drugset / Trial / NCT05438407
Pediatric Patients Aged 4 to 11 Years With APDS
Phase 3
Active not recruiting
15 enrolled
Pharming Technologies B.V.
Aixial Group · collabCMIC Co, Ltd. Japan · collabFortrea · collabLabcorp Central Laboratory · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 4 to 11 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
Timeline
- Start
- 2023-02-01
- Primary completion
- 2026-11-30
- Completion
- 2026-12-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Leniolisib | Small molecule | 20 mg | Oral |
| Subject | Leniolisib | Small molecule | 70 mg | Oral |