EUCTR2019-000720-17-FRActive, not recruitingPhase 1
Efficacy and Safety of M281 in Adults with Warm Autoimmune Hemolytic Anemia: A Multicenter, Randomized, Double blind, Placebo controlled Study
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Enrollment
- 90
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •Inclusion Criteria -
- •1. Male or female =18 years of age.
- •2. Diagnosed with active primary or secondary wAIHA, defined as having
- •all of the following:
- •a.Hemoglobin level <9 g/dL or <10 g/dL if symptomatic or on
- •corticosteroids/immunosuppressants AND
- •b.Signs of hemolysis, defined as: lactate dehydrogenase (LDH) levels
- •above the upper limit of normal (ULN), or haptoglobin below the lower
- •limit of normal, or total bilirubin above the ULN AND
- •c.Serological evidence of anti-erythrocyte antibodies associated with a
- •positive DAT that is either positive for IgG only or is positive for IgG and
- •C3d (fragment of the third component of complement) at time of
- •3.Has been diagnosed with wAIHA for at least 3 months, has received
- •standard of care treatment for wAIHA but is not currently under
- •adequate control
- •4.If on corticosteroids, are on a dose that has been stable for at least 14
- •days prior to randomization.
- •5.If currently receiving immunosuppressants, has been on a stable dose
- •for 30 days prior to Screening. Allowed concomitant
- •immunosuppressants are azathioprine, mycophenolate
- •mofetil/mycophenolic acid, cyclosporine, cyclophosphamide.
- •6.Have a platelet count =30 × 109/L.
- •7.Have screening serum albumin and serum calcium concentrations
- •within the normal range.
- •8.Have screening total serum IgG of at least 600 mg/dL.
- •9.Have a screening creatine kinase (CK) value <2 × ULN.
- •10.Have a negative QuantiFERON®-TB Gold test.
- •11.Patients who have undergone splenectomy must be at least 3 months
- •post resection prior to screening and must be vaccinated as per the
- •United States Center for Disease Control and Prevention (CDC) annual
- •Recommended Immunization Schedule for Adults Aged 19 Years or
- •Older, United States
- •12.Patients with autoimmune disease (eg, systemic lupus erythematosus
- •or rheumatoid arthritis) and lymphoproliferative disorders may be
- •eligible if they are stable (no changes in concomitant disease-related
- •medications and severity of disease for at least 3 months). Patients with
- •lymphoproliferative disease must have a low grade, be stable and be, in
- •the opinion of the Investigator, unlikely to require chemotherapy or
- •monoclonal antibody therapy during the study. Patients requiring change
- •of treatment or new treatment (but not rescue therapy) during the study
- •will be terminated from the study
- •13.Have sufficient venous access to allow drug administration by IV
- •infusion and blood sampling as per the protocol.
- •14.Women of childbearing potential (WOCBP), 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 folliclestimulating
- •hormone (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
- +8 more not shown
Exclusion Criteria
- •Exclusion Criteria -
- •1.Have received a transfusion within 30 days prior to randomization.
- •2.Have any other associated cause of hereditary or acquired hemolytic
- •3.Have received rituximab within 6 months prior to randomization.
- •Note: Patients who received rituximab within 3 months but have
- •evidence of worsening hemolysis (defined as LDH levels above ULN or
- •haptoglobin levels below the lower limit of the normal range (LLN) or
- •total bilirubin levels above the ULN) may be included in the study.
- •4. Has received IVIG within 6 weeks prior to randomization on Day 1. The patient may be
- •re-screened after the exclusionary period of 6 weeks has passed.
- •5.Have cold antibody AIHA, cold agglutinin syndrome, mixed type (ie,
- •warm and cold) AIHA, or paroxysmal cold hemoglobinuria.
- •6.Have a severe infection (eg, pneumonia, biliary tract infection,
- •diverticulitis, Clostridium difficile 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. The patient may be re-screened after the 8 week exclusionary
- •period has passed. Any patient with an infection requiring oral antiinfectives
- •(eg, sinusitis, bronchitis, uncomplicated urinary tract
- •infection) within 4 weeks prior to screening will be excluded, but may be
- •subsequently re-screened after the 4 week exclusionary period has
- •7.Have a chronic infection (eg, bronchiectasis, chronic osteomyelitis,
- •chronic pyelonephritis) or require chronic treatment with anti-infectives
- •(eg, antibiotics, antivirals).
- •8.Have received a live vaccine within 3 months prior to screening, or
- •have a known need to receive a live vaccine during the study or within at
- •least 3 months after the last dose of study drug. The patient may be re
- •screened after the 3 month exclusionary period has ended.
- •9. Has any confirmed or suspected clinical immunodeficiency syndrome not related to
- •treatment of his/her wAIHA, or has a family history of congenital or hereditary
- •immunodeficiency unless confirmed absent in the patient.
- •10.Have a known history of, or positive test result for human
- •immunodeficiency virus-1 (HIV 1) and HIV 2 antibodies, hepatitis B virus
- •(HBV core antigen), or hepatitis C virus (HCV).
- •Note: Patients with past HCV may be included in the study if they have a
- •documented negative HCV ribonucleic acid level in the serum at 12
- •weeks or longer after the completion of HCV therapy.
- •11.Are currently breastfeeding, pregnant, intend to become pregnant
- •during the study, or are planning egg donation during the study or
- •within 30 days after the last dose of study drug.
- •12.Have current alcohol/substance abuse/dependence, a history of
- •alcohol/substance abuse/dependence within the 12 months prior to
- •screening, or, in the Investigator's opinion, show evidence of ongoing
- •alcohol/substance abuse/dependence.
- •13.Are currently participating in another interventional clinical trial or
- •have received any investigational drug within the past 3 months.
- •14.Have had any major surgery within 3 months prior to screening or
- •have plans for or have been scheduled for any elective surgery or major
- •dental procedure during the study.
- •15.Have a history of a major organ transplant (eg, heart, lung, kidney,
- +3 more not shown
Investigators
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