PIDs Linked to PI3KPhase 2NCT06990529
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
A Phase 2 study testing Leniolisib in people with PIDs Linked to PI3K, run by Pharming Technologies B.V. at 5 sites in 3 countries.
Trial summary
This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE 7201. The primary objective is to assess long-term safety and tolerability of leniolisib. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- PIDs Linked to PI3K
- 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
- 12 participants
- Where
- 5 sitesin 3 countries
- Study dates
- 29 Apr 2025to 12 Aug 2029
- Phase
- Phase 2
- 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 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject must have participated in LE 7201.
- Subject is deemed by the Investigator to benefit from continued leniolisib therapy.
- Subject or their legal representatives (for a patient under the age of 18 years) must be able to communicate with the Investigator and understand and comply with the requirements of the study, including an ability to provide written informed consent before any assessment is performed.
Who cannot join
- Subject has had a successful allogeneic hematopoietic stem cell transplant.
- Previous or concurrent use of immunosuppressive medication, such as:
- Use of an mTOR inhibitor or a PI3K delta inhibitor, besides leniolisib, within 3 weeks prior to first dosing of study medication.
- Rituximab or other B-cell depleting antibodies, belimumab, cyclophosphamide, or alemtuzumab within 6 months prior to first dosing of study medication.
- Cyclosporine A, mycophenolate mofetil, 6-mercaptopurine, azathioprine, methotrexate, tacrolimus, ruxolitinib or other Janus kinase (JAK) inhibitors within 3 weeks prior to first dosing of study medication.
- Corticosteroids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dosing of study medication.
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- Other immunosuppressive agents expected to have a significant impact on immune cell number or function.
- Subject is receiving concurrent treatment with another investigational therapy or use of another investigational therapy less than 4 weeks or 5 half lives (whichever is longer) prior to first dosing of study medication.
- History of hypersensitivity to the study drug or to drugs of similar chemical classes.
- Current use of medication known to be a strong inhibitor or moderate or strong inducer, of isoenzyme cytochrome P450 (CYP)3A.
- Current use of medications that to a larger extent are breast cancer resistant protein (BCRP), organic anion transporting polypeptide (OATP)1B1, and/or OATP1B3 substrates.
- History of acquired immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result at screening.
- Uncontrolled chronic or recurrent infectious disease (except those considered to be characteristic of PID), or evidence of tuberculosis (TB) infection as defined by a positive QuantiFERON TB-Gold test at Screening.
- Any surgical or medical condition which may jeopardize the subject in case of participation in the study, or might significantly alter the absorption, distribution, metabolism, or excretion of drugs (conditions due to underlying clinical PID phenotype may be permitted):
- Uncontrolled hypertension
- Congestive heart failure (New York Heart Association status of class III or IV)
- Diagnosis of electrocardiogram (ECG) abnormalities indicating a significant risk of safety
- Chronic obstructive pulmonary disease (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage 3-4)
- Chronic need for supplemental oxygen or invasive or non-invasive respiratory support
- Major GI tract surgery that may affect drug absorption (such as gastric bypass surgery, gastroenterostomy)
- Acute pancreatitis
- Liver failure or clinically significant liver disease or dysfunction as indicated by ALT or AST greater than 2.5 times the upper limit of normal, bilirubin greater than 2 times the upper limit of normal, INR greater than 1.5 in the absence of anticoagulation, or presence of diuretic refractory ascites
- History of significant renal injury/renal disease severely affecting renal function or presence of impaired renal function as indicated by estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73 m2.
- A positive hepatitis B surface antigen (HBsAg), positive hepatitis B polymerase chain reaction (PCR), positive hepatitis C PCR, or positive hepatitis C antibody result at screening.
- Administration of live vaccines (this includes any attenuated live vaccines) starting from 6 weeks prior to first dosing of study medication, during the study, and up to 7 days after the last dose of leniolisib.
- Subject has a previous diagnosis of lymphoma that has been treated with chemotherapy, radiotherapy, or transplant within 1 year prior to first dosing of study medication or is anticipated to require lymphoma treatment within 6 months of the first dose of study medication.
- Subject has a history of malignancy (except lymphoma) within 3 years prior to first dosing of study medication or has evidence of residual disease from a previously diagnosed malignancy, except for adequately treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the uterine cervix.
- Subject has uncontrolled post-transplant lymphoproliferative disease-like Epstein-Barr-virus-related lymphoproliferative disease.
- Subject has had major surgery requiring hospitalization or radiotherapy within 4 weeks prior to first dosing of study medication or has a planned or expected major surgical procedure during the study period.
- Pregnant or nursing (lactating) women.
- An individual of child-bearing potential who is physiologically capable of becoming pregnant, unless using highly effective methods of contraception.
Trial locations and contacts
5 sites in 3 countries.
5 sites in 3 countries
United States3 sites
National Institute of Health
Bethesda, Maryland, 20892
Lahey Hospital & Medical Center
Burlington, Massachusetts, 01805
Mount Sinai Hospital
New York, New York, 10029
About this study
Official title: An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders
This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE 7201. The primary objective is to assess long-term safety and tolerability of leniolisib. Secondary and exploratory objectives include various efficacy and immunophenotyping measures for leniolisib.
- Conditions
- PIDs Linked to PI3K
- Being tested
- Leniolisib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeniolisibDrug
All subjects will receive leniolisib film-coated tablets (FCTs) at the same dose they were receiving when they completed the preceding study (10, 30, or 70 mg twice daily [BID]).
