Cold Agglutinin DiseasePhase 3NCT05096403
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
A Phase 3 study testing Pegcetacoplan in people with Cold Agglutinin Disease, run by Swedish Orphan Biovitrum at 44 sites in 14 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cold Agglutinin Disease
- What is being tested?
- Pegcetacoplan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 44 sitesin 14 countries
- Study dates
- 20 Oct 2022to 11 Sep 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
44 sites in 14 countries.
44 sites in 14 countries
Spain7 sites
Hospital Clinic de Barcelona
Barcelona
Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas de Gran Canaria
Hospital Clínico Universitario de Salamanca
Madrid
Hospital Universitario Infanta Leonor
Madrid
Hospital Universitario La Paz
Madrid
Hospital Universitario Virgen del Rocio
Seville
Hospital Universitario y Politecnico La Fe
Valencia
Italy6 sites
A.O.R.N. S.G. Moscati di Avellino
Avellino, 83100
ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia
Brescia, 25123
"FOND IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan
AOU Maggiore della Carità SCDU Ematologia
Novara
Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello
Palermo, 90146
Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii
Reggio Calabria, 89133
Japan6 sites
Fukushima Medical University Hospital
Fukushima
University of Tsukuba Hospital
Ibaraki
Ishikawa Prefectural Central Hospital
Kanazawa, 9208530
Aichi Medical University Hospital
Nagakute, 480-1195
Shinshu University Hospital
Nagano
Osaka University Hospital
Osaka, 5650871
United States5 sites
East Carolina University Division of Hematology/ Oncology
Greenville, North Carolina, 27858
University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)
Iowa City, Iowa, 52242
Lakes Research
Miami Lakes, Florida, 33014
Weill Cornell Medicine / NewYork Presbyterian Hospital
New York, New York, 10021
The Oncology Institute of Hope and Innovation
Whittier, California, 90603
United Kingdom5 sites
St James' University Hospital
Leeds, LS9 7TF
Royal London Hospital
London, E1 2ES
Cancer Clinical Trials Unit, Haematology -University College London -
London
Russell Centre for Clinical Haematology
Nottingham, NG5 1PB
Churchill Hospital
Oxford
Belgium3 sites
Algemeen Ziekenhuis Klina
Brasschaat, 2930
UZ Gasthuisberg
Leuven, 3000
CHU de Liège
Liège, 4000
Norway3 sites
Haukeland University Hospital
Bergen
Sykehuset Østfold Kalnes
Grålum
St Olavs Hospital, Avdeling for blodsykdommer
Trondheim
Georgia2 sites
"LTD Medinvest Institute of Hematology and Transfusiology "
Tbilisi
Ltd M. Zodelava Hematology Centre
Tbilisi
Germany2 sites
Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum
Essen, 45147
Institut f. Transfusionsmedizin - Universität Ulm
Ulm
Finland1 site
Helsinki University Hospital - Comprehensive Cancer Center
Helsinki
About this study
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)
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).
Read the detailed description
This is a blind (actual treatment not disclosed to Investigator or participant) study to study pegcetacoplan in people with cold agglutinin disease. The study will consist of a 4-week screening period where selected tests will be conducted to ensure that the patient is eligible to participate in the study, followed by Part A, a 24-week blinded treatment period where the participants will receive either pegcetacoplan or a placebo treatment, looking like pegcetacoplan but with no effect. After this period, the participants will move into Part B, a 24-week period where they will all receive pegcetacoplan. Part C is a 48-week maintenance period with pegcetacoplan for all participants. After the end of treatment participants will undergo a safety follow visit about 8 weeks after last dose.
