APDSPhase 3NCT05693129
Pediatric Patients Aged 1 to 6 Years With APDS
A Phase 3 study testing Leniolisib in people with APDS, run by Pharming Technologies B.V. at 9 sites in 5 countries.
Trial summary
This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) Read more about this study
- Who is it for?
- Ages 1 year to 6 years
- Conditions the study is for
- APDS
- What is being tested?
- Leniolisib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 30 Aug 2023to 28 Oct 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 1 year to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patient is male or female and between the age of 1 to 6 years old at time of the first study procedure.
- Patient weighs ≥8 and ≤37 kg at baseline.
- Patient has a confirmed PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
- Patient has at least 1 measurable nodal lesion on MRI or low-dose CT within 6 months of screening.
- Patient has nodal or extranodal lymphoproliferation and clinical findings consistent with APDS (eg, a history of repeated oto-sino-pulmonary infections or organ dysfunction consistent with APDS).
- Patient has the ability to ingest unaltered study-related medications without difficulty in the investigator's opinion.
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- At screening, vital signs (body temperature, systolic BP, diastolic BP, and pulse rate [PR]) will be assessed in the sitting position after the patient has been at rest for at least 3 minutes. Patient's sitting vital signs should be within the following ranges:
- Systolic BP: Less than the 95th percentile adjusted for sex, age, and height percentile. See Section 10.5, Appendix 5 to determine BP percentiles adjusted for sex, age, and height percentile. (National High Blood Pressure Education Program Working Group on High Blood Pressure in Children and Adolescents, 2004). See Section 10.5, Appendix 5 to determine height percentiles.
- Diastolic BP: Less than the 95th percentile adjusted for sex, age, and height percentile. See Section 10.5, Appendix 5 to determine BP percentiles adjusted for sex, age, and height percentile. See Section 10.6, Appendix 6 to determine height percentiles.
- Pulse rate (Fleming 2011):
- Age <2 years: 100 to 190 bpm ii. Age 2 to 6 years: 60 to 140 bpm
- Institutional review board- or IEC-approved written informed consent or assent and privacy language as per national and local regulations must be obtained from the patient and/or parent or legal guardian prior to any study-related procedures.
- Patient parent or legal guardian is willing and able to complete the informed consent or assent process and comply with study procedures and visit schedule.
- Patient parent or legal guardian agrees patient will not participate in any other interventional study while enrolled in this study.
Who cannot join
- Patient has previous or concurrent use of immunosuppressive medication such as:
- an mTOR inhibitor (eg, sirolimus, rapamycin, everolimus) or a PI3Kδ inhibitor (selective or non-selective PI3K inhibitors) within 6 weeks prior to first dose.
- o Short-term use for up to a total of 5 days is allowed but only up to 1 month prior to enrollment in the study.
- B cell depleters (eg, rituximab) within 6 months prior to first dose of study medication.
- o If patient has received prior treatment with a B cell depleter, absolute B lymphocyte counts in the blood must have regained normal values.
- Belimumab or cyclophosphamide within 6 months prior to first dose of study medication.
Show all 36Show fewer
- Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study medication.
- Glucocorticoids above a dose equivalent to either ≥2 mg/kg of body weight for weights less than 10 kg or ≥20 mg/day for weights ≥ 10 kg of prednisone or prednisolone or equivalent within 2 weeks prior to first dose of study medication.
- Other immunosuppressive medication where effects are expected to persist at start of dosing of study medication.
- Patient has a history or current diagnosis of ECG abnormalities indicating significant risk of safety for patients participating in the study such as:
- History of familial long QT syndrome or known family history of Torsades de Pointes.
- Concomitant clinically significant cardiac arrhythmias, eg, sustained ventricular tachycardia, and clinically significant second or third degree atrioventricular block without a pacemaker.
- Resting QTc (Fridericia preferred, but Bazett acceptable) >460 msec if the measurement is confirmed with an additional ECG repeated as soon as possible.
- Concomitant use of agents known to prolong the QT interval unless it can be permanently discontinued for the duration of the study.
- Patient is currently using a medication known to be strong inhibitor or moderate or strong inducer of isoenzyme CYP3A (see Table 2), if treatment cannot be discontinued or switched to a different medication prior to starting study treatment.
- Patient is currently using medications that are metabolized by isoenzyme CYP1A2 and have a narrow therapeutic index (NTI) (drugs whose exposure response indicates that increases in their exposure levels by the concomitant use of potent inhibitors may lead to serious safety concerns [eg, Torsades de Pointes]).
- Patient had been administered live vaccines (this includes any attenuated live vaccines) starting from 6 weeks before the anticipated first study drug administration, during the study, and up to 7 days after the last dose of leniolisib.
