Primary Warm Autoimmune Hemolytic Anemia MedDRA version: 20.0 Level: LLT Classification code 10003825 Term: Autoimmune hemolytic anemia System Organ Class: 100000004851
Conditions
Interventions
Product Name: Parsaclisib (1 mg)
Product Code: INCB050465
Pharmaceutical Form: Tablet
INN or Proposed INN: Paraclisib
Current Sponsor code: INCB050465
Other descriptive name: INCB050465 HYDROCHLORIDE
Sponsors
Incyte Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Ability to comprehend and willingness to sign a written ICF for the study. 2. Men or women, age = 18 years at the time of signing the ICF. 3. Diagnosis of primary wAIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the DAT positive for IgG only or IgG plus C3d. Note: A DAT performed at screening is preferred; however, prior documentation of DAT results within 3 months of randomization is permitted. 4. Participants who were inadequately controlled with, were intolerant to, or have a contraindication to other therapies. There is no limit to the number of prior treatment regimens. 5. Hemoglobin = 6.5 to =65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1. Women currently pregnant or breastfeeding or participants expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 90 days from the date of last dose of study drug. 2. A diagnosis of other types of AIHA; CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria. 3. Warm AIHA suspected to be secondary to a lymphoproliferative malignancy or secondary to an autoimmune disease (eg, systemic lupus erythematosus, Castleman's disease, Sjögren's syndrome, or other autoimmune diseases or diagnosis of Evans syndrome). 4. A splenectomy less than 3 months before randomization. 5. Concurrent conditions or history of other diseases: a. History or clinical manifestations of significant unstable metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, urological, neurological, or psychiatric disorders. b. Current or previous malignancy within 5 years of study entry, except basal or squamous cell skin cancer with removal considered to be curative, superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy without sponsor approval. c. Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, and/or cardiac conduction issues within 6 months of randomization. d. Current NY Heart Association Class II to IV congestive heart failure or uncontrolled arrhythmia. 6. Known diagnosis of anti-phospholipid syndrome or history of persistent anti-phospholipid antibodies. Note: Participants at risk for thrombosis who are on adequate prophylaxis or those with thrombosis on stable treatment for 3 months prior to randomization are eligible. 7. Hepatitis B or C infection: those who are positive for the hepatitis B surface antibody or hepatitis B core antibody will be eligible if they are negative for HBV-DNA; these participants should be considered for prophylactic antiviral therapy. Those who are positive for the anti-HCV antibody will be eligible if they are negative for HCV-RNA. 8. Known HIV infection or positivity on immunoassay. 9. History or presence of an abnormal ECG that, in the investigator's opinion, is clinically meaningful. Participants with screening QTc interval > 470 milliseconds for males and > 480 milliseconds for females (corrected by Fridericia) are excluded. In the event that a single QTcF is > 470 milliseconds for males or > 480 milliseconds for females, the participant may enroll if the average QTcF for triplicate ECGs is < 470 milliseconds for males or < 480 milliseconds for females. 10. Use of the following medications: a. Treatment with rituximab within 6 weeks of the randomization. b. Use of immunosuppressive therapy within 28 days of the randomization. Immunosuppressive therapy includes, but is not limited to, cyclosporine A, azothioprine, mycophenolate mofetil, or cyclophosphamide. Note: Participants receiving corticosteroids must be at a stable dose level (no change in daily dose) = 40 mg/day (prednisone or equivalent corticosteroid dose) for at least 14 days prior to randomization. c. Use of IVIG or erythropoietin within 2 weeks of randomization. d. Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine within 30 days of randomization. e. Use or expected use during the study of any prohibited medications, including
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.;Secondary Objective: Key secondary objective: •To further evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA. Other secondary objectives: •To further evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA. •To evaluate the safety and tolerability of parsaclisib in participants with wAIHA. Exploratory objectives: •To further evaluate the efficacy of parsaclisib. •To evaluate the participant's quality of life and other PROs. •To characterize serum biomarkers and/or leukocyte profiles in participants with wAIHA treated with parsaclisib. •To evaluate PK of parsaclisib in participants with wAIHA.;Primary end point(s): Proportion of participants attaining a durable hemoglobin response, defined as hemoglobin = 10 g/dL with an increase from baseline of = 2 g/dL not attributed to rescue therapy at = 3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.;Timepoint(s) of evaluation of this end point: The primary analysis will occur after the primary database lock, when the last randomized participant has completed the double-blind treatment period. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Proportion of participants with a = 3-point increase from baseline in FACIT-F score at Week 24.;Timepoint(s) of evaluation of this end point: Week 24 | — |
Countries
Austria, Belgium, Canada, France, Germany, Hungary, Israel, Italy, Japan, Netherlands, Poland, Spain, Ukraine, United Kingdom, United States
Contacts
Public ContactMedical Information
Incyte Corporation
Outcome results
None listed