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
- Cold Agglutinin Disease
- Being tested
- Pegcetacoplan, Placebo matching Pegcetacoplan
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegcetacoplanDrug
Also called Aspaveli, Empaveli
Pegcetacoplan taken twice weekly as subcutaneous injection
Placebo matching PegcetacoplanDrug
Placebo matching pegcetacoplan taken twice weekly as subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Pegcetacoplan Double Blind During Part A | Active comparator | 1080 mg, subcutaneous injection, twice weekly Receives: Drug: Pegcetacoplan |
| Placebo Matching Pegcetacoplan-Double-blind During Part A | Placebo comparator | Sodium acetate, subcutaneous injection, twice weekly Receives: Drug: Placebo matching Pegcetacoplan |
| Open-label Pegcetacoplan During Parts B and C | Active comparator | 1080 mg, subcutaneous injection, twice weekly Receives: Drug: Pegcetacoplan |
| Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C | Placebo comparator | Sodium acetate, subcutaneous injection, twice weekly Receives: Drug: Placebo matching Pegcetacoplan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 24
Secondary outcomes (32)
Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange) Mean change from Baseline to Week 24 in Hemoglobin (Hgb) level.
Measured: Week 24
Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A
Percentage of patients who did not receive a blood transfusion between Week 5 and Week 24 was assessed
Measured: 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)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
FACT-An is used to measure quality of life (QoL) in patients with anemia. Each item is rated on a 5-point Likert scale:0=Not at all, 1=A little bit ,2=Somewhat, 3=Quite a bit, 4=Very much. Some items are reverse scored. Higher scores in this scale denote a better QoL with less impact of anemia.The total FACT-An scale score ranges from 0 to 160. The total score gives a comprehensive view of a patient's well-being. It combines the FACT-G with an Anemia subscale. FACT-G (27 items):Physical Well-Being (PWB) and Social/Family Well-Being (SWB) 14 items in total, Emotional Well-Being (EWB)-6 items,Functional Well-Being (FWB)-7 items, Anemia Subscale (AnS): 13 items. Total FACT-An score=FACT-G + Anemia Subscale=40 item
Measured: Week 24
Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A
Number of blood transfusions received between Week 5 and Week 24 were assessed.
Measured: Week 24
Change From Baseline to Week 24 in LDH Levels-Part A in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange) Mean change from baseline to Week 24 in Lactate dehydrogenase (LDH) levels
Measured: Week 24
Change From Baseline to Week 24 in Haptoglobin Levels-Part A in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from baseline to Week 24 in Haptoglobin level
Measured: Week 24
Change From Baseline to Week 24 in Indirect Bilirubin-Part A in the Absence of Intercurrent Events (ICE)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from baseline to Week 24 in Indirect bilirubin level
Measured: Week 24
Change From Baseline to Week 24 in ARC-Part A in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from baseline to Week 24 in Absolute reticulocyte counts (ARC).
Measured: Week 24
Change From Baseline to Week 24 in D-dimer Levels-Part A in the Absence of Intercurrent Events (ICEs
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange) change from baseline to Week 24 in D-dimer levels.
Measured: Week 24
Normalization of Markers of Hemolysis (LDH) at Week 24-Part A
Percentage of patients with LDH level within normal ranges and with an abnormal value at baseline.
Measured: Week 24
Normalization of Markers of Hemolysis (Indirect Bilirubin) at Week 24-Part A
Percentage of patients with Indirect Bilirubin level within normal ranges and with an abnormal value at baseline.
Measured: Week 24
Normalization of Markers of Hemolysis (ARC) at Week 24-Part A
Percentage of patients with ARC level within normal ranges and with an abnormal value at baseline.
Measured: Week 24
Normalization of Markers of Hemolysis (Haptoglobin) at Week 24-Part A
Percentage of patients with haptoglobin level within normal ranges and with an abnormal value at baseline.
Measured: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Haptoglobin Levels-Part A
Percentage of patients with haptoglobin level normalization during the initial 24 weeks of the study
Measured: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Hemoglobin Levels-Part A
Percentage of patients with Hemoglobin level normalization during the initial 24 weeks of the study
Measured: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for LDH Levels-Part A
Percentage of patients with LDH level normalization during the initial 24 weeks of the study
Measured: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Indirect Bilirubin Levels-Part A
Percentage of patients with Indirect Bilirubin level normalization during the initial 24 weeks of the study
Measured: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for ARC Levels-Part A
Percentage of patients with ARC level normalization during the initial 24 weeks of the study
Measured: Week 24
Number of Packed Red Blood Cell Units Transfused From Week 5 to Week 24-Part A
Number of PRBC units transfused from Week 5 and Week 24 was assessed
Measured: Week 5 to Week 24
Change From Baseline to Week 24 in FACIT-F Subscale Score-Part A in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
The FACIT-F subscale is used to measure fatigue and its impact upon daily activities and function in patients with chronic illnesses and contains 20 items related to the impact of fatigue. Each item is scored on a 0-4 scale, with some items reverse-scored. Total scores range from 0 to 52, where higher scores indicate less fatigue and better outcomes, and lower scores reflect greater fatigue. It can be used alone or with other FACIT subscales as part of broader quality of life assessments.
