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

NCT05096403 | Completed Phase 3 Results FDA Drug

• 1 other identifier: Sobi.PEGCET-101
• Study Type: interventional
• Target: 24
• Locations: 14 countries
• Sites: 44

Brief 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).

Conditions

Cold Agglutinin Disease

Outcome Measures

Primary Outcomes (1)

• Number of Patients Achieving a Response (R) at Week 24

  A participant was considered to have a response if the Hgb level increased greater than or equal to (\>=) 1.5 gram per deciliter (g/dL) from baseline and this increase was maintained from Week 16 through Week 24 in absence of blood transfusion from Week 5 through Week 24.

  Week 24

Secondary Outcomes (32)

• Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs).

  Week 24

• Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A

  Week 24

• Change From Baseline to Week 24 in FACT-An Scale Score (Quality of Life)-Part A in the Absence of Intercurrent Events (ICEs)

  Week 24

• Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A

  Week 24

• Change From Baseline to Week 24 in LDH Levels-Part A in the Absence of Intercurrent Events (ICEs)

  Week 24

Study Arms (4)

Pegcetacoplan Double Blind During Part A

ACTIVE COMPARATOR

1080 mg, subcutaneous injection, twice weekly

Drug: Pegcetacoplan

Placebo Matching Pegcetacoplan-Double-blind During Part A

PLACEBO COMPARATOR

Sodium acetate, subcutaneous injection, twice weekly

Drug: Placebo matching Pegcetacoplan

Open-label Pegcetacoplan During Parts B and C

ACTIVE COMPARATOR

1080 mg, subcutaneous injection, twice weekly

Drug: Pegcetacoplan

Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C

PLACEBO COMPARATOR

Sodium acetate, subcutaneous injection, twice weekly

Drug: Placebo matching Pegcetacoplan

Interventions

Pegcetacoplan DRUG

Pegcetacoplan taken twice weekly as subcutaneous injection

Also known as: Aspaveli, Empaveli

Open-label Pegcetacoplan During Parts B and C; Pegcetacoplan Double Blind During Part A

Placebo matching Pegcetacoplan DRUG

Placebo matching pegcetacoplan taken twice weekly as subcutaneous injection

Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C; Placebo Matching Pegcetacoplan-Double-blind During Part A

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (44)

The Oncology Institute of Hope and Innovation

Whittier, California, 90603, United States

Location

Lakes Research

Miami Lakes, Florida, 33014, United States

Location

University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)

Iowa City, Iowa, 52242, United States

Location

Weill Cornell Medicine / NewYork Presbyterian Hospital

New York, New York, 10021, United States

Location

East Carolina University Division of Hematology/ Oncology

Greenville, North Carolina, 27858, United States

Location

Medical University

Vienna, Austria

Location

Algemeen Ziekenhuis Klina

Brasschaat, 2930, Belgium

Location

UZ Gasthuisberg

Leuven, 3000, Belgium

Location

CHU de Liège

Liège, 4000, Belgium

Location

St. Michael's Hospital

Toronto, Canada

Location

Helsinki University Hospital - Comprehensive Cancer Center

Helsinki, Finland

Location

"LTD Medinvest Institute of Hematology and Transfusiology "

Tbilisi, Georgia

Location

Ltd M. Zodelava Hematology Centre

Tbilisi, Georgia

Location

Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum

Essen, 45147, Germany

Location

Institut f. Transfusionsmedizin - Universität Ulm

Ulm, Germany

Location

Semmelweis Egyetem

Budapest, Hungary

Location

A.O.R.N. S.G. Moscati di Avellino

Avellino, 83100, Italy

Location

ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia

Brescia, 25123, Italy

Location

"FOND IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Italy

Location

AOU Maggiore della Carità SCDU Ematologia

Novara, Italy

Location

Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello

Palermo, 90146, Italy

Location

Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii

Reggio Calabria, 89133, Italy

Location

Fukushima Medical University Hospital

Fukushima, Japan

Location

University of Tsukuba Hospital

Ibaraki, Japan

Location

Ishikawa Prefectural Central Hospital

Kanazawa, 9208530, Japan

Location

Aichi Medical University Hospital

Nagakute, 480-1195, Japan

Location

Shinshu University Hospital

Nagano, Japan

Location

Osaka University Hospital

Osaka, 5650871, Japan

Location

Amsterdam UMC

Amsterdam, Netherlands

Location

Haukeland University Hospital

Bergen, Norway

Location

Sykehuset Østfold Kalnes

Grålum, Norway

Location

St Olavs Hospital, Avdeling for blodsykdommer

Trondheim, Norway

Location

Hospital Clinic de Barcelona

Barcelona, Spain

Location

Hospital Universitario de Gran Canaria Dr. Negrin

Las Palmas de Gran Canaria, Spain

Location

Hospital Clínico Universitario de Salamanca

Madrid, Spain

Location

Hospital Universitario Infanta Leonor

Madrid, Spain

Location

Hospital Universitario La Paz

Madrid, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, Spain

Location

Hospital Universitario y Politecnico La Fe

Valencia, Spain

Location

St James' University Hospital

Leeds, LS9 7TF, United Kingdom

Location

Royal London Hospital

London, E1 2ES, United Kingdom

Location

Cancer Clinical Trials Unit, Haematology -University College London -

London, United Kingdom

Location

Russell Centre for Clinical Haematology

Nottingham, NG5 1PB, United Kingdom

Location

Churchill Hospital

Oxford, United Kingdom

Location

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Interventions

pegcetacoplan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Luis López Lazaro
• Organization: Swedish Orphan Biovitrum AB

luis.lopezlazaro@sobi.com

46733319155

Study Officials

• Study physician

  Swedish Orphan Biovitrum AB

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2021
• First Posted: October 27, 2021
• Study Start: October 20, 2022
• Primary Completion: May 27, 2024
• Study Completion: September 11, 2024
• Last Updated: October 30, 2025
• Results First Posted: October 30, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

NO (3); HU (1); ES (7); GB (5); CA (1); BE (3); IT (6); US (5); GE (2); JP (6); NL (1); FI (1); DE (2); AU (1)