Efficacy and Safety of M281 in Adults with Warm Autoimmune Hemolytic Anemia

Phase 1

• Conditions: MedDRA version: 20.1Level: LLTClassification code 10002285Term: Anemia hemolytic autoimmune (NOS)System Organ Class: 100000004851; Adults with Warm Autoimmune Hemolytic Anemia; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2019-000720-17-FR
• Lead Sponsor: Momenta Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-17
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 90

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
diagnosis.
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
pregnancy test at Baseline to be eligible.
15.Women of childbearing potential (including menopausal women who
do not have elevated FSH) must agree to remain totally abstinent (ie,
refrain from sexual intercourse during the study) or to consistently use a
reliable and highly effective method of contraception (eg, condom plus
diaphragm, condom plus spermicide, diaphragm plus spermicide, or
intrauterine device or oral/injectable/implanted hormonal contraceptive
used in combination with an additional barrier method) during the

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
anemia.
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
passed.
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,
liver), or hematopoietic stem cell/marrow transplant.
16.Have a history of severe and/or uncontrolled hepatic,
gastrointestinal, renal, pulmonary, cardiovascular, psychiatric,

Study & Design

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