Measured: Week 24
Change From Baseline to Week 24 in SF-12-Part A in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange.
SF-12 is a 12-item health survey assessing physical and mental health. Higher scores mean better health with scores above 50 indicating better than average health. It produces two summary scores: the Physical Component Summary (PCS) and Mental Component Summary (MCS), both norm-based (mean=50, SD=10), with ranges of ~5-80 (PCS) and ~-3.3-80 (MCS). Higher scores=better health. It also covers 8 subscales-Physical Functioning (PF), Role-Physical (RP), Bodily Pain (BP), General Health (GH), Vitality (VT), Social Functioning (SF), Role-Emotional (RE), and Mental Health (MH), each scored between 0-100, where higher=better functioning. Scores are computed using weighted formulas from item responses (not simple averages).
Measured: Week 24
Change From Baseline to Week 24 in EQ-5D-5L Questionnaire -Part A in the Absence of Intercurrent Events (ICEs)
The EQ-5D-5L measures total health across 5 dimensions: Mobility, Self-care, Usual Activities, Pain/Discomfort, and Anxiety/Depression-each scored from 1 (no problems) to 5 (extreme problems). Scores form a 5-digit health profile (e.g., 12345). Each individual score plus a VAS for perceived health status today are separately reported. Higher scores reflect better total health. The EQ VAS is a patient-rated score from 0 (worst) to 100 (best health).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Measured: Week 24
Change From Baseline to Week 48 in Hemoglobin (Hgb) Level-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from Baseline to Week 48 in Hemoglobin (Hgb) level.
Measured: Week 48
Change From Baseline to Week 48 in LDH Level-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from Baseline to Week 48 in LDH level.
Measured: Week 48
Change From Baseline to Week 48 in Haptoglobin Level-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from Baseline to Week 48 in Haptoglobin level
Measured: Week 48
Change From Baseline to Week 48 in Indirect Bilirubin Level-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from Baseline to Week 48 in Indirect Bilirubin level.
Measured: Week 48
Change From Baseline to Week 48 in ARC-Part B in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from baseline to Week 48 in Absolute reticulocyte counts (ARC).
Measured: Week 48
Change From Baseline to Week 48 in D-dimer Levels-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Mean change from baseline to Week 48 in D-dimer levels.
Measured: Week 48
Change From Baseline to Week 48 in FACT-An Scale Score (Quality of Life)-Part B in the Absence of Intercurrent Events (ICEs)
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug and plasma exchange.
The FACT-An is used to measure quality of life (QoL) in patients with anemia. Each item is rated on a 5-point Likert scale:0=Not at all, 1=A little bit ,2=Somewhat, 3=Quite a bit, 4=Very much.Some items are reverse scored. Higher scores in this scale denote a better QoL with less impact of anemia. The total FACT-An scale score ranges from 0 to 160. The total score gives a comprehensive view of a patient's well-being. It combines the FACT-G with an Anemia subscale.FACT-G (27 items):Physical Well-Being (PWB) and Social/Family Well-Being (SWB) 14 items in total, Emotional Well-Being (EWB)-6 items, Functional Well-Being (FWB)-7 items, Anemia Subscale (AnS): 13 items. Total FACT-An score=FACT-G + Anemia Subscale=40 items
Measured: Week 48
Change From Baseline to Week 48 in FACIT-F Subscale Score-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
The FACIT-F subscale is used to measure fatigue and its impact upon daily activities and function in patients with chronic illnesses and contains 20 items related to the impact of fatigue. Each item is scored on a 0-4 scale, with some items reverse-scored. Total scores range from 0 to 52, where higher scores indicate less fatigue and better outcomes, and lower scores reflect greater fatigue. It can be used alone or with other FACIT subscales as part of broader quality of life assessments.
