Primary Immunodeficiency Disorders (PIDs)Phase 2NCT06549114
Leniolisib for Immune Dysregulation in PIDs
A Phase 2 study testing Leniolisib in people with Primary Immunodeficiency Disorders (PIDs), run by Pharming Technologies B.V. at 1 site in United States.
Trial summary
This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib. Secondary objectives include assessments of PK/PD, and to explore clinical efficacy measures with administration of three different dose levels of… Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Primary Immunodeficiency Disorders (PIDs)
- 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
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Oct 2024to 23 Jun 2026
- Phase
- Phase 2
- 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
- Subjects 12 to 75 years of age.
- Diagnosed with a PID due to disease-causing pathogenic or likely pathogenic variant(s) in the following genes: SOCS1, PTEN, CTLA4, NFKB1 (only those variants leading to NFKB pathway activation), or FAS (germline or somatic), or diagnosed with RAS associated leukoproliferative disorder (and not juvenile myelomonocytic leukemia [JMML]) due to somatic variants in NRAS or KRAS.
- Subjects must have 1 or more of the following:
- One or more blood cytopenias related to the underlying PID defined as hemoglobin <10 g/dL, platelet count <100,000/µL, or neutrophil count <1000/µL
- Splenomegaly evident by CT imaging with craniocaudal spleen measurement >10 cm
- Lymphadenopathy evident by CT imaging with at least 1 measurable index lymph node (long axis >1.5 cm) as per Cheson methodology
Show all 13Show fewer
- GLILD or other PID-related ILD with quantifiable CT chest imaging findings evident on baseline CT scan
- At screening, vital signs.
- Systolic blood pressure 80-139 mm Hg
- Diastolic blood pressure 50-89 mm Hg
- Pulse rate 50-110 bpm
- Oxygen saturation 93-100%
- Subjects or their legal representatives (for subjects under the age of 18 years) must be able to provide written informed consent.
Who cannot join
- Subject has had a successful hematopoietic stem-cell transplant (HSCT).
- Previous or concurrent use of immunosuppressive medication, such as:
- Use of an mTOR inhibitor or a PI3Kδ inhibitor within 3 weeks prior to first dosing .
- Rituximab or other B-cell depleting antibodies, belimumab, cyclophosphamide, or alemtuzumab within 6 months prior to first dosing.
- Cyclosporine A, mycophenolate mofetil, 6-mercaptopurine, azathioprine, methotrexate, tacrolimus, ruxolitinib, or other JAK inhibitors within 3 weeks prior to first dosing.
- Corticosteroids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dosing.
Show all 25Show fewer
- Other immunosuppressive agents expected to have a significant impact on immune cell number or function.
- Abatacept is allowed during study if the subject has been receiving a stable dosing regimen for more than 3 months prior to first dosing.
- 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.
- 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 P450 CYP3A.
- Current use of medications that act as BCRP, OATP1B1, and OATP1B3 substrates.
- Subject has a history or current electrocardiogram (ECG) abnormalities indicating a significant risk of safety for subjects participating in the study
- History of acquired immunodeficiency diseases, including a positive HIV test result at screening.
- Uncontrolled chronic or recurrent infectious disease (except those considered to be characteristic of a PID) or evidence of tuberculosis infection
- 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.
- A positive hepatitis B surface antigen, positive hepatitis B PCR, positive hepatitis C PCR, or positive hepatitis C antibody result at screening.
- Administration of live vaccines starting from 6 weeks before first dose of study medication.
- Subject has a previous diagnosis of lymphoma within 1 year of the first dose of study medication.
- Subject has a history of malignancy (except lymphoma) within 3 years before the first dose of study medication, 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 (PTLD)-like EBV related lymphoproliferative disease.
- Donation or loss of 400 mL or more of blood within 8 weeks before the first dose.
- Subject has had major surgery requiring hospitalization or radiotherapy within 4 weeks prior to the first dose or has a planned or expected major surgical procedure during the study period.
- Pregnant or nursing (lactating) individuals,.
- Individuals of child-bearing potential, unless they are using highly effective methods of contraception.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Health
Bethesda, Maryland, 20892
About this study
Official title: A Study to Assess Safety and Tolerability, and Explore Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders
This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib. Secondary objectives include assessments of PK/PD, and to explore clinical efficacy measures with administration of three different dose levels of leniolisib.
