Adults 12 to 75, any sex, with Common Variable Immunodeficiency (CVID), APDS / PASLI. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Part I: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Primary· From the start of treatment to 30 days after end of treatment, assessed up to maximum duration of 114 days
Number of participants with AEs and SAEs, including significant changes from baseline in physical findings, vital signs, electrocardiograms and laboratory values qualifying and reported as AEs. The number of participants in each category (AEs and SAEs) is reported per dose level: CDZ173 10 mg from Day 1 to Day 28, CDZ173 30 mg from day 29 to day 56 and CDZ173 70 mg from day 57 to day 84.
CDZ173 10 mg AEs
Group
Value
95% CI
Part I: CDZ173 10 mg
2
CDZ173 10 mg SAEs
Group
Value
95% CI
Part I: CDZ173 10 mg
0
CDZ173 30 mg AEs
Group
Value
95% CI
Part I: CDZ173 10 mg
2
CDZ173 30 mg SAEs
Group
Value
95% CI
Part I: CDZ173 10 mg
0
CDZ173 70 mg AEs
Group
Value
95% CI
Part I: CDZ173 10 mg
4
CDZ173 70 mg SAEs
Group
Value
95% CI
Part I: CDZ173 10 mg
0
Part I: CDZ173 Dose ConcentrationPrimary· Days 1, 29 and 57 (0.25 and 3 h post morning dose) and Day 84
Venous whole blood samples were collected for the assessment of the dose-PD and the PK/PD relationship of CDZ173 in participants with APDS/PASLI for dose selection in Part II. CDZ173 was determined by a validated Liquid chromatography - Mass spectometry (LC-MS) method; anticipated Lower Limit of Quantification (LLOQ) was 3 ng/mL. Concentrations below the LLOQ were reported as "zero" and no methods for imputation of missing data were used.
Day 1: 0.25 h post-dose
Group
Value
95% CI
Part I: CDZ173
10.10
± 1.10
Day 1: 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
321.00
± 115.00
Day 29: 0.25 h post-dose
Group
Value
95% CI
Part I: CDZ173
249.00
± 540.00
Day 29: 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
916.00
± 185.00
Day 57: 0.25 h post-dose
Group
Value
95% CI
Part I: CDZ173
150.00
± 143.00
Day 57: 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
1710.00
± 782.00
Day 84
Group
Value
95% CI
Part I: CDZ173
998.00
± 455.00
Part I: Percentage of Inhibition of Unstimulated and Stimulated pAkt Levels in B CellsPrimary· Baseline, days 29 and 57 (3 and 12 h post-dose) and day 84
Phosphorylation of Akt in ex vivo stimulated and unstimulated B cells was quantified at baseline and at the end of the 4-week treatment period for each of the three dose levels. Determination of the percentage (%) of CD20B+ phospho-Akt positive cells after ex vivo stimulation of whole blood was performed by flow cytometry analysis. The percentage of inhibition of pAkt was defined as (-1) \* percent change from baseline pAkt value. Unstimulated cells served as controls at each time point. Baseline was defined as the mean of the day -1 value and the pre-dose value on Day 1 when both were availab
CD20B Unstimulated: Day 29 - 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
82.07
± 7.25
CD20B Stimulated: Day 29 - 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
78.00
± 7.25
CD20B Unstimulated: Day 29 - 12 h post-dose
Group
Value
95% CI
Part I: CDZ173
50.58
± 18.73
CD20B Stimulated: Day 29 - 12 h post-dose
Group
Value
95% CI
Part I: CDZ173
47.14
± 7.83
CD20B Unstimulated: Day 57 - 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
86.61
± 5.26
CD20B Stimulated: Day 57 - 3 h post-dose
Group
Value
95% CI
Part I: CDZ173
60.98
± 54.05
CD20B Unstimulated: Day 57 - 12 h post-dose
Group
Value
95% CI
Part I: CDZ173
53.18
± 16.59
CD20B Stimulated: Day 57 - 12 h post-dose
Group
Value
95% CI
Part I: CDZ173
63.65
± 21.03
Part II: Change From Baseline in the log10 Transformed Sum of Product of Diameters (SPD) in the Index LesionsPrimary· Baseline and Day 85
