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Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome

NCT03383380 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2024-07-09

No results posted yet for this study

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).

Conditions

  • Activated PI3K-delta Syndrome
  • Immunodeficiency Primary

Interventions

DRUG

Rapamycin

Gain-of-function (GOF) PIK3CD mutations lead to PI3Kδ hyperactivity, with the downstream mediators Akt and mTOR hyperphosphorylated. The mammalian/mechanistic target of rapamycin inhibitor rapamycin may be effective to control the progress of this disease.

Sponsors & Collaborators

  • Children's Hospital of Fudan University

    lead OTHER

Principal Investigators

  • Jinqiao Sun, Ph.D.,M.D · Children's Hospital of Fudan University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-01
Primary Completion
2022-11-30
Completion
2023-11-30

Countries

  • China

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03383380 on ClinicalTrials.gov