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

Primary Purpose

Cold Agglutinin Disease

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Pegcetacoplan
Sponsored by
Swedish Orphan Biovitrum
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cold Agglutinin Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Age 18 years or older.
  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 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:

  1. Have received other anti-complement therapies (approved or investigational) within 5 half-lives of the agent prior to randomization.
  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 ULN, or direct bilirubin levels > 2 x ULN.
  8. Inability to cooperate with study procedures.

Sites / Locations

  • The Oncology Institute of Hope and InnovationRecruiting
  • Lakes ResearchRecruiting
  • University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)
  • Weill Cornell Medicine / NewYork Presbyterian Hospital
  • East Carolina University Division of Hematology/ Oncology
  • Medical UniversityRecruiting
  • Algemeen Ziekenhuis Klina
  • UZ GasthuisbergRecruiting
  • CHU de Liège
  • St. Michael's Hospital
  • Helsinki University Hospital - Comprehensive Cancer Center
  • "LTD Medinvest Institute of Hematology and Transfusiology "Recruiting
  • Ltd M. Zodelava Hematology CentreRecruiting
  • Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum
  • Institut f. Transfusionsmedizin - Universität Ulm
  • Semmelweis Egyetem
  • A.O.R.N. S.G. Moscati di Avellino
  • ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia
  • "FOND IRCCS Cà Granda Ospedale Maggiore PoliclinicoRecruiting
  • AOU Maggiore della Carità SCDU Ematologia
  • Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello
  • Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii
  • Fukushima Medical University Hospital
  • University of Tsukuba HospitalRecruiting
  • Ishikawa Prefectural Central HospitalRecruiting
  • Aichi Medical University HospitalRecruiting
  • Shinshu University HospitalRecruiting
  • Osaka University HospitalRecruiting
  • Amsterdam UMCRecruiting
  • Haukeland University Hospital
  • Sykehuset Østfold Kalnes
  • St Olavs Hospital, Avdeling for blodsykdommerRecruiting
  • Hospital Clinic de BarcelonaRecruiting
  • Hospital Universitario de Gran Canaria Dr. Negrin
  • Hospital Clínico Universitario de Salamanca
  • Hospital Universitario Infanta Leonor
  • Hospital Universitario La Paz
  • Hospital Universitario Virgen del Rocio
  • Hospital Universitario y Politecnico La Fe
  • St James' University HospitalRecruiting
  • Royal London Hospital
  • Cancer Clinical Trials Unit, Haematology -University College London -Recruiting
  • Russell Centre for Clinical Haematology
  • Churchill HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Pegcetacoplan

Placebo

Arm Description

1080 mg, subcutaneus injection, twice weekly

Sodium acetate, subcutaneus injection, twice weekly

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change from Baseline to Week 24 in Hemoglobin (Hgb) level.
Mean change from Baseline to Week 24 in Hemoglobin (Hgb) level will be assessed
Transfusion avoidance from Week 5 to Week 24
Percentage of patients who did not received a blood transfusion between Week 5 and Week 24 will be assessed
Change From Baseline to Week 24 in Functional Assessment of Cancer Therapy-Anemia/Fatigue (FACT-An) Scale Score (Quality of Life)
FACT-An consists consists of 33 questions related to general quality of life and to the impact of fatigue and other anemia-related symptoms assessed using a 5 point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question are added to obtain a total score.
Number of PRBC transfusions from Week 5 to Week 24.
Number of blood transfusions received between Week 5 and Week 24 will be assessed
Change from Baseline to Week 24 in markers of hemolysis
Mean change from baseline to Week 24 in Lactate dehydrogenase (LDH) level; Haptoglobin level; Indirect bilirubin level; Absolute reticulocyte counts (ARC).
Normalization of markers of hemolysis at Week 24
Percentage of patients with LDH level; Indirect bilirubin level and ARC within normal ranges.
Change From Baseline to Week 24 in 12-item short form survey (SF-12)
SF-12 health survey is a self-reported questionnaire to measure participant's profile of functional health and well-being. It includes 12 questions.
Change From Baseline to Week 24 in five level EuroQol five dimensions questionnaire (EQ-5D-5L)
The EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has a 5-level response: no problems, slight problems, moderate problems, severe problems, and extreme problems. A scale with score 0-100 is used to collect response on current health status.