For the assessment of the impact of CDZ173 on lymphadenopathy, participants were scanned in a magnetic resonance imaging (MRI) or a computed tomography (CT) scanner as based on clinical practice and local regulation. MRI or CT imaging of the neck, chest, abdomen and pelvis was performed. Index lesions were selected from measurable nodal and extranodal lesions as per the Cheson methodology. A maximum of six of the largest dominant lesions were selected and documented at baseline and assessed again at the end of treatment. The change in lymph node size was measured using the log10 transformed su
Group
Value
95% CI
Part II: CDZ173
-0.30
± 0.04
Part II: Placebo
-0.06
± 0.06
Part II: Change From Baseline in Percentage of naïve B Cells Out of Total B CellsPrimary· Baseline and Day 85
APDS/PASLI patients suffer from dysregulation in B cell function and differentiation with low numbers of naive B cells. Change from baseline in percentage of naïve B cells out of total B cells at the end of treatment was assessed by flow cytometry to evaluate the pharmacodynamic effect of CDZ173 on B cell immunophenotyping. A higher percentage in naïve B out of total B cells is a positive outcome. A positive change from baseline indicates improvement.
Group
Value
95% CI
Part II: CDZ173
34.76
± 3.08
Part II: Placebo
-5.37
± 3.95
Part I & II: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) for CDZ173Secondary· Part I: Days 1, 29 and 57 / Part II: Day 1
Venous whole blood samples were collected for activity-based pharmacokinetics characterization. AUClast was calculated from plasma concentration-time data using non-compartmental methods. AUClast was calculated at the first day of every CDZ173 dose level (10, 30 and 70 mg) for Part I and (70 mg) for Part II. No methods for imputation of missing data were used.
Day 1
Group
Value
95% CI
Part I: CDZ173
1760.0
± 441.0
Part II: CDZ173
10400.0
± 2800.0
Day 29
Group
Value
95% CI
Part I: CDZ173
4760.0
± 816.0
Day 57
Group
Value
95% CI
Part I: CDZ173
10800.0
± 3310.0
Part I & II: Maximum Observed Plasma Concentration (Cmax) for CDZ173Secondary· Part I: Days 1, 29 and 57 / Part II: Day 1
Venous whole blood samples were collected for activity-based pharmacokinetics characterization. Cmax was calculated from plasma concentration-time data using non-compartmental methods. Cmax was calculated at the first day of every CDZ173 dose level (10, 30 and 70 mg) for Part I and (70 mg) for Part II. No methods for imputation of missing data were used.
Day 1
Group
Value
95% CI
Part I: CDZ173
393.0
± 137.0
Part II: CDZ173
2150.0
± 576.0
Day 29
Group
Value
95% CI
Part I: CDZ173
1060.0
± 222.0
Day 57
Group
Value
95% CI
Part I: CDZ173
2540.0
± 747.0
Part I & II: Mental Component Summary (MCS) and Physical Component Summary (PCS) From Short Form 36 (SF-36) SurveySecondary· Part I: Baseline and Days 29, 57 and 84 / Part II: Baseline and Days 29, 57 and 85
The SF-36 is a widely used and extensively studied instrument to measure health-related quality of life (HRQoL) among healthy subjects and patients with acute and chronic conditions. It consists of eight subscales that can be scored individually: Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role-Emotional, and Mental Health. The subscales are aggregated to derive two overall summary scores: the Physical Component Summary (PCS) and the Mental Component Summary (MCS) scores. PCS and MCS scores range from 0 to 100 with a higher score indicating a
Day -1: Mental Component Summary
Group
Value
95% CI
Part I: CDZ173
47.94
± 8.22
Part II: CDZ173
47.36
± 7.98
Part II: Placebo
45.94
± 8.14
Day -1: Physical Component Summary
Group
Value
95% CI
Part I: CDZ173
47.54
± 9.24
Part II: CDZ173
44.49
± 7.08
Part II: Placebo
44.06
± 8.59
Day 29: Mental Component Summary
Group
Value
95% CI
Part I: CDZ173
47.94
± 8.22
Part II: CDZ173
49.98
± 8.06
Part II: Placebo
49.52
± 6.70
Day 29: Physical Component Summary
Group
Value
95% CI
Part I: CDZ173
47.54
± 9.24
Part II: CDZ173
47.87
± 7.66
