APDS Gene MutationPhase 3NCT06249997
An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS
A Phase 3 study testing Leniolisib in people with APDS Gene Mutation, run by Pharming Technologies B.V. at 2 sites in Japan.
Trial summary
An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By an Open-Label Long-Term Extension. For the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS). Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- APDS Gene Mutation
- 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
- 3 participants
- Where
- 2 sitesJapan
- Study dates
- 3 Aug 2023to 31 Mar 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Patient is Japanese.
- Patient is male or female and 12 to 75 years of age (inclusive) at the time of the first study procedure.
- Patient weighs ≥35 kg at baseline.
- Patient has a PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
- Patient has at least 1 measurable nodal lesion on computed tomography (CT) or magnetic resonance imaging (MRI) scan within 6 months of Screening.
- Patient has nodal and/or extranodal lymphoproliferation and clinical findings and manifestations consistent with APDS (e.g., a history of repeated oto-sino-pulmonary infections and/or organ dysfunction consistent with APDS).
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- At Screening, patient has sitting vital signs (with patient rested for at least 3 minutes) within the following ranges:
- Systolic blood pressure, 90-160 mm Hg
- Diastolic blood pressure, 50-95 mm Hg
- Pulse rate, 40-100 bpm; up to 110 bpm in adolescents
Who cannot join
- Patient has previous or concurrent use of immunosuppressive medication such as the following:
- A mammalian target of rapamycin inhibitor (e.g., sirolimus, rapamycin, or everolimus) or a PI3Kδ inhibitor (selective or non-selective phosphoinositide 3-kinase inhibitors) within 6 weeks prior to first dose.
- 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 (e.g., rituximab) within 6 months prior to first dose of study treatment.
- 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 treatment.
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- Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study treatment.
- Glucocorticoids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dose of study treatment.
- Other immunosuppressive medications where effects are expected to persist at start of dosing of study treatment.
- Patient has had a hematopoietic stem-cell transplant, hematopoietic cell transplant, or bone marrow transplant.
- Patient is currently using a medication known to be a strong inhibitor or moderate or strong inducer of isoenzyme cytochrome P450 (CYP)3A 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 (drugs whose exposure response indicates that increases in their exposure levels by the concomitant use of potent inhibitors may lead to serious safety concerns [e.g., Torsades de Pointes]).
- Patient had been administered a live vaccine (this includes any attenuated live vaccines) starting from 6 weeks before the anticipated first dose of study treatment, during the treatment period, and up to 7 days after the last dose of leniolisib.
- Patient is a pregnant or nursing (lactating) woman, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotropin laboratory test.
- Patient is a woman of child-bearing potential, defined as a woman physiologically capable of becoming pregnant, unless she is using highly effective methods of contraception during dosing of study treatment and for 30 days after the last study procedure.
Trial locations and contacts
2 sites in 1 country.
Japan2 sites, 2 recruiting
- Recruiting
Hiroshima University Hospital
Hiroshima, Hiroshima City, 734-8551
Satoshi Okada, MD, sokada@hiroshima-u.ac.jp
Noma Kosuke, MD
- Recruiting
Tokyo Medical And Dental University Hospital
Tokyo, Bunkyo-ku, 113-8510
Hirokazu Kanegane, Prof., hkanegane.ped@tmd.ac.jp
Takeshi Isoda, MD
About this study
Official title: An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By An Open-Label Long-Term Extension
An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By an Open-Label Long-Term Extension.
For the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
Read the detailed description
This is a 2-part, open-label, non-randomized study to assess the safety and efficacy of leniolisib in Japanese patients with APDS. At least3 patients, aged 12 to 75 years (inclusive), will be enrolled. Patient eligibility will be assessed during a 7-week Screening Period (Day -50 to Day -1). This will be followed by a 12-week Treatment Period (Part 1), in which patients will be administered leniolisib doses ranging from 40 to 70 mg twice daily (BID) based on body weight (see dose regimen table below). A Part 1 clinical study report will be generated once the last patient completes the Day 85 Visit for Part 1. Patients who complete the Day 85 Visit will enter the Extension Period of the study (Part 2), in which patients will be administered leniolisib doses ranging from 40 to 70 mg BID (based on body weight) for 1 year or until marketing approval in Japan, whichever is longer. A 4-week Follow-up Period will occur after the last dose of study treatment is received.