Full Information

First Posted
October 15, 2021
Last Updated
June 1, 2023
Sponsor
Swedish Orphan Biovitrum
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1. Study Identification

Unique Protocol Identification Number
NCT05096403
Brief Title
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
Official Title
A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 20, 2022 (Actual)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
April 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Swedish Orphan Biovitrum

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cold Agglutinin Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
57 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Pegcetacoplan
Arm Type
Active Comparator
Arm Description
1080 mg, subcutaneus injection, twice weekly
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Sodium acetate, subcutaneus injection, twice weekly
Intervention Type
Drug
Intervention Name(s)
Pegcetacoplan
Intervention Description
Pegcetacoplan taken twice weekly as subcutaneous injection
Primary Outcome Measure Information:
Title
Response (R)
Description
A participant will be considered to have a response if the Hgb level increases greater than or equal to (>=) 1.5 gram per deciliter (g/dL) from baseline and this increase is maintained from Week 16 through Week 24 in absence of blood transfusion from Week 5 through Week 24.
Time Frame
Week 24
Secondary Outcome Measure Information:
Title
Change from Baseline to Week 24 in Hemoglobin (Hgb) level.
Description
Mean change from Baseline to Week 24 in Hemoglobin (Hgb) level will be assessed
Time Frame
Week 24
Title
Transfusion avoidance from Week 5 to Week 24
Description
Percentage of patients who did not received a blood transfusion between Week 5 and Week 24 will be assessed
Time Frame
Week 24
Title
Change From Baseline to Week 24 in Functional Assessment of Cancer Therapy-Anemia/Fatigue (FACT-An) Scale Score (Quality of Life)
Description
FACT-An consists consists of 33 questions related to general quality of life and to the impact of fatigue and other anemia-related symptoms assessed using a 5 point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question are added to obtain a total score.
Time Frame
Week 24
Title
Number of PRBC transfusions from Week 5 to Week 24.
Description
Number of blood transfusions received between Week 5 and Week 24 will be assessed
Time Frame
Week 24
Title
Change from Baseline to Week 24 in markers of hemolysis
Description
Mean change from baseline to Week 24 in Lactate dehydrogenase (LDH) level; Haptoglobin level; Indirect bilirubin level; Absolute reticulocyte counts (ARC).
Time Frame
Week 24
Title
Normalization of markers of hemolysis at Week 24
Description
Percentage of patients with LDH level; Indirect bilirubin level and ARC within normal ranges.
Time Frame
Week 24
Title
Change From Baseline to Week 24 in 12-item short form survey (SF-12)
Description
SF-12 health survey is a self-reported questionnaire to measure participant's profile of functional health and well-being. It includes 12 questions.
Time Frame
24 weeks
Title
Change From Baseline to Week 24 in five level EuroQol five dimensions questionnaire (EQ-5D-5L)
Description
The EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has a 5-level response: no problems, slight problems, moderate problems, severe problems, and extreme problems. A scale with score 0-100 is used to collect response on current health status.
Time Frame
Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Luis López Lazaro, MD
Phone
+41 79 834 17 78
Email
Luis.LopezLazaro@sobi.com
First Name & Middle Initial & Last Name or Official Title & Degree
Bodil Svanberg
Phone
+46733319155
Email
Bodil.Svanberg@sobi.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bernd Jilma, MD
Organizational Affiliation
Medical University of Vienna
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Oncology Institute of Hope and Innovation
City
Whittier
State/Province
California
ZIP/Postal Code
90603
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Merrill Shum, MD
Phone
562-693-4477
Email
mshum@airesearch.us
First Name & Middle Initial & Last Name & Degree
Kirsten Bettino
Phone
5626934477
Email
kbettino@airesearch.us
Facility Name
Lakes Research
City
Miami Lakes
State/Province
Florida
ZIP/Postal Code
33014
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eloy Roman, MD
Phone
786-362-5763
Email
eRoman@lakesresearch.com
First Name & Middle Initial & Last Name & Degree
Yamile Sanchez
Phone
786-362-5763
Ext
121
Email
ysanchez@lakesresearch.com
Facility Name
University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Weill Cornell Medicine / NewYork Presbyterian Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
East Carolina University Division of Hematology/ Oncology
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27858
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Medical University
City
Vienna
Country
Austria
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Bernd Jilma, Prof. Dr.
Facility Name
Algemeen Ziekenhuis Klina
City
Brasschaat
ZIP/Postal Code
2930
Country
Belgium
Individual Site Status
Active, not recruiting
Facility Name
UZ Gasthuisberg
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dierickx, MD
Facility Name
CHU de Liège
City
Liège
ZIP/Postal Code
4000
Country
Belgium
Individual Site Status
Active, not recruiting
Facility Name
St. Michael's Hospital
City
Toronto
Country
Canada
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pavenski, MD
Facility Name
Helsinki University Hospital - Comprehensive Cancer Center
City
Helsinki
Country
Finland
Individual Site Status
Active, not recruiting
Facility Name
"LTD Medinvest Institute of Hematology and Transfusiology "
City
Tbilisi
Country
Georgia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Iosava, MD
Facility Name
Ltd M. Zodelava Hematology Centre
City
Tbilisi
Country
Georgia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zodelava, MD
Facility Name
Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum
City
Essen
ZIP/Postal Code
45147
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Institut f. Transfusionsmedizin - Universität Ulm
City
Ulm
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Semmelweis Egyetem
City
Budapest
Country
Hungary
Individual Site Status
Active, not recruiting
Facility Name
A.O.R.N. S.G. Moscati di Avellino
City
Avellino
ZIP/Postal Code
83100
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia
City
Brescia
ZIP/Postal Code
25123
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
"FOND IRCCS Cà Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Fattizzo, MD
Facility Name
AOU Maggiore della Carità SCDU Ematologia
City
Novara
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello
City
Palermo
ZIP/Postal Code
90146
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii
City
Reggio Calabria
ZIP/Postal Code
89133
Country
Italy
Individual Site Status
Active, not recruiting
Facility Name
Fukushima Medical University Hospital
City
Fukushima
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
University of Tsukuba Hospital
City
Ibaraki
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Obara, MD
Facility Name
Ishikawa Prefectural Central Hospital
City
Kanazawa
ZIP/Postal Code
9208530
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yamaguchi, MD
Facility Name
Aichi Medical University Hospital
City
Nagakute
ZIP/Postal Code
480-1195
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Takami, MD
Facility Name
Shinshu University Hospital
City
Nagano
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nakazawa, MD
Facility Name
Osaka University Hospital
City
Osaka
ZIP/Postal Code
5650871
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ueda, MD
Facility Name
Amsterdam UMC
City
Amsterdam
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Vos, MD
Facility Name
Haukeland University Hospital
City
Bergen
Country
Norway
Individual Site Status
Active, not recruiting
Facility Name
Sykehuset Østfold Kalnes
City
Grålum
Country
Norway
Individual Site Status
Active, not recruiting
Facility Name
St Olavs Hospital, Avdeling for blodsykdommer
City
Trondheim
Country
Norway
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hansen, MD
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cid Vidal, MD
Facility Name
Hospital Universitario de Gran Canaria Dr. Negrin
City
Las Palmas De Gran Canaria
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Clínico Universitario de Salamanca
City
Madrid
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Universitario Infanta Leonor
City
Madrid
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Universitario La Paz
City
Madrid
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Hospital Universitario y Politecnico La Fe
City
Valencia
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
St James' University Hospital
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hill, MD
Facility Name
Royal London Hospital
City
London
ZIP/Postal Code
E1 2ES
Country
United Kingdom
Individual Site Status
Active, not recruiting
Facility Name
Cancer Clinical Trials Unit, Haematology -University College London -
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
D'Sa, MD
Facility Name
Russell Centre for Clinical Haematology
City
Nottingham
ZIP/Postal Code
NG5 1PB
Country
United Kingdom
Individual Site Status
Active, not recruiting
Facility Name
Churchill Hospital
City
Oxford
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Desborough, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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