimus

Criteria

Inclusion Criteria:

1. Patients with activated phosphoinositide 3-kinase δ syndrome

2. No more than 18 years old

Exclusion Criteria:

1. Patients with serious fungous infection

2. Patients with serious complications

3. Lack of parental consent