A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
NCT ID: NCT05096403
Last Updated: 2025-10-30
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
24 participants
INTERVENTIONAL
2022-10-20
2024-09-11
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Pegcetacoplan Double Blind During Part A
1080 mg, subcutaneous injection, twice weekly
Pegcetacoplan
Pegcetacoplan taken twice weekly as subcutaneous injection
Placebo Matching Pegcetacoplan-Double-blind During Part A
Sodium acetate, subcutaneous injection, twice weekly
Placebo matching Pegcetacoplan
Placebo matching pegcetacoplan taken twice weekly as subcutaneous injection
Open-label Pegcetacoplan During Parts B and C
1080 mg, subcutaneous injection, twice weekly
Pegcetacoplan
Pegcetacoplan taken twice weekly as subcutaneous injection
Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C
Sodium acetate, subcutaneous injection, twice weekly
Placebo matching Pegcetacoplan
Placebo matching pegcetacoplan taken twice weekly as subcutaneous injection
Interventions
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Pegcetacoplan
Pegcetacoplan taken twice weekly as subcutaneous injection
Placebo matching Pegcetacoplan
Placebo matching pegcetacoplan taken twice weekly as subcutaneous injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of primary CAD.
3. Hb level ≤ 9 g/dL.
4. Documented results from bone marrow biopsy within 1 year of screening
5. 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.
6. 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.
7. Men must agree to the following for the duration of the study and 8 weeks after their last IMP dose:
1. Avoid fathering a child.
2. Use protocol-defined methods of contraception.
3. Refrain from donating sperm.
8. Willing and able to give written informed consent.
Exclusion Criteria
2. 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.
3. Diagnosis of systemic lupus erythematosus or other autoimmune diseases with antinuclear antibodies.
4. History of an aggressive lymphoma or presence of a lymphoma requiring therapy.
5. Have received an organ transplant.
6. Cold agglutinin syndrome secondary to Mycoplasma pneumoniae, Epstein-Barr virus or other specific causative infection.
7. 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.
8. Inability to cooperate with study procedures.
18 Years
ALL
No
Sponsors
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Swedish Orphan Biovitrum
INDUSTRY
Responsible Party
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Principal Investigators
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Study physician
Role: STUDY_DIRECTOR
Swedish Orphan Biovitrum AB
Locations
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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
Fukushima Medical University Hospital
Fukushima, , Japan
University of Tsukuba Hospital
Ibaraki, , Japan
Ishikawa Prefectural Central Hospital
Kanazawa, , Japan
Aichi Medical University Hospital
Nagakute, , Japan
Shinshu University Hospital
Nagano, , Japan
Osaka University Hospital
Osaka, , 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
Seville, , Spain
Hospital Universitario y Politecnico La Fe
Valencia, , Spain
St James' University Hospital
Leeds, , United Kingdom
Royal London Hospital
London, , United Kingdom
Cancer Clinical Trials Unit, Haematology -University College London -
London, , United Kingdom
Russell Centre for Clinical Haematology
Nottingham, , United Kingdom
Churchill Hospital
Oxford, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol: Clinical Study Protocol v2.0
Document Type: Study Protocol: Clinical Study Protocol v4.0
Document Type: Study Protocol: Clinical Study Protocol v3.0
Document Type: Study Protocol: Clinical Study Protocol v5.0
Document Type: Study Protocol: Clinical Study Protocol v1.0
Document Type: Statistical Analysis Plan: Statistical Analysis Plan v2.0
Document Type: Statistical Analysis Plan: Statistical Analysis Plan v1.0
Other Identifiers
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Sobi.PEGCET-101
Identifier Type: -
Identifier Source: org_study_id
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