Read the detailed description
Patients ages 12-75 diagnosed with genetically defined PID disorders linked to PI3K signaling. This includes disorders caused by pathogenic variants in SOCS1, PTEN, CTLA4, NFKB1 (variants leading to NFKB pathway activation), FAS (germline or somatic), or RAS-associated leukoproliferative disorder caused by somatic variants in NRAS or KRAS (not juvenile myelomonocytic leukemia [JMML]). All subjects participating will receive leniolisib film-coated tablets (FCTs) with a planned dose regimen consisting of a starting dose of 10 mg twice daily (BID) for 4 weeks, followed by 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks. Leniolisib dose increase at the individual subject level will occur if no safety or tolerability issues have been identified by the Investigator that precludes the planned dose escalation.
Subjects not continuing leniolisib treatment outside of the current protocol will be followed up, with the EOS visit planned to occur approximately 28 days after last dose of leniolisib.
- Being tested
- Leniolisib
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeniolisibDrug
Also called Joenja
The doses selected will range from 10 to 70 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 140 mg per day).
| Study group | Role | What they receive |
|---|---|---|
| Leniolisib | Experimental | All subjects participating will receive Leniolisib film-coated tablets (FCTs) at a starting dose of 10 mg BID for 4 weeks, followed by 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks. Receives: Drug: Leniolisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)
- To assess the number of AEs/SAEs and number of participants with AEs/SAEs
Measured: From Baseline to the end of 20 weeks of Treatment
Secondary outcomes (26)
To assess the PK of leniolisib in PID disorders linked to PI3K
- PK parameters for leniolisib defined by Cmax
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the PK of leniolisib in PID disorders linked to PI3K
- PK parameters for leniolisib defined by Tmax
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the PK of leniolisib in PID disorders linked to PI3K
- PK parameters for leniolisib defined by t1/2
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the PK of leniolisib in PID disorders linked to PI3K
- PK parameters for leniolisib defined by AUC (0-t)
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on lymphocyte PI3K/AKT/mTOR pathway activity in PID disorders linked to PI3K
- Percent change in pAKT and/or pS6 in B cells and/or T cells, under resting and stimulated conditions, at different dose levels of leniolisib relative to baseline pAKT/pS6 levels, as feasible
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on hemoglobin counts in PID disorders linked to PI3K
- Hemoglobin over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on platelet counts in PID disorders linked to PI3K
- Platelet count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on absolute neutrophil counts in PID disorders linked to PI3K
- Absolute neutrophil count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PID-related ILD in PID disorders linked to PI3K
- Change in CT evidence of GLILD or other PID-related ILD evaluated using Hartmann scoring methodology
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
- Change in forced expiratory volume 1 (FEV1)
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
- Change in forced vital capacity (FVC)
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
- Change in total lung capacity (TLC)
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
- Change in diffusing capacity (DLCO)
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on lymphoproliferation in PID disorders linked to PI3K
- Percent change from baseline in lymphadenopathy measured as the sum of product of diameters (SPD) in the index lesions selected per the Cheson methodology
- Percent change from baseline in lymphadenopathy measured as SPD of measurable non-index lesions selected as per the Cheson methodology
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on splenomegaly in PID disorders linked to PI3K
- Percent change from baseline in splenomegaly measured by three-dimensional (3D) volume and bi-dimensional (2D) size of spleen
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- White blood cell count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- Absolute monocyte count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- Absolute lymphocyte count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- Absolute eosinophil count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- Absolute basophil count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- CD4+ T cell count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- CD8+ T cell count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- Natural killer (NK) cell count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
- B cell count over time
Measured: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on B cell and T cell phenotypic populations of interest in PID disorders linked to PI3K
- Percentages of naïve B cells, CD21lo B cells and T regulatory cells over time
Measured: From Baseline to the end of 20 weeks of Treatment
To examine levels of CXCL13 and soluble IL-2Rα and assess the impact of leniolisib in PID disorders linked to PI3K
- Levels of CXCL13 over time
- Levels of soluble IL-2Rα over time
Measured: From Baseline to the end of 20 weeks of Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2024
- Study start21 Oct 2024
- Primary completion23 Jun 2026
- Study completion23 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Immunodeficiency Disorders (PIDs)Studies 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
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gulbu Uzel, M.D., Principal investigator, National Institutes of Health (NIH)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06549114
- Sponsor's protocol ID
- LE 7201
- Status last verified
- Oct 2026
- First submitted
- 17 Jul 2024
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hassan C, Ponstein Y, Hanssen R, Thorneloe KS, Marsh RA, Rao VK. Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome. J Hum Immun. 2025 Nov 17;2(1):e20250125. doi: 10.70962/jhi.20250125. eCollection 2026 Jan 5.
