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Pediatric Patients Aged 4 to 11 Years With APDS

NCT05438407 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-05-06

No results posted yet for this study

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).

Conditions

  • APDS

Interventions

DRUG

Leniolisib

The doses selected range from 20 to 70 mg BID (resulting in total daily doses ranging from 40 to 140 mg per day) based on weight. The doses will be administered as (a combination of) 10 mg and 30 mg tablets

Sponsors & Collaborators

  • CMIC Co, Ltd. Japan

    collaborator INDUSTRY

  • Labcorp Central Laboratory

    collaborator UNKNOWN

  • Fortrea

    collaborator INDUSTRY

  • Aixial Group

    collaborator INDUSTRY

  • Pharming Technologies B.V.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-01
Primary Completion
2026-11-30
Completion
2026-12-30
FDA Drug
Yes

Countries

  • United States
  • France
  • Japan

Study Locations

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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 NCT05438407 on ClinicalTrials.gov