- Patient has clinically significant abnormalities in hematology or clinical chemistry (blood chemistry or urinalysis) parameters as determined by the investigator or medical monitor.
- Patient has liver disease or liver injury as indicated by clinically significant abnormal liver function tests (LFTs) (alanine aminotransferase and aspartate aminotransferase >2.5 times upper limit of normal), history of renal injury or renal disease (eg, renal trauma, glomerulonephritis, or one kidney only), or presence of impaired renal function as indicated by a serum creatinine level >1.5 mg/dL (133 μmol/L).
- Patient has moderate or severe hepatic impairment (Child-Pugh Class B or C).
- Patient is receiving concurrent treatment with another investigational therapy or use of another investigational therapy less than 4 weeks from the first study procedure.
- Patient has active hepatitis B (eg, hepatitis B surface antigen reactive) or active hepatitis C (eg, hepatitis C virus RNA [qualitative] is detected) at screening.
- Patient has human immunodeficiency virus (HIV) infection (HIV 1 or 2) at screening.
- Patient has a positive COVID-19 result (polymerase chain reaction or antigen) within 1 week prior to first dose. The patient can be rescreened after a subsequent negative result.
- Patient has a history of malignancy (except lymphoma) within 3 years before the first study procedure or has evidence of residual disease from a previously diagnosed malignancy.
- Patient has a previous diagnosis of lymphoma that has been treated with chemotherapy, radiotherapy, or transplant within 1 year of the first study procedure or is anticipated to require lymphoma treatment within 6 months of the first study procedure.
- Patient has a history of uncontrolled diabetes mellitus within 3 months of the first study procedure.
- Patient has had major surgery requiring hospitalization or radiotherapy within 4 weeks prior to the first study procedure.
- Patient has uncontrolled chronic or recurrent infectious disease (with the exception of those that are considered to be characteristic of APDS) or evidence of tuberculosis infection as defined by a positive QuantiFERON Gold test at screening. If presence of latent tuberculosis is established, then treatment according to local country guidelines must have been completed before patients can be considered for enrollment.
- Patient has a known allergy or hypersensitivity to study defined medications or their excipients.
- Patient has a planned or expected major surgical procedure.
- Patient or parent or legal guardian is unable or unwilling to comply with study procedures or is unable to travel for repeat visits.
- Patient or parent or legal guardian is unwilling to keep study results or observations confidential or to refrain from posting confidential study results or observations on social media sites.
- Patient or parent or legal guardian refuses to sign consent or assent form.
- Patient has other underlying medical condition that, in the opinion of the investigator, would impair the ability of the patient to receive or tolerate the planned procedures or follow-up.
- -
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
United States4 sites
National Institutes of Health
Bethesda, Maryland, 20814
Texas Children's Hospital
Houston, Texas, 77030
University of California Los Angeles
Los Angeles, California, 90095
Rainbow Childrens Hospital
Shaker Heights, Ohio, 44122
Japan2 sites
Kyoto University Hospital
Kyoto, 606-8507
Institute of Science Tokyo Hospital
Tokyo, 113-8519
Portugal1 site
Hospital Pediátrico de Coimbra da ULS Coimbra UNIDADE LOCAL DE SAÚDE DE COIMBRA
Coimbra, 3000-075
About this study
Official title: An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension
This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Read the detailed description
Part I will consist of a 12-week period to assess the safety and efficacy of treatment with leniolisib. Part II will consist of a 1-year, long-term, safety follow-up extension with a possible interim analysis.
The leniolisib doses to be used in study were selected based on safety, tolerability, PK, and PDx data from the adult Phase 2/3 study, as well as PK modeling data. In both parts of the study, leniolisib will be administered orally based on weight.
- Conditions
- APDS
- Being tested
- Leniolisib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeniolisibDrug
The doses selected will range from 10 to 50 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 100 mg per day).
| Study group | Role | What they receive |
|---|---|---|
| Leniolisib | Experimental | Leniolisib film-coated granules in 10, 15 and 20 mg strengths administered orally BID by body weight for 12 weeks for Part I and for 1 year for Part II. Receives: Drug: Leniolisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part I & II: Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)
To assess number of Participants with TEAEs, SAEs, and AEs leading to discontinuation of study drug
Measured: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days
Part I & II: Change from baseline in clinical laboratory test results
Number of Participants with change in clinical laboratory test results (hematology, blood
Measured: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Part I & II: Change from baseline in vital signs
Number of Participants with change in vital signs
Measured: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Part I & II: Change from baseline in physical examination findings
Number of Participants with change in physical examination findings
Measured: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Part I & II: Change from baseline in electrocardiograms (ECGs)
Number of Participants with change in electrocardiograms (ECGs)
Measured: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days
Part I & II: Change from baseline in growth and physical development
Number of Participants with change in growth and physical development
Measured: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Part I & II: Reduction in lymphadenopathy as measured by MRI or low-dose CT
To assess the impact of leniolisib on lymphadenopathy, patients will be scanned in an MRI or a CT scanner as based on clinical practice and local regulation. Index lesions will be selected from measurable nodal and extra nodal lesions as per the Cheson methodology. The same imaging modality will be used throughout the study for the same patient. Patients will be assessed by MRI, or in sites where local practice and local authorities/IECs/IRBs approve CT scans for research purposes using a low-dose CT scan.
