A Phase 2/3 interventional study of CDZ173 in Activated PI3Kdelta Syndrome (APDS); PASLI Disease, sponsored by Pharming Technologies B.V.. Terminated at 8 sites in 7 countries. Open to participants aged 12 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-03-30.
Sponsored by Pharming Technologies B.V. · Phase 2/3, Interventional, and Treatment
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.
105 studies on the registry are indexed under Lymphadenopathy; 14 are open to participants now.
This study's enrollment of 37 is below the median of 98 across 69 interventional studies indexed under Lymphadenopathy.
Browse Lymphadenopathy studies →Pharming Technologies B.V. is the lead sponsor of 22 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
- Any medically significant disease or condition that is unrelated to APDS/PASLI
Other protocol-defined inclusion/exclusion criteria may apply.
140mg/day
Drug: CDZ173
140 mg/day
To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173
Number of participants with adverse events reported, including serious adverse events
Time frame: 6 years 3 months
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.
Time frame: At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation
Responder Analysis Soluble Protein Biomarkers - naïve B Cells
Responder analysis soluble protein biomarkers - Change in naïve b cells from baseline up to Day 252
Time frame: up to 252 days
Sum of Product of Diameters (SPD) of Index Lesions
Participants were scanned through MRI or CT imaging
Time frame: Baseline and D252
Spleen Organ Evaluation - Volume in mm3
Participants were scanned through MRI or CT
Time frame: Baseline and D252
| Milestone | CDZ173 |
|---|---|
| Started | 37 |
| Completed | 3 |
| Not completed | 34 |
| Withdrew: Death | 1 |
| Withdrew: Physician decision | 3 |
| Withdrew: Withdrawal by subject | 2 |
| Withdrew: Adverse event | 1 |
| Withdrew: Study termination | 25 |
| Withdrew: Lost to follow-up | 2 |
Number of participants with adverse events reported, including serious adverse events
| Participants | CDZ173 |
|---|---|
| To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173 | 37 |
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.
| score on a scale | CDZ173 at Baseline | CDZ173 After 1 Year | CDZ173 After 3 Years | CDZ173 After 4 Years | CDZ173 After 5 Years |
|---|---|---|---|---|---|
| To Evaluate the Long Term Efficacy of CDZ173 Using SF-36 General Health Score | 36.540 (18.95 to 60.32) | 46.050 (26.08 to 66.50) | 48.430 (29.41 to 62.70) | 48.430 (34.17 to 62.70) | 47.240 (36.54 to 57.94) |
Responder analysis soluble protein biomarkers - Change in naïve b cells from baseline up to Day 252
| percentage of naive B cells | CDZ173 at Baseline | CDZ173 After D84 | CDZ173 After D252 |
|---|---|---|---|
| Responder Analysis Soluble Protein Biomarkers - naïve B Cells | 58.16 ± 20.922 | 23.58 ± 16.177 | 32.42 ± 25.293 |
Participants were scanned through MRI or CT imaging
| mm2 | CDZ173 at Baseline | CDZ173 at D252 |
|---|---|---|
| Sum of Product of Diameters (SPD) of Index Lesions | 1648.283 ± 1754.6045 | 565.125 ± 424.4619 |
Participants were scanned through MRI or CT
| Organ volume in mm3 | CDZ173 at Baseline | CDZ173 at D252 |
|---|---|---|
| Spleen Organ Evaluation - Volume in mm3 | 606893.175 ± 349359.8341 | 385025.500 ± 228724.7324 |
Collected over Adverse events are collected throughout participation in the study, from signing informed consent up to the last study visit (EoS). Study duration differs per participant with a maximum of 6 years.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| CDZ173 | 1/37 (2.7%) | 10/37 (27%) | 34/37 (91.9%) |
| Event | CDZ173 |
|---|---|
| Abdominal painGastrointestinal disorders | 2/37 |
| PneumoniaInfections and infestations | 2/37 |
| Hodgkin's diseaseNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/37 |
| Transitional cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/37 |
| deep vein thrombosisVascular disorders | 1/37 |
| HypotentionVascular disorders | 1/37 |
| Orthostatic hypotensionVascular disorders | 1/37 |
| Facial painGeneral disorders | 1/37 |
| PyrexiaGeneral disorders | 1/37 |
| Anaphylactic reactionImmune system disorders | 1/37 |
| Event | CDZ173 |
|---|---|
| SARS-CoV-2 test negativeInvestigations | 15/37 |
| COVID-19Infections and infestations | 12/37 |
| Upper respiratory tract infectionInfections and infestations | 10/37 |
| pyrexiaGeneral disorders | 9/37 |
| HeadacheNervous system disorders | 8/37 |
| Otitis externaInfections and infestations | 7/37 |
| SinusitisInfections and infestations | 6/37 |
| NasopharyngitisInfections and infestations | 6/37 |
| weight increasedInvestigations | 5/37 |
| VomitingGastrointestinal disorders | 5/37 |
All enrolled and treated participants
| Age, Categorical(Participants) | CDZ173 |
|---|---|
| <=18 years | 13 |
| Between 18 and 65 years | 24 |
| >=65 years | 0 |
| Age, Continuous(years) | CDZ173 |
|---|---|
| Mean | 22.7 (12 to 46) |
| Sex: Female, Male(Participants) | CDZ173 |
|---|---|
| Female | 16 |
| Male | 21 |
| Race (NIH/OMB)(Participants) | CDZ173 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 31 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
| Region of Enrollment(participants) | CDZ173 |
|---|---|
| Netherlands | 2 |
| Czechia | 4 |
| United States | 20 |
| Italy | 5 |
| Belarus | 1 |
| Germany | 3 |
| Russia | 2 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: 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
This study is terminated, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Activated PI3K-delta Syndrome
Pharming Technologies B.V.