A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Dis… (NCT05096403) | Clinical Trial Compass
CompletedPhase 3
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
United States24 participantsStarted 2022-10-20
Plain-language 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).
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 investigational medicinal product (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.
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.
✕
What they're measuring
1
Number of Patients Achieving a Response (R) at Week 24
. 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 upper limit of normal (ULN), or direct bilirubin levels \> 2 x ULN.
✕. Inability to cooperate with study procedures.