It is anticipated that a total of 3 patients will be enrolled into the study.
Objectives:
Part 1:
Primary:
- To assess the safety and tolerability of leniolisib
- To assess the efficacy of leniolisib on lymphoproliferation (sum of product diameters [SPD] of index lymph node lesions) and immunophenotype normalization (percentage of naïve B cells out of total B cells)
Secondary:
- To assess the efficacy of leniolisib on lymphoproliferation (non-index lymph node lesions and spleen)
- To assess the pharmacokinetics (PK) of leniolisib in the Japanese population
- To assess the efficacy of leniolisib to modify health-related quality of life
- To assess the efficacy of leniolisib by the Patient's and Physician's Global Assessments
- To assess the frequency of infections, antibiotic use, and immunoglobulin (Ig) replacement therapy and assessment of impact on other disease-related outcomes (e.g., cytopenia, colitis, and lung function)
- To assess biomarkers reflecting the efficacy of leniolisib to reduce systemic inflammatory components of the disease
- To assess the treatment benefit to individual patients
Part 2:
Primary:
- To assess the long-term safety and tolerability of leniolisib
Secondary:
- To assess the long-term efficacy of leniolisib to modify health-related quality of life
- To assess the long-term efficacy of leniolisib on lymphoproliferation (non-index lymph node lesions and spleen)
- Conditions
- APDS Gene Mutation
- 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 range from 40 to 70 mg BID (based on body weight, resulting in total daily doses ranging from 80 to 140 mg a day for 12 weeks in Part I and 1 year in Part II, or until marketing approval in Japan, whichever is longer.
| Study group | Role | What they receive |
|---|---|---|
| Leniolisib | Experimental | Leniolisib - Film coated tablets
Leniolisib tablets in doses ranging from 40 to 70 mg twice daily (BID) based on body weight.
A Part 1 clinical study report will be generated once the last patient completes the Day 85 Visit for Part 1. Patients who complete the Day 85 Visit will enter the Extension Period of the study (Part 2), in which patients will be administered leniolisib doses ranging from 40 to 70 mg BID (based on body weight) for 1 year or until marketing approval in Japan, whichever is longer. A 4-week Follow-up Period will occur after the last dose of study treatment is received. Receives: Drug: Leniolisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part I: Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs
Incidence of treatment-emergent adverse events (AEs), serious adverse events (SAEs), and AEs leading to discontinuation of study treatment
Measured: Between baseline until Day 85
Part II: Long-term Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs
Long-term Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs leading to discontinuation of study treatment
Measured: At Day 252, through study completion, an average of 1 year
Part I: Change from baseline in clinical laboratory test results
Number of participants with change in clinical laboratory test results (hematology, blood chemistry, urinalysis). Absolute values and change from baseline values of vital signs (including body weight) and ECG results at each visit will be listed for each patient and may be summarized using descriptive statistics. Tanner staging will be listed for each patient at each visit. Plots may also be used to display the patient data over time.
Measured: Between baseline until Day 85
Part II: Long-term change from baseline in clinical laboratory test results
Number of participants with long-term change in clinical laboratory test results (hematology, blood chemistry, urinalysis). Absolute values and change from baseline values of vital signs (including body weight) and ECG results at each visit will be listed for each patient and may be summarized using descriptive statistics. Tanner staging will be listed for each patient at each visit. Plots may also be used to display the patient data over time.