PubMed 41608120
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Assess Safety and Tolerability, and Explore Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders
- ClinicalTrials.gov ID
- NCT06549114
- Acronym / sponsor ID
- LE 7201
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- 21 Oct 2024 (actual)
- Primary completion
- 23 Jun 2026 (actual)
- Study completion
- 23 Jun 2026 (actual)
- First posted
- 12 Aug 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.
This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib. Secondary objectives include assessments of PK/PD, and to explore clinical efficacy measures with administration of three different dose levels of leniolisib.
Detailed description
Patients ages 12-75 diagnosed with genetically defined PID disorders linked to PI3K signaling. This includes disorders caused by pathogenic variants in SOCS1, PTEN, CTLA4, NFKB1 (variants leading to NFKB pathway activation), FAS (germline or somatic), or RAS-associated leukoproliferative disorder caused by somatic variants in NRAS or KRAS (not juvenile myelomonocytic leukemia [JMML]). All subjects participating will receive leniolisib film-coated tablets (FCTs) with a planned dose regimen consisting of a starting dose of 10 mg twice daily (BID) for 4 weeks, followed by 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks. Leniolisib dose increase at the individual subject level will occur if no safety or tolerability issues have been identified by the Investigator that precludes the planned dose escalation.
Subjects not continuing leniolisib treatment outside of the current protocol will be followed up, with the EOS visit planned to occur approximately 28 days after last dose of leniolisib.
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: LeniolisibAll subjects participating will receive Leniolisib film-coated tablets (FCTs) at a starting dose of 10 mg BID for 4 weeks, followed by 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks. |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Subjects 12 to 75 years of age.
- Diagnosed with a PID due to disease-causing pathogenic or likely pathogenic variant(s) in the following genes: SOCS1, PTEN, CTLA4, NFKB1 (only those variants leading to NFKB pathway activation), or FAS (germline or somatic), or diagnosed with RAS associated leukoproliferative disorder (and not juvenile myelomonocytic leukemia [JMML]) due to somatic variants in NRAS or KRAS.
- Subjects must have 1 or more of the following:
- At screening, vital signs.
- Subjects or their legal representatives (for subjects under the age of 18 years) must be able to provide written informed consent.
Key exclusion criteria (25 listed)
- Subject has had a successful hematopoietic stem-cell transplant (HSCT).
- 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.
- 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 P450 CYP3A.
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 |
|---|---|---|
| LeniolisibAlso: JoenjaThe doses selected will range from 10 to 70 mg twice daily (BID) (resulting in total daily doses ranging from 20 to 140 mg per day). | Drug | Leniolisib |
Primary outcome
Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)
Time frame: From Baseline to the end of 20 weeks of Treatment
• To assess the number of AEs/SAEs and number of participants with AEs/SAEs
26 secondary outcomes
To assess the PK of leniolisib in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the PK of leniolisib in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the PK of leniolisib in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the PK of leniolisib in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on lymphocyte PI3K/AKT/mTOR pathway activity in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on hemoglobin counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on platelet counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on absolute neutrophil counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PID-related ILD in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on lymphoproliferation in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the clinical efficacy of leniolisib on splenomegaly in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on white blood cell counts in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To assess the impact of leniolisib on B cell and T cell phenotypic populations of interest in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
To examine levels of CXCL13 and soluble IL-2Rα and assess the impact of leniolisib in PID disorders linked to PI3K
Time frame: From Baseline to the end of 20 weeks of Treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
- Derived from this study: PubMed 41608120Hassan C, Ponstein Y, Hanssen R, Thorneloe KS, Marsh RA, Rao VK. Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome. J Hum Immun. 2025 Nov 17;2(1):e20250125. doi: 10.70962/jhi.20250125. eCollection 2026 Jan 5.
- Individual participant dataSharing planned: No
Related trials for Primary Immunodeficiency Disorders (PIDs)
Active, not recruitingPhase 2
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
PIDs Linked to PI3K
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.
Gulbu Uzel, M.D. principal investigator
National Institutes of Health (NIH)
1 site in 1 country: 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 Primary Immunodeficiency Disorders (PIDs), run by Pharming Technologies B.V. at 1 site in United States.
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 13 inclusion and 25 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: finished, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs) (time frame: From Baseline to the end of 20 weeks of Treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06549114, which also lists 1 linked publication.
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 19 Oct 2026.