Clinical trial NCT05676697: PI3K Delta Inhibitor in Relapsed / Refractory Autoimmune Hemolytic Anemia Patients After Receiving Two or More Lines of Therapy
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Autoimmune Hemolytic Anemia
Failure of Two Rounds of Treatment
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
Collaborators: name: YL-Pharma
First posted: 2023-01-09
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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
* 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

Primary outcomes:
measure: Overall response rate
timeFrame: 6-12 weeks
Secondary outcomes:
measure: Incidence of the adverse event
timeFrame: 12 weeks
measure: Complete response rate
timeFrame: 6-12 weeks
measure: Complete response with incomplete hemolysis recovery, CRi
timeFrame: 6-12 weeks
measure: Mean change from baseline in hemoglobin (Hgb) levels
timeFrame: 6-12 weeks
measure: Time to achieve partial response (PR)
timeFrame: 6-12 weeks
measure: Time to achieve complete response (CR)
timeFrame: 6-12 weeks
measure: Change of the health-related quality of life
timeFrame: Baseline and 12 weeks
Other outcomes:
Locations:
city: Zhoukou
country: China
facility: Zhoukou Central Hospital
state: Henan
city: Tianjin
country: China
facility: Regenerative Medicine Center
state: Tianjin
Study officials:
References: