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ACTIVE NOT RECRUITING Phase 3

Pediatric Patients Aged 1 to 6 Years With APDS

NCT05693129 · View on ClinicalTrials.gov ↗

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)

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

FieldValue
Enrollment Target 16 participants
Start Date 2023-08-30
Est. Completion 2026-10-28
Phase Phase 3

Sponsor

Pharming Technologies B.V.

5 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT05693129

The ClinicalTrials.gov registry entry for NCT05693129 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 16 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Pharming Technologies B.V., which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT05693129 reports 9 study locations spanning 5 distinct geographic areas — top geographies include Other, California, Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT05693129?

The interventions under investigation include: Leniolisib (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT05693129?

This trial is sponsored by Pharming Technologies B.V., which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial