NCT02859727

Brief Summary

This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were treated previously with PI3Kδ inhibitors other than CDZ173. The study is open-label designed to establish the long-term safety, tolerability, efficay and pharmacokinetics of CDZ173 in the target population.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
37

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2016

Longer than P75 for phase_2

Geographic Reach
7 countries

8 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 4, 2016

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 9, 2016

Completed
1 month until next milestone

Study Start

First participant enrolled

September 8, 2016

Completed
8.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 30, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 30, 2025

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

March 30, 2026

Completed
Last Updated

March 30, 2026

Status Verified

March 1, 2026

Enrollment Period

8.4 years

First QC Date

August 4, 2016

Results QC Date

December 11, 2025

Last Update Submit

March 9, 2026

Conditions

Activated PI3Kdelta Syndrome (APDS); PASLI Disease

Keywords

APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;

Outcome Measures

Primary Outcomes (1)

  • To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173

    Number of participants with adverse events reported, including serious adverse events

    6 years 3 months

Secondary Outcomes (1)

  • To Evaluate the Long Term Efficacy of CDZ173 Using SF-36 General Health Score

    At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation

Other Outcomes (3)

  • Responder Analysis Soluble Protein Biomarkers - naïve B Cells

    up to 252 days

  • Sum of Product of Diameters (SPD) of Index Lesions

    Baseline and D252

  • Spleen Organ Evaluation - Volume in mm3

    Baseline and D252

Study Arms (1)

CDZ173

EXPERIMENTAL

140mg/day

Drug: CDZ173

Interventions

CDZ173DRUG

140 mg/day

CDZ173

Eligibility Criteria

Age12 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Written informed consent must be obtained before any assessment is performed.
  • Paients must have participated in the study CCDZ173X2201 or were treated previously with PI3Kδ inhibitors other than CDZ173.
  • Patients who are deemed by the Investigator to benefit from PI3Kδ inhibitor therapy.
  • Patients or their legal representatives (for patients under the age of 18 years) must be able to communicate well with the Investigator, to understand and comply with the requirements of the study.
  • Documented APDS/PASLI-associated genetic PI3K delta mutation.

You may not qualify if:

  • \- Any medically significant disease or condition that is unrelated to APDS/PASLI

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Pharming Investigative Site

Bethesda, Maryland, 20892, United States

Location

Pharming Investigative Site

Minsk, 223053, Belarus

Location

Pharming Investigative Site

Prague, CZE, 15006, Czechia

Location

Pharming Investigative Site

Dresden, 01307, Germany

Location

Pharming Investigative Site

Brescia, BS, 25123, Italy

Location

Pharming Investigative Site

Palermo, PA, 90127, Italy

Location

Pharming Investigative Site

Rotterdam, 3000 CA, Netherlands

Location

Pharming Investigative Site

Moscow, 117198, Russia

Location

Related Publications (2)

  • Rao VK, Kulm E, Grossman J, Buchbinder D, Chong H, Bradt J, Webster S, Sediva A, Dalm VA, Uzel G. Long-term treatment with selective PI3Kdelta inhibitor leniolisib in adults with activated PI3Kdelta syndrome. Blood Adv. 2024 Jun 25;8(12):3092-3108. doi: 10.1182/bloodadvances.2023011000.

    PMID: 38593221DERIVED

  • Rao VK, Kulm E, Sediva A, Plebani A, Schuetz C, Shcherbina A, Dalm VA, Trizzino A, Zharankova Y, Webster S, Orpia A, Korholz J, Lougaris V, Rodina Y, Radford K, Bradt J, Relan A, Holland SM, Lenardo MJ, Uzel G. Interim analysis: Open-label extension study of leniolisib for patients with APDS. J Allergy Clin Immunol. 2024 Jan;153(1):265-274.e9. doi: 10.1016/j.jaci.2023.09.032. Epub 2023 Oct 4.

    PMID: 37797893DERIVED

MeSH Terms

Conditions

Activated PI3K-delta SyndromeLymphadenopathyImmunologic Deficiency Syndromes

Interventions

leniolisib

Condition Hierarchy (Ancestors)

Lymphatic DiseasesHemic and Lymphatic DiseasesImmune System Diseases

Results Point of Contact

Title
Jason Bradt
Organization
Pharming Technologies B.V.
j.bradt@pharming.com
0715247400

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 4, 2016

First Posted

August 9, 2016

Study Start

September 8, 2016

Primary Completion

January 30, 2025

Study Completion

January 30, 2025

Last Updated

March 30, 2026

Results First Posted

March 30, 2026

Record last verified: 2026-03

Locations

CZ(1)US(1)IT(2)NL(1)RU(1)BE(1)DE(1)