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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05096403
Recruitment Status : Recruiting
First Posted : October 27, 2021
Last Update Posted : June 2, 2023
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

Study Description
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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).

Condition or disease Intervention/treatment Phase
Cold Agglutinin Disease Drug: Pegcetacoplan Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 57 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : October 20, 2022
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : April 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Pegcetacoplan
1080 mg, subcutaneus injection, twice weekly
 Drug: Pegcetacoplan
Pegcetacoplan taken twice weekly as subcutaneous injection
Placebo Comparator: Placebo
Sodium acetate, subcutaneus injection, twice weekly
 Drug: Pegcetacoplan
Pegcetacoplan taken twice weekly as subcutaneous injection


Outcome Measures
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Primary Outcome Measures :
  1. Response (R) [ Time Frame: Week 24 ]
    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 :
  1. Change from Baseline to Week 24 in Hemoglobin (Hgb) level. [ Time Frame: Week 24 ]
    Mean change from Baseline to Week 24 in Hemoglobin (Hgb) level will be assessed

  2. Transfusion avoidance from Week 5 to Week 24 [ Time Frame: Week 24 ]
    Percentage of patients who did not received a blood transfusion between Week 5 and Week 24 will be assessed

  3. Change From Baseline to Week 24 in Functional Assessment of Cancer Therapy-Anemia/Fatigue (FACT-An) Scale Score (Quality of Life) [ Time Frame: Week 24 ]
    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.

  4. Number of PRBC transfusions from Week 5 to Week 24. [ Time Frame: Week 24 ]
    Number of blood transfusions received between Week 5 and Week 24 will be assessed

  5. Change from Baseline to Week 24 in markers of hemolysis [ Time Frame: Week 24 ]
    Mean change from baseline to Week 24 in Lactate dehydrogenase (LDH) level; Haptoglobin level; Indirect bilirubin level; Absolute reticulocyte counts (ARC).

  6. Normalization of markers of hemolysis at Week 24 [ Time Frame: Week 24 ]
    Percentage of patients with LDH level; Indirect bilirubin level and ARC within normal ranges.

  7. Change From Baseline to Week 24 in 12-item short form survey (SF-12) [ Time Frame: 24 weeks ]
    SF-12 health survey is a self-reported questionnaire to measure participant's profile of functional health and well-being. It includes 12 questions.

  8. Change From Baseline to Week 24 in five level EuroQol five dimensions questionnaire (EQ-5D-5L) [ Time Frame: Week 24 ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05096403


Contacts
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Contact: Luis López Lazaro, MD +41 79 834 17 78 Luis.LopezLazaro@sobi.com
Contact: Bodil Svanberg +46733319155 Bodil.Svanberg@sobi.com

Locations
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Sponsors and Collaborators
Swedish Orphan Biovitrum
Investigators
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Principal Investigator: Bernd Jilma, MD Medical University of Vienna
More Information
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Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT05096403    
Other Study ID Numbers: Sobi.PEGCET-101
First Posted: October 27, 2021    Key Record Dates
Last Update Posted: June 2, 2023
Last Verified: June 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Hemolytic, Autoimmune
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Autoimmune Diseases
Immune System Diseases