Autoimmune Hemolytic AnemiaPhase 1NCT05676697
PI3K Delta Inhibitor in Relapsed / Refractory Autoimmune Hemolytic Anemia Patients After Receiving Two or More Lines of Therapy
A Phase 1 study testing Linperlisib in people with Autoimmune Hemolytic Anemia and Failure of Two Rounds of Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 2 sites in China.
Trial summary
This is a prospective, multicenter, single-arm, pilot study. The aim of this study is to evaluate the efficacy and safety of Linperlisib, the PI3K delta inhibitor for autoimmune hemolytic anemia patients who failed the second line therapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Hemolytic Anemia, Failure of Two Rounds of Treatment
- What is being tested?
- Linperlisib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesChina
- Study dates
- 13 Jan 2023to 6 Feb 2024
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female age ≥ 18 years
- Diagnosis of primary warm antibody hemolytic anemia (AIHA).
- Hemoglobin < 100g/L
- Refractory to or relapsed after at least 2 prior treatment line.
- ECOG performance status ≤ 2
- Willing and able to comply with the requirements for this study and written informed consent.
Who cannot join
- Neutrophils counts < 0.5×10^9/L or platelet counts < 50 x 10^9/L
- Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH).
- Diagnosis of the active stage of the connective tissue or systemic autoimmune rheumatic diseases (SARDs)
- History of lymphoproliferative neoplasms
- Had other inherited or acquired hemolytic diseases.
- Secondary AIHA caused by drugs or infection
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- Previously received organ or stem cell transplantation.
- Had malignant tumor within 5 years before enrollment, exclusive of cured basal or squamous cell skin cancer, superficial bladder cancer, prostate intraepithelial tumor, cervical carcinoma in situ or other indolent tumors
- Patients with HBV, HCV, HIV or other infections that require treatment.
- Abnormal liver function: two consecutive examinations with an interval of ≥1 week suggest that ALT and AST are 2.5 times higher than the upper limit of normal values
- Renal impairment: creatinine clearance <60ml/min
- Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study, including clinically significant cardiac diseases, refractory hypertension, metabolic disorders and other diseases that seriously affect the function of the gastrointestinal tract.
- Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury.
- Received rituximab in 6 weeks before enrollment.
- Received attenuated vaccine 4 in weeks before enrollment
- Participation in another clinical trial within 4 weeks before the start of this trial
- Have an allergy to Linperlisib or any other part of this medicine.
- Previously treated with other PI3Kδ inhibitor.
- Pregnant or breast-feeding patients
- Patients considered to be ineligible for the study by the investigator for reasons other than the above
Trial locations and contacts
2 sites in 1 country.
China2 sites
Regenerative Medicine Center
Tianjin, Tianjin Municipality
Zhoukou Central Hospital
Zhoukou, Henan
About this study
Official title: Safety and Efficacy Study of PI3K Delta Inhibitor in Relapsed / Refractory Autoimmune Hemolytic Anemia Patients After Receiving Two or More Lines of Therapy
This is a prospective, multicenter, single-arm, pilot study. The aim of this study is to evaluate the efficacy and safety of Linperlisib, the PI3K delta inhibitor for autoimmune hemolytic anemia patients who failed the second line therapy.
- Being tested
- Linperlisib
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LinperlisibDrug
The phosphatidylinositol 3-kinase delta (PI3Kδ) signaling pathway plays a critical role in the activation, proliferation, and tissue homing of self-reactive B cells that contribute to autoimmune diseases. B cells play an essential role in immune system function and dysfunction (e.g., autoimmunity) by producing antibodies and by acting as antigen-presenting cells (APCs) for T cells. Signaling via PI3K controls many essential B cell functions and is therefore a promising target for preventing aberrant B cell activation.
| Study group | Role | What they receive |
|---|---|---|
| PI3K Delta Inhibitor | Experimental | Not described Receives: Drug: Linperlisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
Measured: 6-12 weeks
Secondary outcomes (7)
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event.
Measured: 12 weeks
Complete response rate
Percentage of patients with hematological complete response. Hematological response is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
Measured: 6-12 weeks
Complete response with incomplete hemolysis recovery, CRi
Percentage of patients with CRi which is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
Measured: 6-12 weeks
Mean change from baseline in hemoglobin (Hgb) levels
Measured: 6-12 weeks
Time to achieve partial response (PR)
Duration time was calculated from enrollment to PR. PR is assessed by hemoglobin and blood transfusion.
