Activated PI3Kdelta Syndrome (APDS); PASLI DiseasePhase 2/3NCT02859727
Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
A Phase 2/3 study testing CDZ173 in people with Activated PI3Kdelta Syndrome (APDS); PASLI Disease, run by Pharming Technologies B.V. at 8 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Activated PI3Kdelta Syndrome (APDS); PASLI Disease
- What is being tested?
- CDZ173
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 8 sitesin 7 countries
- Study dates
- 8 Sep 2016to 30 Jan 2025
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Any medically significant disease or condition that is unrelated to APDS/PASLI
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
8 sites in 7 countries.
8 sites in 7 countries
Italy2 sites
Pharming Investigative Site
Brescia, BS, 25123
Pharming Investigative Site
Palermo, PA, 90127
United States1 site
Pharming Investigative Site
Bethesda, Maryland, 20892
About this study
Official title: An Open-label, Non-randomized Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)
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.
- Being tested
- CDZ173
- Keywords
- APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;
- Medical subject headings
- Activated PI3K-delta Syndrome, Lymphadenopathy, Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CDZ173Drug
140 mg/day
| Study group | Role | What they receive |
|---|---|---|
| CDZ173 | Experimental | 140mg/day Receives: Drug: CDZ173 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173
Number of participants with adverse events reported, including serious adverse events
Measured: 6 years 3 months
Secondary outcomes (1)
To Evaluate the Long Term Efficacy of CDZ173 Using SF-36 General Health Score
SF-36 (Short Form 36) Survey general health score per participant reported for duration of particpation in study. Scores are reported on a scale of 0 - 100. All items are scored so that a high score defines a more favorable health state.
Measured: At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2016 (estimated)
- Study start8 Sep 2016
- Primary completion30 Jan 2025
- Study completion30 Jan 2025
Why it stopped: All participant have transitioned to commercial product or compassionate use.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Activated PI3Kdelta Syndrome (APDS); PASLI DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02859727
- Sponsor's protocol ID
- CCDZ173X2201E1
- Other IDs
- 2016-000468-41, 2016
- Status last verified
- Mar 2026
- First submitted
- 4 Aug 2016
- Last updated
- 30 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Publications
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.
PubMed 38593221Rao 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.
PubMed 37797893
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-randomized Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)
- ClinicalTrials.gov ID
- NCT02859727
- Acronym / sponsor ID
- CCDZ173X2201E1
- Keywords
- APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: All participant have transitioned to commercial product or compassionate use.
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- 8 Sep 2016 (actual)
- Primary completion
- 30 Jan 2025 (actual)
- Study completion
- 30 Jan 2025 (actual)
- First posted
- 9 Aug 2016 (estimated)
- Results
- Posted 30 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CDZ173 | Experimental | Drug: CDZ173140mg/day |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- Any medically significant disease or condition that is unrelated to APDS/PASLI
- Other protocol-defined inclusion/exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CDZ173140 mg/day | Drug | CDZ173 |
Primary outcome
To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173
Time frame: 6 years 3 months
Number of participants with adverse events reported, including serious adverse events
1 secondary outcome
To Evaluate the Long Term Efficacy of CDZ173 Using SF-36 General Health Score
Time frame: At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38593221Rao 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.
- Derived from this study: PubMed 37797893Rao 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.
- Individual participant dataSharing planned: Undecided. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing CDZ173 in people with Activated PI3Kdelta Syndrome (APDS); PASLI Disease, run by Pharming Technologies B.V. at 8 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Italy, United States, Belarus, Czechia, Germany and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173 (time frame: 6 years 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02859727, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2026.