Measured: Part I: Baseline and Day 85 Part II: at Day 252, through study completion, an average of 1 year
Part I: A Percentage of Inhibition of Unstimulated and Stimulated pAkt Levels in B Cells
To assess change in the PDx effect of leniolisib will be assessed using ex vivo stimulated and unstimulated
Measured: Part I: Baseline, Days 29, 57 and 85
Secondary outcomes (8)
Part I: To assess the total drug exposure (AUC) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
Population pharmacokinetics (popPK) model that describes the appropriate covariates (eg, body weight and age) that influence leniolisib PK in pediatric patients.
Measured: From baseline to end of 12 weeks of treatment
Part I: To assess the maximum concentration (Cmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
Population pharmacokinetics (popPK) model that describes the appropriate covariates (eg, body weight and age) that influence leniolisib PK in pediatric patients.
Measured: From baseline to end of 12 weeks of treatment
Part I: To assess the time to maximum concentration (Tmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
Population pharmacokinetics (popPK) model that describes the appropriate covariates (eg, body weight and age) that influence leniolisib PK in pediatric patients.
Measured: From baseline to end of 12 weeks of treatment
Part II: Changes from baseline for Reduction in lymphadenopathy as measured by MRI or low-dose CT
Parameters for reduction in lymphadenopathy as a key secondary endpoint of Part II may include 3D volume of index and measurable non-index lesions selected as per the Cheson methodology. Summary statistics will be provided by visit. Arithmetic mean with SD of absolute values and change from baseline values may be plotted across time.
Measured: Day 85 to through study completion, an average of 1 year
Part II: Changes from baseline for Reduction in lymphadenopathy as measured by MRI or low-dose CT
Parameters for reduction in 3D volume and bi-dimensional or 3D sizes of spleen, where appropriate. Summary statistics will be provided by visit. Arithmetic mean with SD of absolute values and change from baseline values may be plotted across time.
Measured: Day 85 to through study completion, an average of 1 year
Part II: Changes from baseline for Reduction in lymphadenopathy as measured by MRI or low-dose CT
Parameters for reduction in 3D volume and bi-dimensional or 3D sizes of liver, where appropriate. Summary statistics will be provided by visit. Arithmetic mean with SD of absolute values and change from baseline values may be plotted across time.
Measured: Day 85 to through study completion, an average of 1 year
Part I and II: Key secondary efficacy outcomes for Part I include incidence of infections and use of antibiotics.
The number and percentage of patients with infections, and the total number of infections will be summarized. The number and percentage of antibiotics taken will be presented along with number of patients for Part I. Use of immunoglobulin replacement therapy over time will be summarized.
Measured: Part I: Baseline to Day 85 Part II: through study completion, an average of 1 year
Part I and II: Pediatric Quality of Life Inventory (PedsQLTM) Parent Report for Children Questionnaire 4.0 Generic Core Scales
To assess the ability of leniolisib to modify health related quality of life in pediatric patients with APDS
Measured: Part I: Baseline, Day 29, 57, & 85 Part II: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2023
- Study start30 Aug 2023
- Primary completion9 Jul 2028 (estimated)
- Study completion28 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for APDSStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- CMIC Co, Ltd. Japan, Labcorp Central Laboratory, Fortrea, Aixial Group
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05693129
- Sponsor's protocol ID
- LE 3302
- Status last verified
- Jun 2026
- First submitted
- 17 Oct 2022
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension
- ClinicalTrials.gov ID
- NCT05693129
- Acronym / sponsor ID
- LE 3302
- Conditions
- APDS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Collaborators
- CMIC Co, Ltd. Japan, Labcorp Central Laboratory, Fortrea, Aixial Group
- Study start
- 30 Aug 2023 (actual)
- Primary completion
- 9 Jul 2028 (estimated)
- Study completion
- 28 Oct 2028 (estimated)
- First posted
- 20 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Detailed description
Part I will consist of a 12-week period to assess the safety and efficacy of treatment with leniolisib. Part II will consist of a 1-year, long-term, safety follow-up extension with a possible interim analysis.