Part II: Placebo
44.62
± 7.57
Day 57: Mental Component Summary
Group
Value
95% CI
Part I: CDZ173
47.94
± 8.22
Part II: CDZ173
49.12
± 8.17
Part II: Placebo
45.92
± 7.31
Day 57: Physical Component Summary
Group
Value
95% CI
Part I: CDZ173
47.54
± 9.24
Part II: CDZ173
47.04
± 7.30
Part II: Placebo
47.20
± 9.75
Day 84 (Part I) / Day 85 (Part II): Mental Component Summary
Group
Value
95% CI
Part I: CDZ173
47.94
± 8.22
Part II: CDZ173
49.22
± 8.17
Part II: Placebo
47.32
± 8.73
Day 84 (Part I) / Day 85 (Part II): Physical Component Summary
Group
Value
95% CI
Part I: CDZ173
47.54
± 9.24
Part II: CDZ173
47.59
± 6.22
Part II: Placebo
47.48
± 8.48
Part I & II: Overall Work Impairment Due to Health Score From Work Productivity Activity Impairment and Classroom Impairment Questionnaire (WPAI-CIQ)Secondary· Part I: Baseline and Days 29, 57 and 84 / Part II: Baseline and Days 29, 57 and 85
The Work Productivity Activity Impairment (WPAI) questionnaire measures the amount of absence or presence for work attendance and daily work activity impairment attributable to APDS/PASLI. As younger participants (age 12 and above) were enrolled in the study the WPAI-CIQ was used for all participants as it also measures the amount of absence or presence for school attendance and daily classroom activity impairment. Participants responded for classroom or work-related questions depending on their situation. WPAI-CIQ consists of 10 questions that yield 4 types of scores: absenteeism, presenteeis
Baseline
Group
Value
95% CI
Part I: CDZ173
25.00
± 35.36
Part II: CDZ173
51.25
± 37.23
Part II: Placebo
5.00
± 7.07
Day 29
Group
Value
95% CI
Part I: CDZ173
44.00
± 0
Part II: CDZ173
35.31
± 23.12
Part II: Placebo
5.00
± 7.07
Day 57
Group
Value
95% CI
Part I: CDZ173
62.31
± 0
Part II: CDZ173
35.49
± 24.70
Part II: Placebo
10.00
± 14.14
Day 84 (Part I) / Day 85 (Part II)
Group
Value
95% CI
Part I: CDZ173
44.41
± 7.90
Part II: CDZ173
35.59
± 31.85
Part II: Placebo
25.00
± 7.07
Part I & II: Overall Classroom Impairment Due to Health Score From the Work Productivity Activity Impairment and Classroom Impairment Questionnaire (WPAI-CIQ)Secondary· Part I: Baseline and Days 29, 57 and 84 / Part II: Baseline and Days 29, 57 and 85
The Work Productivity Activity Impairment (WPAI) questionnaire measures the amount of absence or presence for work attendance and daily work activity impairment attributable to APDS/PASLI. As younger participants (age 12 and above) were enrolled in the study the WPAI-CIQ was used for all participants as it also measures the amount of absence or presence for school attendance and daily classroom activity impairment. Participants responded for classroom or work-related questions depending on their situation. WPAI-CIQ consists of 10 questions that yield 4 types of scores: absenteeism, presenteeis
Baseline
Group
Value
95% CI
Part I: CDZ173
65.68
± 33.49
Part II: CDZ173
47.33
± 44.75
Part II: Placebo
23.75
± 30.92
Day 29
Group
Value
95% CI
Part I: CDZ173
57.30
± 18.09
Part II: CDZ173
0.00
± 0
Part II: Placebo
22.65
± 24.37
Day 57
Group
Value
95% CI
Part I: CDZ173
70.13
± 18.68
Part II: CDZ173
12.14
± 3.03
Part II: Placebo
22.40
± 26.16
Day 84 (Part I) / Day 85 (Part II)
Group
Value
95% CI
Part I: CDZ173
68.52
± 18.74
Part II: CDZ173
51.00
± 0.00
Part II: Placebo
5.00
± 7.07
Part I & II: Physician's Global Assessment (PGA)Secondary· Part I: Baseline and Days 29, 57 and 84 / Part II: Baseline and Days 29, 57 and 85
In the physician's global assessment questionnaire the Investigator rated the disease activity of their patient using 100 mm Visual analogue Scale (VAS) ranging from "no disease activity" (0) to "maximal disease activity" (100). To enhance objectivity, the physician was not aware of the specific patient's global assessment, when performing his own assessment on that patient. No methods for imputation of missing data were used.
Baseline
Group
Value
95% CI
Part I: CDZ173
34.7
± 17.07
Part II: CDZ173
47.10
± 17.65
Part II: Placebo
41.38
± 17.78
Day 29
Group
Value
95% CI
Part I: CDZ173
21.8
± 9.70
Part II: CDZ173
38.81
± 23.73
Part II: Placebo
29.75
± 9.99
Day 57
Group
Value
95% CI
Part I: CDZ173
22.5
± 13.55
Part II: CDZ173
34.02
± 18.89
Part II: Placebo
24.13
± 18.34
Day 84 (Part I) / Day 85 (Part II)
Group
Value
95% CI
Part I: CDZ173
8.8
± 4.12
Part II: CDZ173
26.70
± 22.82
Part II: Placebo
25.88
± 16.15
Part I & II: Patient's Global Assessment (PtGA)Secondary· Part I: Baseline and Days 29, 57 and 84 / Part II: Baseline and Days 29, 57 and 85
In the patient's global assessment questionnaire patients are asked about their APDS/PASLI related well-being using 100 mm visual analogue scale (VAS) ranging from "very poor" (0) to "very good" (100). No methods for imputation of missing data were used.
Baseline
Group
Value
95% CI
Part I: CDZ173
62.0
± 21.90
Part II: CDZ173
54.53
± 21.47
Part II: Placebo
62.50
± 26.56
Day 29
Group
Value
95% CI
Part I: CDZ173
65.0
± 22.21
Part II: CDZ173
68.37
± 19.31
Part II: Placebo
57.75
± 24.03
Day 57
Group
Value
95% CI
Part I: CDZ173
67.5
± 21.83
Part II: CDZ173
64.79
± 19.61
Part II: Placebo
69.50
± 21.93
Day 84 (Part I) / Day 85 (Part II)
Group
Value
95% CI
Part I: CDZ173
72.5
± 13.37
Part II: CDZ173
67.58
± 16.57
Part II: Placebo
60.25
± 23.66
Adverse events — posted to ClinicalTrials.gov
Time frame: Part I and Part II: Adverse events (AEs) were reported from the start of treatment to 30 days after end of treatment, assessed up to maximum duration of 114 days for Part I and 115 days for Part II. For the subset of participants in Part II that rolled over to the extension study (CCDZ173X2201E1) directly after the last treatment dose in Part II, AEs were reported from the start of treatment to end of treatment, assessed up to maximum duration of 85 days..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This study was designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency (APDS/PASLI).
The study consisted of two parts: Part I was the open label part designed to establish the safety and pharmacokinetics of CDZ173 in the target population, as well as to select the optimal dose to be tested in Part II. Part II was designed to assess efficacy and safety of CDZ173 in the target population.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT02859727 — Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
· Phase 2, PHASE3
· terminated
NCT02775916 — Safety, Pharmacokinetics, and Preliminary Efficacy Study of CDZ173 in Patients With Primary Sjögren's Syndrome
· Phase 2
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 10 August 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02435173.