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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-000468-41 | EudraCT Number |
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All participant have transitioned to commercial product or compassionate use.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CDZ173 | Experimental | 140mg/day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CDZ173 | Drug | 140 mg/day |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation |
| Measure | Description | Time Frame |
|---|---|---|
| Responder Analysis Soluble Protein Biomarkers - naïve B Cells | Responder analysis soluble protein biomarkers - Change in naïve b cells from baseline up to Day 252 | up to 252 days |
| Sum of Product of Diameters (SPD) of Index Lesions |
Inclusion Criteria:
Exclusion Criteria:
- Any medically significant disease or condition that is unrelated to APDS/PASLI
Other protocol-defined inclusion/exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pharming Investigative Site | Bethesda | Maryland | 20892 | United States | ||
| Pharming Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38593221 | Derived | 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. | |
| 37797893 | Derived | 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. |
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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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| ID | Title | Description |
|---|---|---|
| FG000 | CDZ173 | 140mg/day CDZ173: 140 mg/day |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All enrolled and treated participants
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| ID | Title | Description |
|---|---|---|
| BG000 | CDZ173 | 140mg/day CDZ173: 140 mg/day |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Number of participants per age group |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173 | Number of participants with adverse events reported, including serious adverse events | Number of participants with adverse events reported, including serious adverse events | Posted | Count of Participants | Participants | 6 years 3 months |
|
|
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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | CDZ173 | 140mg/day CDZ173: 140 mg/day | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hodgkin's disease | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| pyrexia | General disorders | MedDRA 27.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jason Bradt | Pharming Technologies B.V. | 0715247400 | j.bradt@pharming.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 13, 2022 | Dec 11, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 13, 2025 | Dec 11, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| C585640 | Activated PI3K-delta Syndrome |
| D000072281 | Lymphadenopathy |
| D007153 | Immunologic Deficiency Syndromes |
| ID | Term |
|---|---|
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000625376 | leniolisib |
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Participants were scanned through MRI or CT imaging
| Baseline and D252 |
| Spleen Organ Evaluation - Volume in mm3 | Participants were scanned through MRI or CT | Baseline and D252 |
| Minsk |
| 223053 |
| Belarus |
| Pharming Investigative Site | Prague | CZE | 15006 | Czechia |
| Pharming Investigative Site | Dresden | 01307 | Germany |
| Pharming Investigative Site | Brescia | BS | 25123 | Italy |
| Pharming Investigative Site | Palermo | PA | 90127 | Italy |
| Pharming Investigative Site | Rotterdam | 3000 CA | Netherlands |
| Pharming Investigative Site | Moscow | 117198 | Russia |
| Adverse Event |
|
| Study termination |
|
| Lost to Follow-up |
|
| Count of Participants |
| Participants |
|
| Age, Continuous | Age at screening | Mean | Full Range | years |
|
| Sex: Female, Male | Number of participants per group | Count of Participants | Participants |
|
| Race (NIH/OMB) | Number of participants per race group | Count of Participants | Participants |
|
| Region of Enrollment | Number of participants per region | Number | participants |
|
|
| Secondary | 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. | SF-36 general health score during participation in study | Posted | Median | Full Range | score on a scale | At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation |
|
|
|
| Other Pre-specified | Responder Analysis Soluble Protein Biomarkers - naïve B Cells | Responder analysis soluble protein biomarkers - Change in naïve b cells from baseline up to Day 252 | Posted | Mean | Standard Deviation | percentage of naive B cells | up to 252 days |
|
|
|
| Other Pre-specified | Sum of Product of Diameters (SPD) of Index Lesions | Participants were scanned through MRI or CT imaging | PD analysis set | Posted | Mean | Standard Deviation | mm2 | Baseline and D252 |
|
|
|
| Other Pre-specified | Spleen Organ Evaluation - Volume in mm3 | Participants were scanned through MRI or CT | PD analysis set | Posted | Mean | Standard Deviation | Organ volume in mm3 | Baseline and D252 |
|
|
|
| 37 |
| 10 |
| 37 |
| 34 |
| 37 |
| Transitional cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Non-systematic Assessment |
|
| deep vein thrombosis | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Hypotention | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Orthostatic hypotension | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Facial pain | General disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Anaphylactic reaction | Immune system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Suicidal ideation | Psychiatric disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Alanine aminotrasferase increased | Investigations | MedDRA 27.0 | Non-systematic Assessment |
|
| Angina pectoris | Cardiac disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Cardiac arrest | Cardiac disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Anal fissure | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Colitits | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Gastroenteritis | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Pancreatitis acute | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Arthritis reactive | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Abscess | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Acute sinusitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Parotitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Periorbital cellulitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Hypcalcaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Non Cardiac chest pain | General disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| seasonal allergy | Immune system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Illusion | Psychiatric disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| weight increased | Investigations | MedDRA 27.0 | Non-systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 27.0 | Non-systematic Assessment |
|
| Aspartate amoniotransferase increased | Investigations | MedDRA 27.0 | Non-systematic Assessment |
|
| SARS-CoV-2 test negative | Investigations | MedDRA 27.0 | Non-systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Non-systematic Assessment |
|
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 27.0 | Non-systematic Assessment |
|
| Limb injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Haematochezia | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Anal fissure | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Seborrhoeic dermatitis | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Keratosis pilaris | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Skin papilloma | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Otitis externa | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| gastroenteritis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Oral herpes | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Urinary traxt infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Herpes zoster | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Conjunctivitis allergic | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Folliculitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Herpes simplex | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Lyme disease | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Paronychia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Obesity | Metabolism and nutrition disorders | MedDRA 27.0 | Non-systematic Assessment |
|
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