Study of the Efficacy and Safety of Parsaclisib in Participants with Primary Warm Autoimmune Hemolytic Anemia

Phase 1

• Conditions: Primary Warm Autoimmune Hemolytic Anemia; MedDRA version: 20.0Level: LLTClassification code 10003825Term: Autoimmune hemolytic anemiaSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2021-002844-66-HU
• Lead Sponsor: Incyte Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-11-15
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 100

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 < 10 g/dL with symptoms of anemia as assessed by the investigator at screening (hemoglobin as determined by local laboratory).
6. FACIT-F score = 43 at screening.
7. Willingness to avoid pregnancy or fathering children based on the criteria below.
a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children (with 99% certainty) from screening through 90 days after the last dose of study drug and must refrain from donating sperm during this period. Permitted methods in that are at least 99% effective preventing
pregnancy should be communicated to the participants and their understanding confirmed.
b. Female participants who are WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test before randomization and must agree to take appropriate precautions to avoid pregnancy (with 99% certainty) from screening through 90 days after the last dose of study drug and must refrain from donating oocytes during this period. Permitted methods in that are at least 99% effective preventing pregnancy should be communicated to the participants and their understanding confirmed.
b. A female participant not considered to be of childbearing potential is eligible.
Note: This criterion does not apply to women of nonchildbearing potential (i.e., surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, defined as amenorrhea at least 12 months before screening, confirmed by FSH levels at screening).
8. Willingness to receive PJP prophylaxis during the study period from Day 1 through at least 2 to 6 months after the last dose of study drug.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 80
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

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 New York 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.
7. Hepatitis B (HBV) or hepatitis C (HCV) infection: Participants 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. Participants 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 3 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 potent CYP3A4 inhibitors or moderate or potent CYP3A4 inducers, within 14 days or 5 half-lives (whichever is longer) before randomizatio

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified