A Phase 3 Study of Obexelimab in Patients with Warm Autoimmune Hemolytic Anemia
- Conditions
- WARM AUTOIMMUNE HEMOLYTIC ANEMIAMedDRA version: 20.0Level: PTClassification code: 10047822Term: Warm type haemolytic anaemia Class: 100000004851Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- CTIS2022-501005-12-00
- Lead Sponsor
- Zenas Biopharma (USA) LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 24
PARTS A AND B: INCLUSION CRITERIA 1.Males and females = 18 years of age at the time of signing the informed consent, 10.Screening neutrophil count = 1,000 mm3, 11.Screening serum albumin and serum calcium concentrations within the normal range, 12.Screening total serum IgG of = 600 mg/dL, 13.Screening creatine kinase value < 2 × ULN, 14.Patients with a history of splenectomy must be at least 4 months post resection prior to randomization and must be vaccinated as per country-specific immunization schedules, 15.Patients with autoimmune disorders (e.g., systemic lupus erythematosus, rheumatoid arthritis) may be eligible if they are receiving stable treatment (no changes in disease related concomitant medications), and the severity of disease has been stable for at least 4 months prior to randomization, 16.For the SRP only (Part A), patients with LPDs (Cohort 2) may be eligible if they are receiving stable treatment (no changes in concomitant disease–related medications), the severity of disease has been stable for at least 4 months prior to randomization, and, in the opinion of the Investigator, they are unlikely to require chemotherapy or mAb therapy during the study, 17.Females not pregnant (see Appendix 4), not breastfeeding, and for whom at least one of the following conditions applies: a.Not of childbearing potential, as defined in Appendix 4 OR b.Of childbearing potential; females of childbearing potential (FOCBP) must have a negative serum pregnancy test at screening and a negative urine pregnancy test prior to the first dose of study drug and agree to follow the contraceptive guidance in Appendix 4 during the SRP/RCP and for at least 1 month (i.e., approximately 5 half-lives) after the last administration of study drug, 18.Males must: a.Agree to (i) abstain from intercourse or (ii) use contraception (as detailed in Appendix 4) during the SRP/RCP and for at least 1 month (i.e., approximately 5 half lives) after the last dose of IMP, or (iii) be surgically sterile for the duration of the study AND b.Agree to refrain from donating sperm for the duration of the study and for at least 1 month (i.e., approximately 5 half-lives) after the last dose of IMP, 19.Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol, 2.Diagnosed with wAIHA for at least 3 months and currently receiving treatment for wAIHA or have previously received treatment for wAIHA (treatment-naive patients are not eligible), PART C: OLE PERIOD INCLUSION CRITERIA 1.Completed the Week 24 SRP or RCP visit 2.Have not had IMP discontinued due to any of the following safety reasons: a.Pregnancy b.Malignancy c.Hypersensitivity to IMP d.Determination that the patient was ineligible for the SRP and RCP e.For any reason deemed necessary by the investigator for patient safety 3.Have not discontinued from IMP due to unblinding of a patient 4.FOCBP must have a negative serum pregnancy test prior to enrollment in the OLE Period 5.Have not received a transfusion within 2 weeks prior to first dose in the OLE Period 6.Not receiving more than 2 concomitant medications for the treatment of wAIHA, excluding vitamins or other supplements, at the time of enrollment in the OLE Period 7.Patients must receive first dose of obexelimab in the OLE Period within 14 days of the Week 24 SRP/RCP visit 8.Willing to comply with all study protocol procedures and complete all study
PARTS A AND B: EXCLUSION CRITERIA 1.Have cold antibody AIHA, cold agglutinin syndrome, mixed type (i.e., warm and cold) AIHA, or paroxysmal cold hemoglobinuria, 10. Evidence of active tuberculosis (TB) or at high risk for TB based on at least one of the following: a. History of active TB or latent TB, unless completion of treatment according to local guidelines is documented b. Positive, indeterminate, or invalid interferon-gamma (IFN?) release assay results at screening, unless treatment is documented. Patients with an indeterminate test result can repeat the test once either centrally or locally, but if the repeat test is also indeterminate, the patient is excluded c. Signs of symptoms that could represent active TB d. Chest radiograph, computed tomography scan, or magnetic resonance imaging that suggests possible diagnosis of TB, 11. History or evidence of a clinically unstable/uncontrolled disorder, condition, or disease (including, but not limited to, cardiopulmonary, oncologic, renal, hepatic, metabolic, hematologic, psychiatric, active infection), that, in the opinion of the investigator, would pose a risk to patient safety or interfere with the study evaluations, procedures, or completion, 12. Known allergy to mAb therapy, 13. Known hypersensitivity to dextran or components of dextran, 14. Active infection (e.g., pneumonia, biliary tract infection, diverticulitis, Clostridium difficile infection) that requires parenteral or oral anti-infectives and/or hospitalization, and/or is assessed as serious/clinically significant by the investigator, within 8 weeks prior to screening. Patients may be re-screened after the 8-week exclusionary period has passed, 15. Chronic infection (e.g., bronchiectasis, chronic osteomyelitis, chronic pyelonephritis) or requiring chronic treatment with anti-infectives (e.g., antibiotics, antivirals), 16. Confirmed or suspected clinical immunodeficiency syndrome not related to treatment of wAIHA, or has a family history of congenital or hereditary immunodeficiency, unless confirmed absent in the patient, 17. Acute hepatitis B infection (hepatitis B surface antigen-positive), active hepatitis C virus (HCV), or HIV infection. Patients will be excluded from the study if they have a positive test for active hepatitis B through detection of hepatitis B surface antigen. In Japan, patients will be excluded if there is detection of (a) hepatitis B surface antigen or (b) hepatitis B surface antibody or (c) hepatitis B core antibody. Patients with a history of HCV will be excluded in the study unless there is documentation of a negative HCV ribonucleic acid level in the serum at 12 weeks or longer after the completion of HCV therapy, 18. Intend to become pregnant, breastfeed, or are planning egg donation during the study or within 30 days after the last dose of study drug, 19. Current alcohol/substance abuse/dependence, a history of alcohol/substance abuse/dependence within the 12 months prior to randomization, or, in the investigator’s opinion, there is evidence of ongoing alcohol/substance abuse/dependence, 2. Have any other associated cause of hereditary or acquired hemolytic anemia, 20. Major surgery within 4 months prior to randomization or have plans for or have been scheduled for any elective surgery or major dental procedure during the study, 21. Have a history of a major organ transplant (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant, 22. Malignancy within 5 years of rando
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method