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NCT05693129 · ClinicalTrials.gov registry record · Phase 3
Pediatric Patients Aged 1 to 6 Years With APDS
A Phase 3 study of APDS, sponsored by Pharming Technologies B.V..
- Active
- Registry status
- Phase 3
- Development phase
- 16
- Enrollment target
- 9
- Study locations
NCT05693129: Active Phase 3 study of APDS, sponsored by Pharming Technologies B.V..
NCT05693129 is a Phase 3 study of APDS that is active but no longer recruiting, run by Pharming Technologies B.V.. The registered enrollment target is 16 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower). The trial reports 9 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05693129, a Phase 3 study of APDS, is active but no longer recruiting, sponsored by Pharming Technologies B.V..
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 16 participants
- Enrollment target
- 9
- Study locations
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 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Primary Outcome
To assess number of Participants with TEAEs, SAEs, and AEs leading to discontinuation of study drug
Conditions Studied
Interventions
- DRUG Leniolisib
Study Locations (9)
Other
- Kyoto University Hospital - Kyoto
- Institute of Science Tokyo Hospital - Tokyo
- Hospital Pediátrico de Coimbra da ULS Coimbra UNIDADE LOCAL DE SAÚDE DE COIMBRA - Coimbra
- Hospital Universitario Virgen del Rocío - Seville
- Great Ormond Street Hospital - London
California
- University of California Los Angeles - Los Angeles
Maryland
- National Institutes of Health - Bethesda
Ohio
- Rainbow Childrens Hospital - Shaker Heights
Texas
- Texas Children's Hospital - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2023-08-30 |
| Est. Completion | 2026-10-28 |
| Phase | Phase 3 |
What the registry record for NCT05693129 still lists
NCT05693129 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower).
The record links to 1 condition, with APDS appearing as the primary indexed condition, and to 1 intervention - of which Leniolisib is the first listed.
NCT05693129 lists 9 locations in 5 states (Other, California, Maryland).
Frequently Asked Questions
What is clinical trial NCT05693129 about?
NCT05693129 is a clinical study titled "Pediatric Patients Aged 1 to 6 Years With APDS". 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) syndro...
What is the current status of trial NCT05693129?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 16 participants. The study started on 2023-08-30. Estimated completion is 2026-10-28.
What conditions does trial NCT05693129 study?
This clinical trial studies the following conditions: APDS.
What interventions are being tested in trial NCT05693129?
The interventions under investigation include: Leniolisib (DRUG).
Who is sponsoring clinical trial NCT05693129?
This trial is sponsored by Pharming Technologies B.V., which has 11 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05693129 being conducted?
This trial has 9 study locations across California, Maryland, Ohio, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for APDS
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05693129's enrollment target sits among peer trials
16 1974th of 2000 higher than 24 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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