Measured: At Day 252, through study completion, an average of 1 year
Part I: Change from baseline in vital signs
Number of Participants with change in vital signs
Measured: Between baseline until Day 85
Part II: Long-term change from baseline in vital signs
Number of Participants with long-term change in vital signs
Measured: At Day 252, through study completion, an average of 1 year
Part I: Change from baseline in physical examination findings
Number of participants with change in physical examination findings
Measured: Between baseline until Day 85
Part II: Long-term change from baseline in physical examination findings
Number of participants with long-term change in physical examination findings
Measured: At Day 252, through study completion, an average of 1 year
Part I: Change from baseline in electrocardiograms (ECGs)
Number of participants with change in electrocardiograms (ECGs)
Measured: Between baseline until Day 85
Part II: Long-term change from baseline in electrocardiograms (ECGs)
Number of participants with long-term change in electrocardiograms (ECGs)
Measured: At Day 252, through study completion, an average of 1 year
Part I: To assess the efficacy of leniolisib on lymphoproliferation (SPD of index lymph node lesions) and immunophenotype normalization (percentage of naïve B cells out of total B cells)
Number of patients with change in SPD of index lesions (selected as per the Cheson methodology from magnetic resonance imaging [MRI] or computed tomography [CT] imaging) at the end of treatment
Measured: Between baseline until Day 85
Part I: Change from baseline in the percentage of naïve B cells out of total B cells at the end of treatment
Number of participants with change in percentage of naïve B cells out of total B cells at the end of treatment
Measured: Between baseline until Day 85
Secondary outcomes (9)
Part I: Change from baseline in lymphoproliferation measured using MRI, CT imaging, or ultrasound
Parameters for change from baseline in lymphoproliferation as a secondary endpoint may include 3-dimensional (3D) volume of index and measurable non-index lesions (selected as per the Cheson methodology) and 3D volume and bi-dimensional sizes of spleen and liver, where appropriate.
Measured: Between baseline until Day 85
Part II: Long-term change from baseline in lymphoproliferation measured using MRI, CT imaging, or ultrasound
Number of participants with change from in lymphoproliferation measured using MRI, CT imaging, or ultrasound (e.g., 3D volume of index and measurable non-index lesions [selected as per the Cheson methodology] and 3D volume and bi-dimensional sizes of spleen and liver, where appropriate) at the end of treatment.
Measured: At Day 252, through study completion, an average of 1 year
Part I: To assess the PK of leniolisib in the Japanese population
Number of participants with change in PK parameters (including but not limited to area under the plasma concentration-time curve from time zero to 12 hours after dosing at steady state [AUC0-12,ss] and maximum plasma concentration following drug administration at steady state [Cmax,ss])
Measured: Between baseline until Day 85
Part I: To assess the efficacy of leniolisib to modify health-related quality of life
Number of participants with change in Short Form-36 (SF-36) Survey and Work Productivity and Activity Impairment-Classroom Impairment Questionnaire (WPAI-CIQ) summary scores
Measured: Between baseline until Day 85
Part II: To assess the long-term efficacy of leniolisib to modify health-related quality of life
Number of participants with long-term change in Short Form-36 (SF-36) Survey and Work Productivity and Activity Impairment-Classroom Impairment Questionnaire (WPAI-CIQ) summary scores
Measured: At Day 252, through study completion, an average of 1 year
Part I: To assess the efficacy of leniolisib by the Patient's and Physician's Global Assessments
Number of participants with long-term change in visual analog scales (VAS) for Patient's and Physician's Global Assessments, measured by scale with no and maximal activity score
Measured: Between baseline until Day 85
Part I: To assess the frequency of infections and assessment of impact on other disease-related outcomes (e.g., cytopenia, colitis, and lung function)
Number of participants with change in frequency of infections, use of antibiotics, Ig replacement therapy, and other disease complications
Measured: Between baseline until Day 85
Part I: To assess biomarkers reflecting the efficacy of leniolisib to reduce systemic inflammatory components of the disease
Number of participants with change in High-sensitivity C-reactive protein (hsCRP), lactate dehydrogenase (LDH), beta2 microglobulin, ferritin, fibrinogen, and erythrocyte sedimentation rate
Measured: Between baseline until Day 85
Part I: To assess the treatment benefit to individual patients
Number of patients that benefit from the treatment via narratives by the Investigator
Measured: Between baseline until Day 85
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2024
- Study start3 Aug 2023
- Primary completion31 Jan 2025 (estimated)
- Study completion31 Mar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for APDS Gene MutationStudies 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
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Laboratory Corporation of America, Axial Biotech, Inc, CMIC Co, Ltd. Japan
- Responsible party
- Sponsor
- Investigators
- Hirokazu Kanegane, Prof., Principal investigator, Tokyo Medical And Dental University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06249997
- Sponsor's protocol ID
- LE 4301
- Status last verified
- Jan 2024
- First submitted
- 21 Nov 2023
- Last updated
- 8 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By An Open-Label Long-Term Extension
- ClinicalTrials.gov ID
- NCT06249997
- Acronym / sponsor ID
- LE 4301
- Conditions
- APDS Gene Mutation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Collaborators
- Laboratory Corporation of America, Axial Biotech, Inc, CMIC Co, Ltd. Japan
- Study start
- 3 Aug 2023 (actual)
- Primary completion
- 31 Jan 2025 (estimated)
- Study completion
- 31 Mar 2025 (estimated)
- First posted
- 8 Feb 2024 (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.
