drugset / Trial / NCT05693129
Pediatric Patients Aged 1 to 6 Years With APDS
Phase 3
Active not recruiting
16 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 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Timeline
- Start
- 2023-08-30
- Primary completion
- 2028-07-09
- Completion
- 2028-10-28
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Leniolisib | Small molecule | 10 mg | Oral |
| Subject | Leniolisib | Small molecule | 50 mg | Oral |