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).

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Full Description

This is a blind (actual treatment not disclosed to Investigator or participant) study to study pegcetacoplan in people with cold agglutinin disease. The study will consist of a 4-week screening period where selected tests will be conducted to ensure that the patient is eligible to participate in the study, followed by Part A, a 24-week blinded treatment period where the participants will receive either pegcetacoplan or a placebo treatment, looking like pegcetacoplan but with no effect. After this period, the participants will move into Part B, a 24-week period where they will all receive pegcetacoplan. Part C is a 48-week maintenance period with pegcetacoplan for all participants. After the end of treatment participants will undergo a safety follow visit about 8 weeks after last dose.

All eligible study participants will receive pegcetacoplan or placebo treatment, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.

View Eligibility Criteria

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.

Study is for people with:

Cold Agglutinin Disease

Phase:

Phase 3

Estimated Enrollment:

57

Study ID:

NCT05096403

Recruitment Status:

Suspended

Sponsor:

Swedish Orphan Biovitrum

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There are 19 Locations for this study

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The Oncology Institute of Hope and Innovation
Whittier California, 90603, United States
Lakes Research
Miami Lakes Florida, 33014, United States
University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)
Iowa City Iowa, 52242, United States
Weill Cornell Medicine / NewYork Presbyterian Hospital
New York New York, 10021, United States
East Carolina University Division of Hematology/ Oncology
Greenville North Carolina, 27858, United States
Medical University
Vienna , , Austria
Algemeen Ziekenhuis Klina
Brasschaat , 2930, Belgium
UZ Gasthuisberg
Leuven , 3000, Belgium
CHU de Liège
Liège , 4000, 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 , 45147, Germany
Institut f. Transfusionsmedizin - Universität Ulm
Ulm , , Germany
Semmelweis Egyetem
Budapest , , Hungary
A.O.R.N. S.G. Moscati di Avellino
Avellino , 83100, Italy
ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia
Brescia , 25123, Italy
"FOND IRCCS Cà Granda Ospedale Maggiore Policlinico
Milano , , Italy
AOU Maggiore della Carità SCDU Ematologia
Novara , , Italy
Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello
Palermo , 90146, Italy
Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii
Reggio Calabria , 89133, Italy
Fukushima Medical University Hospital
Fukushima , , Japan
University of Tsukuba Hospital
Ibaraki , , Japan
Ishikawa Prefectural Central Hospital
Kanazawa , 92085, Japan
Aichi Medical University Hospital
Nagakute , 480-1, Japan
Shinshu University Hospital
Nagano , , Japan
Osaka University Hospital
Osaka , 56508, Japan
Amsterdam UMC
Amsterdam , , Netherlands
Haukeland University Hospital
Bergen , , Norway
Sykehuset Østfold Kalnes
Grålum , , Norway
St Olavs Hospital, Avdeling for blodsykdommer
Trondheim , , Norway
Hospital Clinic de Barcelona
Barcelona , , Spain
Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas De Gran Canaria , , Spain
Hospital Clínico Universitario de Salamanca
Madrid , , Spain
Hospital Universitario Infanta Leonor
Madrid , , Spain
Hospital Universitario La Paz
Madrid , , Spain
Hospital Universitario Virgen del Rocio
Sevilla , , Spain
Hospital Universitario y Politecnico La Fe
Valencia , , Spain
St James' University Hospital
Leeds , LS9 7, United Kingdom
Royal London Hospital
London , E1 2E, United Kingdom
Cancer Clinical Trials Unit, Haematology -University College London -
London , , United Kingdom
Russell Centre for Clinical Haematology
Nottingham , NG5 1, United Kingdom
Churchill Hospital
Oxford , , United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Cold Agglutinin Disease

Phase:

Phase 3

Estimated Enrollment:

57

Study ID:

NCT05096403

Recruitment Status:

Suspended

Sponsor:


Swedish Orphan Biovitrum

How clear is this clinincal trial information?

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