An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By an Open-Label Long-Term Extension.
For the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
Detailed description
This is a 2-part, open-label, non-randomized study to assess the safety and efficacy of leniolisib in Japanese patients with APDS. At least3 patients, aged 12 to 75 years (inclusive), will be enrolled. Patient eligibility will be assessed during a 7-week Screening Period (Day -50 to Day -1). This will be followed by a 12-week Treatment Period (Part 1), in which patients will be administered leniolisib doses ranging from 40 to 70 mg twice daily (BID) based on body weight (see dose regimen table below). A Part 1 clinical study report will be generated once the last patient completes the Day 85 Visit for Part 1. Patients who complete the Day 85 Visit will enter the Extension Period of the study (Part 2), in which patients will be administered leniolisib doses ranging from 40 to 70 mg BID (based on body weight) for 1 year or until marketing approval in Japan, whichever is longer. A 4-week Follow-up Period will occur after the last dose of study treatment is received.
It is anticipated that a total of 3 patients will be enrolled into the study.
Objectives:
Part 1:
Primary:
- To assess the safety and tolerability of leniolisib
- To assess the efficacy of leniolisib on lymphoproliferation (sum of product diameters [SPD] of index lymph node lesions) and immunophenotype normalization (percentage of naïve B cells out of total B cells)
Secondary:
- To assess the efficacy of leniolisib on lymphoproliferation (non-index lymph node lesions and spleen)
- To assess the pharmacokinetics (PK) of leniolisib in the Japanese population
- To assess the efficacy of leniolisib to modify health-related quality of life
- To assess the efficacy of leniolisib by the Patient's and Physician's Global Assessments
- To assess the frequency of infections, antibiotic use, and immunoglobulin (Ig) replacement therapy and assessment of impact on other disease-related outcomes (e.g., cytopenia, colitis, and lung function)
- To assess biomarkers reflecting the efficacy of leniolisib to reduce systemic inflammatory components of the disease
- To assess the treatment benefit to individual patients
Part 2:
Primary:
- To assess the long-term safety and tolerability of leniolisib
Secondary:
- To assess the long-term efficacy of leniolisib to modify health-related quality of life
- To assess the long-term efficacy of leniolisib on lymphoproliferation (non-index lymph node lesions and spleen)
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 tablets Leniolisib tablets in doses ranging from 40 to 70 mg twice daily (BID) based on body weight. A Part 1 clinical study report will be generated once the last patient completes the Day 85 Visit for Part 1. Patients who complete the Day 85 Visit will enter the Extension Period of the study (Part 2), in which patients will be administered leniolisib doses ranging from 40 to 70 mg BID (based on body weight) for 1 year or until marketing approval in Japan, whichever is longer. A 4-week Follow-up Period will occur after the last dose of study treatment is received. |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Patient is Japanese.
- Patient is male or female and 12 to 75 years of age (inclusive) at the time of the first study procedure.
- Patient weighs ≥35 kg at baseline.
- Patient has a PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
- Patient has at least 1 measurable nodal lesion on computed tomography (CT) or magnetic resonance imaging (MRI) scan within 6 months of Screening.
Key exclusion criteria (15 listed)
- Patient has previous or concurrent use of immunosuppressive medication such as the following:
- A mammalian target of rapamycin inhibitor (e.g., sirolimus, rapamycin, or everolimus) or a PI3Kδ inhibitor (selective or non-selective phosphoinositide 3-kinase inhibitors) within 6 weeks prior to first dose.
- B-cell depleters (e.g., rituximab) within 6 months prior to first dose of study treatment.
- Belimumab or cyclophosphamide within 6 months prior to first dose of study treatment.
- Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of 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 range from 40 to 70 mg BID (based on body weight, resulting in total daily doses ranging from 80 to 140 mg a day for 12 weeks in Part I and 1 year in Part II, or until marketing approval in Japan, whichever is longer. | Drug | Leniolisib |
Primary outcome
Part I: Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs
Time frame: Between baseline until Day 85
Incidence of treatment-emergent adverse events (AEs), serious adverse events (SAEs), and AEs leading to discontinuation of study treatment
Part II: Long-term Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs
Time frame: At Day 252, through study completion, an average of 1 year
Long-term Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs leading to discontinuation of study treatment
Part I: Change from baseline in clinical laboratory test results
Time frame: Between baseline until Day 85
Number of participants with change in clinical laboratory test results (hematology, blood chemistry, urinalysis). Absolute values and change from baseline values of vital signs (including body weight) and ECG results at each visit will be listed for each patient and may be summarized using descriptive statistics. Tanner staging will be listed for each patient at each visit. Plots may also be used to display the patient data over time.
Part II: Long-term change from baseline in clinical laboratory test results
Time frame: At Day 252, through study completion, an average of 1 year
Number of participants with long-term change in clinical laboratory test results (hematology, blood chemistry, urinalysis). Absolute values and change from baseline values of vital signs (including body weight) and ECG results at each visit will be listed for each patient and may be summarized using descriptive statistics. Tanner staging will be listed for each patient at each visit. Plots may also be used to display the patient data over time.
Part I: Change from baseline in vital signs
Time frame: Between baseline until Day 85
Number of Participants with change in vital signs
Part II: Long-term change from baseline in vital signs
Time frame: At Day 252, through study completion, an average of 1 year
Number of Participants with long-term change in vital signs
Part I: Change from baseline in physical examination findings
Time frame: Between baseline until Day 85
Number of participants with change in physical examination findings
Part II: Long-term change from baseline in physical examination findings
Time frame: At Day 252, through study completion, an average of 1 year
Number of participants with long-term change in physical examination findings
Part I: Change from baseline in electrocardiograms (ECGs)
Time frame: Between baseline until Day 85
Number of participants with change in electrocardiograms (ECGs)
Part II: Long-term change from baseline in electrocardiograms (ECGs)
Time frame: At Day 252, through study completion, an average of 1 year
Number of participants with long-term change in electrocardiograms (ECGs)
Part I: To assess the efficacy of leniolisib on lymphoproliferation (SPD of index lymph node lesions) and immunophenotype normalization (percentage of naïve B cells out of total B cells)
Time frame: Between baseline until Day 85
Number of patients with change in SPD of index lesions (selected as per the Cheson methodology from magnetic resonance imaging [MRI] or computed tomography [CT] imaging) at the end of treatment
Part I: Change from baseline in the percentage of naïve B cells out of total B cells at the end of treatment
Time frame: Between baseline until Day 85
Number of participants with change in percentage of naïve B cells out of total B cells at the end of treatment
9 secondary outcomes
Part I: Change from baseline in lymphoproliferation measured using MRI, CT imaging, or ultrasound
Time frame: Between baseline until Day 85
Part II: Long-term change from baseline in lymphoproliferation measured using MRI, CT imaging, or ultrasound
Time frame: At Day 252, through study completion, an average of 1 year
Part I: To assess the PK of leniolisib in the Japanese population
Time frame: Between baseline until Day 85
Part I: To assess the efficacy of leniolisib to modify health-related quality of life
Time frame: Between baseline until Day 85
Part II: To assess the long-term efficacy of leniolisib to modify health-related quality of life
Time frame: At Day 252, through study completion, an average of 1 year
Part I: To assess the efficacy of leniolisib by the Patient's and Physician's Global Assessments
Time frame: Between baseline until Day 85
Part I: To assess the frequency of infections and assessment of impact on other disease-related outcomes (e.g., cytopenia, colitis, and lung function)
Time frame: Between baseline until Day 85
Part I: To assess biomarkers reflecting the efficacy of leniolisib to reduce systemic inflammatory components of the disease
Time frame: Between baseline until Day 85
Part I: To assess the treatment benefit to individual patients
Time frame: Between baseline until Day 85
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2024.
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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.
Hirokazu Kanegane, Prof. principal investigator
Tokyo Medical And Dental University Hospital
2 sites in 1 country: 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 Gene Mutation, run by Pharming Technologies B.V. at 2 sites in Japan.
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 10 inclusion and 15 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: status not confirmed, as of the sponsor's last update on 8 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Japan. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jason Bradt, MD (+31715247400, J.Bradt@pharming.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part I: Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs (time frame: Between baseline until Day 85). The study lists 12 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 8 Feb 2024.