A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
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|ClinicalTrials.gov Identifier: NCT05096403|
Recruitment Status : Recruiting
First Posted : October 27, 2021
Last Update Posted : June 2, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Cold Agglutinin Disease||Drug: Pegcetacoplan||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||57 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
Active Comparator: Pegcetacoplan
1080 mg, subcutaneus injection, twice weekly
Pegcetacoplan taken twice weekly as subcutaneous injection
Placebo Comparator: Placebo
Sodium acetate, subcutaneus injection, twice weekly
Pegcetacoplan taken twice weekly as subcutaneous injection
- 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.
- 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
- 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
- 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.
- 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
- 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).
- 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.
- 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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
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
|Contact: Luis López Lazaro, MD||+41 79 834 17 78||Luis.LopezLazaro@sobi.com|
|Contact: Bodil Svanberg||+46733319155||Bodil.Svanberg@sobi.com|
|Principal Investigator:||Bernd Jilma, MD||Medical University of Vienna|
|Responsible Party:||Swedish Orphan Biovitrum|
|Other Study ID Numbers:||
|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|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Anemia, Hemolytic, Autoimmune
Immune System Diseases