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | All subjects will receive leniolisib film-coated tablets (FCTs) Receives: Drug: Leniolisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the long-term safety and tolerability of leniolisib
Adverse events (AEs)
Measured: From Baseline to approximately 3 years of Treatment
Secondary outcomes (21)
Impact of leniolisib on hemoglobin
Hemoglobin over time
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on platelets
Platelet count over time
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on neutrophils
Absolute neutrophil count (ANC) over time
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on GLILD or other PID-related ILD
Computed tomography (CT) evidence of granulomatous lymphocytic interstitial lung disease (ILD) or other PID-related ILD evaluated using Hartmann scoring methodology over time
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Change in FEV1 will be evaluated
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Change in FVC will be evaluated
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Change in TLC will be evaluated
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Change in DLCO will be evaluated)
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on lymphoproliferation measured as index lesions
Percent change of lymphoproliferation over time measured as the sum of product of diameters (SPD) in the index lesions selected at baseline of the preceding study per the Cheson methodology
Measured: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on spleen size
Spleen size over time measured by three-dimensional (3D) volume and two dimensional (2D) size of spleen
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
WBC count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Absolute monocyte count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Absolute eosinophil count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Absolute basophil count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
Absolute lymphocyte count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
CD4+ T cell count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
CD8+ T cell count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the long-term impact of leniolisib on white blood cell (WBC) counts and lymphocyte numbers
B cell count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
Natural killer (NK) cell count over time
Measured: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on B and T cell phenotypic populations
Percentages of naïve B cells, CD21low B cells and T regulatory cells over time
Measured: From Baseline to approximately 3 years of Treatment
To examine the impact of leniolisib on levels of chemokine (C X C motif) ligand (CXCL)13 and soluble interleukin-2 receptor (IL-2R)α
Levels of CXCL13 and soluble IL-2Rα over time
Measured: From Baseline to approximately 3 years of Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2025
- Study start29 Apr 2025
- Primary completion12 Aug 2029 (estimated)
- Study completion12 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PIDs Linked to PI3KStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aixial Group
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06990529
- Sponsor's protocol ID
- LE7X01
- Status last verified
- Sep 2026
- First submitted
- 8 May 2025
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders
- ClinicalTrials.gov ID
- NCT06990529
- Acronym / sponsor ID
- LE7X01
- Conditions
- PIDs Linked to PI3K
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Collaborators
- Aixial Group
- Study start
- 29 Apr 2025 (actual)
- Primary completion
- 12 Aug 2029 (estimated)
- Study completion
- 12 Aug 2029 (estimated)
- First posted
- 25 May 2025 (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 an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE 7201. The primary objective is to assess long-term safety and tolerability of leniolisib. Secondary and exploratory objectives include various efficacy and immunophenotyping measures for leniolisib.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Drug: LeniolisibAll subjects will receive leniolisib film-coated tablets (FCTs) |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Subject must have participated in LE 7201.
- Subject is deemed by the Investigator to benefit from continued leniolisib therapy.
- Subject or their legal representatives (for a patient under the age of 18 years) must be able to communicate with the Investigator and understand and comply with the requirements of the study, including an ability to provide written informed consent before any assessment is performed.
Key exclusion criteria (31 listed)
- Subject has had a successful allogeneic hematopoietic stem cell transplant.
- Previous or concurrent use of immunosuppressive medication, such as:
- Subject is receiving concurrent treatment with another investigational therapy or use of another investigational therapy less than 4 weeks or 5 half lives (whichever is longer) prior to first dosing of study medication.
- History of hypersensitivity to the study drug or to drugs of similar chemical classes.
- Current use of medication known to be a strong inhibitor or moderate or strong inducer, of isoenzyme cytochrome P450 (CYP)3A.
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 |
|---|---|---|
| LeniolisibAll subjects will receive leniolisib film-coated tablets (FCTs) at the same dose they were receiving when they completed the preceding study (10, 30, or 70 mg twice daily [BID]). | Drug | Treatment Arm |
Primary outcome
To assess the long-term safety and tolerability of leniolisib
Time frame: From Baseline to approximately 3 years of Treatment
Adverse events (AEs)
21 secondary outcomes
Impact of leniolisib on hemoglobin
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on platelets
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on neutrophils
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on GLILD or other PID-related ILD
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on pulmonary function
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on lymphoproliferation measured as index lesions
Time frame: From Baseline to approximately 3 years of Treatment
Impact of leniolisib on spleen size
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on white blood cell (WBC) counts
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
Time frame: From Baseline to approximately 3 years of Treatment
To assess the long-term impact of leniolisib on white blood cell (WBC) counts and lymphocyte numbers
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on lymphocyte numbers
Time frame: From Baseline to approximately 3 years of Treatment
To assess the impact of leniolisib on B and T cell phenotypic populations
Time frame: From Baseline to approximately 3 years of Treatment
To examine the impact of leniolisib on levels of chemokine (C X C motif) ligand (CXCL)13 and soluble interleukin-2 receptor (IL-2R)α
Time frame: From Baseline to approximately 3 years of Treatment
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 16 Sep 2026.
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.
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Leniolisib in people with PIDs Linked to PI3K, run by Pharming Technologies B.V. at 5 sites in 3 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 3 inclusion and 31 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 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
To assess the long-term safety and tolerability of leniolisib (time frame: From Baseline to approximately 3 years of Treatment). 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 16 Sep 2026.