A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)

Completed · Phase 3 · Has a placebo group

Conditions studied: Cold Agglutinin Disease

In brief

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).

Key facts

Study ID

NCT05096403

Run by

Swedish Orphan Biovitrum

People needed

24

Starts

2022-10-20

Expected to finish

2024-09-11

Last updated by the study team

2025-10-30

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• Use protocol-defined methods of contraception.
• Refrain from donating sperm.
• Willing and able to give written informed consent.

You may not qualify if…

• 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 upper limit of normal (ULN), or direct bilirubin levels > 2 x ULN.
• Inability to cooperate with study procedures.

Where it is running

• Osaka University Hospital — Osaka, Japan
• Amsterdam UMC — Amsterdam, Netherlands
• Haukeland University Hospital — Bergen, Norway
• The Oncology Institute of Hope and Innovation — Whittier, California, United States
• Lakes Research — Miami Lakes, Florida, United States
• University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC) — Iowa City, Iowa, United States
• Weill Cornell Medicine / NewYork Presbyterian Hospital — New York, New York, United States
• East Carolina University Division of Hematology/ Oncology — Greenville, North Carolina, United States
• Medical University — Vienna, Austria
• Algemeen Ziekenhuis Klina — Brasschaat, Belgium
• UZ Gasthuisberg — Leuven, Belgium
• CHU de Liège — Liège, Belgium
• St. Michael's Hospital — Toronto, Canada
• Helsinki University Hospital - Comprehensive Cancer Center — Helsinki, Finland
• "LTD Medinvest Institute of Hematology and Transfusiology " — Tbilisi, Georgia
• Ltd M. Zodelava Hematology Centre — Tbilisi, Georgia
• Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum — Essen, Germany
• Institut f. Transfusionsmedizin - Universität Ulm — Ulm, Germany
• Semmelweis Egyetem — Budapest, Hungary
• A.O.R.N. S.G. Moscati di Avellino — Avellino, Italy
• ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia — Brescia, Italy
• "FOND IRCCS Cà Granda Ospedale Maggiore Policlinico — Milan, Italy
• AOU Maggiore della Carità SCDU Ematologia — Novara, Italy
• Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello — Palermo, Italy
• Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii — Reggio Calabria, Italy

Full record on ClinicalTrials.gov

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