Measured: 6-12 weeks
Time to achieve complete response (CR)
Duration time was calculated from enrollment to CR.
Measured: 6-12 weeks
Change of the health-related quality of life
Medical Outcomes Study Questionnaire Short Form 36 Health Survey (SF-36) is used to assess the health-related quality of life of patients. The SF-36 has eight scaled scores; the scores are weighted sums of the questions in each section. Scores range from 0 - 100. Lower scores = more disability, higher scores = less disability
Measured: Baseline and 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2023
- Study start13 Jan 2023
- Primary completion31 Jan 2024
- Study completion6 Feb 2024
Why it stopped: Did not meet the anticipated outcome.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- YL-Pharma
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05676697
- Sponsor's protocol ID
- IIT2022067
- Status last verified
- Feb 2024
- First submitted
- 7 Dec 2022
- Last updated
- 11 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Study of PI3K Delta Inhibitor in Relapsed / Refractory Autoimmune Hemolytic Anemia Patients After Receiving Two or More Lines of Therapy
- ClinicalTrials.gov ID
- NCT05676697
- Acronym / sponsor ID
- IIT2022067
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Did not meet the anticipated outcome.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- YL-Pharma
- Study start
- 13 Jan 2023 (actual)
- Primary completion
- 31 Jan 2024 (actual)
- Study completion
- 6 Feb 2024 (actual)
- First posted
- 9 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 prospective, multicenter, single-arm, pilot study. The aim of this study is to evaluate the efficacy and safety of Linperlisib, the PI3K delta inhibitor for autoimmune hemolytic anemia patients who failed the second line therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PI3K Delta Inhibitor | Experimental | Drug: Linperlisib |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female age ≥ 18 years
- Diagnosis of primary warm antibody hemolytic anemia (AIHA).
- Hemoglobin < 100g/L
- Refractory to or relapsed after at least 2 prior treatment line.
- ECOG performance status ≤ 2
Key exclusion criteria (20 listed)
- Neutrophils counts < 0.5×10^9/L or platelet counts < 50 x 10^9/L
- Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH).
- Diagnosis of the active stage of the connective tissue or systemic autoimmune rheumatic diseases (SARDs)
- History of lymphoproliferative neoplasms
- Had other inherited or acquired hemolytic diseases.
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 |
|---|---|---|
| LinperlisibThe phosphatidylinositol 3-kinase delta (PI3Kδ) signaling pathway plays a critical role in the activation, proliferation, and tissue homing of self-reactive B cells that contribute to autoimmune diseases. B cells play an essential role in immune system function and dysfunction (e.g., autoimmunity) by producing antibodies and by acting as antigen-presenting cells (APCs) for T cells. Signaling via PI3K controls many essential B cell functions and is therefore a promising target for preventing aberrant B cell activation. | Drug | PI3K Delta Inhibitor |
Primary outcome
Overall response rate
Time frame: 6-12 weeks
Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin and other hemolysis-related laboratory indicators.
7 secondary outcomes
Incidence of the adverse event
Time frame: 12 weeks
Complete response rate
Time frame: 6-12 weeks
Complete response with incomplete hemolysis recovery, CRi
Time frame: 6-12 weeks
Mean change from baseline in hemoglobin (Hgb) levels
Time frame: 6-12 weeks
Time to achieve partial response (PR)
Time frame: 6-12 weeks
Time to achieve complete response (CR)
Time frame: 6-12 weeks
Change of the health-related quality of life
Time frame: Baseline and 12 weeks
Results and safety
The study was stopped early (reason given: Did not meet the anticipated outcome.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Linperlisib in people with Autoimmune Hemolytic Anemia and Failure of Two Rounds of Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 2 sites in China.
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 6 inclusion and 20 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: stopped early, as of the sponsor's last update on 11 Feb 2026. 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 China. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 was stopped early (reason given: Did not meet the anticipated outcome.). Results are not available in the source record.
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?
Overall response rate (time frame: 6-12 weeks). 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 11 Feb 2026.