Efficacy and Safety of M281 in Adults with Warm Autoimmune Hemolytic Anemia: A Multicenter, Randomized, Double-blind, Placebo-controlled Study with a Long-term Open-label Extensio
- Conditions
- Warm Autoimmune Hemolytic AnemiaMedDRA version: 20.0Level: LLTClassification code: 10073784Term: Anemia hemolytic autoimmune Class: 10005329Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- CTIS2023-505321-14-00
- Lead Sponsor
- Janssen - Cilag International
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 111
Double blind period - 1.Participants =18 years of age, Women of childbearing potential, defined as women physiologically capable of becoming pregnant, must have a negative serum pregnancy test at screening and a negative urine pregnancy test at Baseline. Menopausal women must have an elevated serum FSH level at Screening; if the FSH is not elevated, they are considered to be of childbearing potential and must have a negative serum pregnancy test at screening and a negative urine pregnancy test at Baseline to be eligible., Women of childbearing potential (including menopausal women who do not have elevated FSH) must agree to remain totally abstinent (i.e., refrain from sexual intercourse during the study) or to consistently use a reliable and highly effective method of contraception during the study and for 30 days after the last dose of study drug., Male participants must wear a condom when engaging in any activity that allows for passage of ejaculate to another person during the study and for at least 90 days after receiving the last dose of study drug. In addition, male participants with partners who are a woman of childbearing potential are to be highly encouraged to inform their partner to use highly effective contraception methods that result in a low failure rate (less than 1% per year), Participants who use herbal, naturopathic, traditional Chinese remedies, ayurvedic and nutritional supplements, or medical marijuana (with a doctor's prescription) are eligible if the use of these medications is acceptable to the Investigator. These remedies must be at a stable dose and regimen using the same preparation for =2 months prior to Screening., Are able to understand and voluntarily provide written informed consent to participate in the study and comply with all study procedures., Vaccinations prior to screening as per routine local guidelines (including COVID-19), Open-Label 1. Have completed the double-blind period (through Week 24), or have required rescue therapy at/or after Week 4 of the double-blind period, or failed to demonstrate an increase from baseline in Hgb of at least 1 g/dL and are symptomatic at or after Week 16 of the double-blind period., 2. Are able to understand and voluntarily provide written informed consent to participate in the OLE period and comply with all study procedures., Diagnosed with active primary or secondary wAIHA, defined as: a. Hgb value<10 g/dL AND b. Signs of hemolysis, defined as: lactate dehydrogenase (LDH) levels above upper limit of normal (ULN), or haptoglobin below lower limit of normal, or indirect bilirubin above ULN AND c. Serological evidence of anti-erythrocyte antibodies associated with a DAT that is either positive for IgG only or is positive for IgG+C3d at screening. DAT: negative, can be repeated once. If negative, participant not eligible., Diagnosed with wAIHA for at least 3 months, and currently receiving or previously received treatment for wAIHA (treatment naïve participants not eligible), If on corticosteroids, participants must have been on treatment for at least 4 weeks with a stable dose during the screening period or for at least 14 days prior to randomization, whichever is longer. Note: Investigators can optimize the above background medications prior to randomization if they are following the above rules for stable dose duration., If receiving immunosuppressants, following drugs allowed: concomitant immunosuppressants are azathioprine, mycophenolate mofetil/mycophenolic a
Double-blind period 1.Are currently taking IgG Fc-related protein therapeutics., Have any other associated cause of hereditary or acquired hemolytic anemia., Have received rituximab within 3 months prior to screening, Have received IVIg within 6 weeks prior to screening, Have been diagnosed with cold antibody AIHA, cold agglutinin syndrome, mixed type (ie, warm and cold) AIHA, or paroxysmal cold hemoglobinuria., Have a severe infection that requires parenteral anti-infectives and/or hospitalization, and/or is assessed as serious/clinically significant by the Investigator, within 8 weeks prior to screening. Any participant with an infection requiring oral anti-infectives (eg, sinusitis, bronchitis, uncomplicated urinary tract infection) within 4 weeks prior to screening will be excluded, Have a chronic infection or require chronic treatment with anti-infectives., Have received a live viral or bacterial vaccine within 4 weeks prior to first dose of study drug or have a known need to receive a live viral or bacterial vaccine during the study or within at least 8 weeks after the last dose of study drug., Open Label Extension 1. Met any of the stopping criteria or discontinued study drug during the double-blind period due to treatment- related AE., Currently have a serious or clinically significant infection requiring parenteral anti-infectives and/or hospitalization. The following exclusion criterion from the Double-blind Period also applies to enrollment in the OLE: Exclusion Criteria #8 and #21. Exception: Participants who were previously enrolled in this study and unable to complete the double-blind period due to the Sponsor suspending dosing due to the COVID-19 pandemic can be enrolled in the OLE after meeting all of the double-blind eligibility criteria. Participants who completed the 28-week OLE before Amendment 6 can be re-enrolled in the OLE per investigator's discretion if the participants continue meet the eligibility criteria for the OLE. Participants will resume study treatment calculated from the baseline visit once they have reconsented to the study., Have any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of their wAIHA or have a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant., Have any of the following viral testing outcomes: A history of HIV infection or positive test result for HIV-1 and HIV 2 antibodies; positive test for hepatitis B virus surface antigen. For participants with a negative test for HBsAg along with a positive test for anti-hepatitis B core antibodies and a positive or negative test for anti-HBs antibodies, hepatitis B viral DNA detection will be performed. Participants with a positive hepatitis B viral DNA detection will be excluded. If HBV DNA testing cannot be performed, or there is evidence of chronic liver disease, the participant is not eligible for the protocol; A positive test for hepatitis C virus (HCV) unless 1 of the following conditions are met: (a) Has a history of successful treatment, defined as being negative for HCV RNA at least 24 weeks after completing antiviral treatment, and has a negative HCV RNA test result at screening, OR (b) -Has a negative HCV RNA test result at least 24 weeks prior to screening and a negative HCV RNA test at the screening., Are currently breastfeeding, pregnant, intend to become pregnant during the study, or are planning egg
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