Cold Agglutinin Disease Clinical Trial
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
Summary
The purpose of the study is to determine the efficacy of pegcetacoplan administration compared to placebo in increasing hemoglobin (Hgb) level from baseline and avoiding transfusion in participants with primary cold agglutinin disease (CAD).
Eligibility Criteria
Inclusion Criteria:
Age 18 years or older.
Diagnosis of primary CAD.
Hb level ≤ 9 g/dL.
Documented results from bone marrow biopsy within 1 year of screening
Either have vaccination against Streptococcus pneumoniae, Neisseria meningitidis (Types A, C, W, Y, and B), and Haemophilus influenzae (Type B) within 2 years prior to screening or agree to receive vaccination during screening.
Women of childbearing potential (WOCBP), defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last IMP dose.
Men must agree to the following for the duration of the study and 8 weeks after their last IMP dose:
Avoid fathering a child.
Use protocol-defined methods of contraception.
Refrain from donating sperm.
Willing and able to give written informed consent.
Exclusion Criteria:
Have received other anti-complement therapies (approved or investigational) within 5 half-lives of the agent prior to randomization.
Treatment with rituximab monotherapy within 12 weeks prior to randomization, or rituximab combination therapies (e.g., with bendamustine, fludarabine, other cytotoxic drugs or ibrutinib) within 16 weeks prior to randomization.
Diagnosis of systemic lupus erythematosus or other autoimmune diseases with antinuclear antibodies.
History of an aggressive lymphoma or presence of a lymphoma requiring therapy.
Have received an organ transplant.
Cold agglutinin syndrome secondary to Mycoplasma pneumoniae, Epstein-Barr virus or other specific causative infection.
Presence or suspicion of liver dysfunction as indicated by elevated alanine aminotransferase (ALT) > 2.5 x ULN, or direct bilirubin levels > 2 x ULN.
Inability to cooperate with study procedures.
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There are 19 Locations for this study
Whittier California, 90603, United States
Miami Lakes Florida, 33014, United States
Iowa City Iowa, 52242, United States
New York New York, 10021, United States
Greenville North Carolina, 27858, United States
Vienna , , Austria
Brasschaat , 2930, Belgium
Leuven , 3000, Belgium
Liège , 4000, Belgium
Toronto , , Canada
Helsinki , , Finland
Tbilisi , , Georgia
Tbilisi , , Georgia
Essen , 45147, Germany
Ulm , , Germany
Budapest , , Hungary
Avellino , 83100, Italy
Brescia , 25123, Italy
Milano , , Italy
Novara , , Italy
Palermo , 90146, Italy
Reggio Calabria , 89133, Italy
Fukushima , , Japan
Ibaraki , , Japan
Kanazawa , 92085, Japan
Nagakute , 480-1, Japan
Nagano , , Japan
Osaka , 56508, Japan
Amsterdam , , Netherlands
Bergen , , Norway
Grålum , , Norway
Trondheim , , Norway
Barcelona , , Spain
Las Palmas De Gran Canaria , , Spain
Madrid , , Spain
Madrid , , Spain
Madrid , , Spain
Sevilla , , Spain
Valencia , , Spain
Leeds , LS9 7, United Kingdom
London , E1 2E, United Kingdom
London , , United Kingdom
Nottingham , NG5 1, United Kingdom
Oxford , , United Kingdom
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