The leniolisib doses to be used in study were selected based on safety, tolerability, PK, and PDx data from the adult Phase 2/3 study, as well as PK modeling data. In both parts of the study, leniolisib will be administered orally based on weight.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Leniolisib | Experimental | Drug: LeniolisibLeniolisib film-coated granules in 10, 15 and 20 mg strengths administered orally BID by body weight for 12 weeks for Part I and for 1 year for Part II. |
Who may qualify
- Ages
- 1 year to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- Patient is male or female and between the age of 1 to 6 years old at time of the first study procedure.
- Patient weighs ≥8 and ≤37 kg at baseline.
- Patient has a confirmed PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
- Patient has at least 1 measurable nodal lesion on MRI or low-dose CT within 6 months of screening.
- Patient has nodal or extranodal lymphoproliferation and clinical findings consistent with APDS (eg, a history of repeated oto-sino-pulmonary infections or organ dysfunction consistent with APDS).
Key exclusion criteria (36 listed)
- Patient has previous or concurrent use of immunosuppressive medication such as:
- o Short-term use for up to a total of 5 days is allowed but only up to 1 month prior to enrollment in the study.
- o If patient has received prior treatment with a B cell depleter, absolute B lymphocyte counts in the blood must have regained normal values.
- Patient has a history or current diagnosis of ECG abnormalities indicating significant risk of safety for patients participating in the study such as:
- Patient is currently using a medication known to be strong inhibitor or moderate or strong inducer of isoenzyme CYP3A (see Table 2), if treatment cannot be discontinued or switched to a different medication prior to starting study treatment.
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 |
|---|---|---|
| LeniolisibThe doses selected will range from 10 to 50 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 100 mg per day). | Drug | Leniolisib |
Primary outcome
Part I & II: Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)
Time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days
To assess number of Participants with TEAEs, SAEs, and AEs leading to discontinuation of study drug
Part I & II: Change from baseline in clinical laboratory test results
Time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Number of Participants with change in clinical laboratory test results (hematology, blood
Part I & II: Change from baseline in vital signs
Time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Number of Participants with change in vital signs
Part I & II: Change from baseline in physical examination findings
Time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Number of Participants with change in physical examination findings
Part I & II: Change from baseline in electrocardiograms (ECGs)
Time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days
Number of Participants with change in electrocardiograms (ECGs)
Part I & II: Change from baseline in growth and physical development
Time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
Number of Participants with change in growth and physical development
Part I & II: Reduction in lymphadenopathy as measured by MRI or low-dose CT
Time frame: Part I: Baseline and Day 85 Part II: at Day 252, through study completion, an average of 1 year
To assess the impact of leniolisib on lymphadenopathy, patients will be scanned in an MRI or a CT scanner as based on clinical practice and local regulation. Index lesions will be selected from measurable nodal and extra nodal lesions as per the Cheson methodology. The same imaging modality will be used throughout the study for the same patient. Patients will be assessed by MRI, or in sites where local practice and local authorities/IECs/IRBs approve CT scans for research purposes using a low-dose CT scan.
Part I: A Percentage of Inhibition of Unstimulated and Stimulated pAkt Levels in B Cells
Time frame: Part I: Baseline, Days 29, 57 and 85
To assess change in the PDx effect of leniolisib will be assessed using ex vivo stimulated and unstimulated
8 secondary outcomes
Part I: To assess the total drug exposure (AUC) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
Time frame: From baseline to end of 12 weeks of treatment
Part I: To assess the maximum concentration (Cmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
Time frame: From baseline to end of 12 weeks of treatment
Part I: To assess the time to maximum concentration (Tmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS
Time frame: From baseline to end of 12 weeks of treatment
Part II: Changes from baseline for Reduction in lymphadenopathy as measured by MRI or low-dose CT
Time frame: Day 85 to through study completion, an average of 1 year
Part II: Changes from baseline for Reduction in lymphadenopathy as measured by MRI or low-dose CT
Time frame: Day 85 to through study completion, an average of 1 year
Part II: Changes from baseline for Reduction in lymphadenopathy as measured by MRI or low-dose CT
Time frame: Day 85 to through study completion, an average of 1 year
Part I and II: Key secondary efficacy outcomes for Part I include incidence of infections and use of antibiotics.
Time frame: Part I: Baseline to Day 85 Part II: through study completion, an average of 1 year
Part I and II: Pediatric Quality of Life Inventory (PedsQLTM) Parent Report for Children Questionnaire 4.0 Generic Core Scales
Time frame: Part I: Baseline, Day 29, 57, & 85 Part II: through study completion, an average of 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
- Individual participant dataSharing planned: No
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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.
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Leniolisib in people with APDS, run by Pharming Technologies B.V. at 9 sites in 5 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 14 inclusion and 36 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: under way, not looking for participants, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Japan, Portugal, Spain, United Kingdom. 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
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?
Part I & II: Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs) (time frame: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Jun 2026.