Measured: Week 48
Change From Baseline to Week 48 in EQ-5D-5L-Part B in the Absence of Intercurrent Events (ICEs).
The EQ-5D-5L measures total health across 5 dimensions: Mobility, Self-care, Usual Activities, Pain/Discomfort, and Anxiety/Depression-each scored from 1 (no problems) to 5 (extreme problems). Scores form a 5-digit health profile (e.g., 12345). Each individual score plus a VAS for perceived health status today are separately reported. Higher scores reflect better total health. The EQ VAS is a patient-rated score from 0 (worst) to 100 (best health).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange).
Measured: Week 48
Change From Baseline to Week 48 in SF-12-Part B in the Absence of Intercurrent Events (ICEs).
The ICEs of interest were :
- Withdrawal from treatment or lost to follow-up before the end of the double-blind period
- Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange SF-12 is a 12-item health survey assessing physical and mental health. Higher scores mean better health with scores above 50 indicating better than average health. It produces two summary scores: the Physical Component Summary (PCS) and Mental Component Summary (MCS), both norm-based (mean = 50, SD =10), with ranges of ~5-80 (PCS) and ~-3.3-80 (MCS). Higher scores = better health. It also covers 8 subscales-Physical Functioning (PF), Role-Physical (RP), Bodily Pain (BP), General Health (GH), Vitality (VT), Social Functioning (SF), Role-Emotional (RE), and Mental Health (MH), each scored between 0-100, where higher=better functioning. Scores are computed using weighted formulas from item responses (not simple averages).
Measured: Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2021
- Study start20 Oct 2022
- Primary completion27 May 2024
- Study completion11 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cold Agglutinin DiseaseStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study physician, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05096403
- Sponsor's protocol ID
- Sobi.PEGCET-101
- Status last verified
- Oct 2025
- First submitted
- 15 Oct 2021
- Last updated
- 30 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05096403
- Acronym / sponsor ID
- Sobi.PEGCET-101
- Conditions
- Cold Agglutinin Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 20 Oct 2022 (actual)
- Primary completion
- 27 May 2024 (actual)
- Study completion
- 11 Sep 2024 (actual)
- First posted
- 27 Oct 2021 (actual)
- Results
- Posted 30 Oct 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
This is a blind (actual treatment not disclosed to Investigator or participant) study to study pegcetacoplan in people with cold agglutinin disease. The study will consist of a 4-week screening period where selected tests will be conducted to ensure that the patient is eligible to participate in the study, followed by Part A, a 24-week blinded treatment period where the participants will receive either pegcetacoplan or a placebo treatment, looking like pegcetacoplan but with no effect. After this period, the participants will move into Part B, a 24-week period where they will all receive pegcetacoplan. Part C is a 48-week maintenance period with pegcetacoplan for all participants. After the end of treatment participants will undergo a safety follow visit about 8 weeks after last dose.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Pegcetacoplan Double Blind During Part A | Active comparator | Drug: Pegcetacoplan1080 mg, subcutaneous injection, twice weekly |
| Placebo Matching Pegcetacoplan-Double-blind During Part A | Placebo comparator | Drug: Placebo matching PegcetacoplanSodium acetate, subcutaneous injection, twice weekly |
| Open-label Pegcetacoplan During Parts B and C | Active comparator | Drug: Pegcetacoplan1080 mg, subcutaneous injection, twice weekly |
| Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C | Placebo comparator | Drug: Placebo matching PegcetacoplanSodium acetate, subcutaneous injection, twice weekly |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegcetacoplanAlso: Aspaveli, EmpaveliPegcetacoplan taken twice weekly as subcutaneous injection | Drug | Open-label Pegcetacoplan During Parts B and C, Pegcetacoplan Double Blind During Part A |
| Placebo matching PegcetacoplanPlacebo matching pegcetacoplan taken twice weekly as subcutaneous injection | Drug | Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C, Placebo Matching Pegcetacoplan-Double-blind During Part A |
Primary outcome
Number of Patients Achieving a Response (R) at Week 24
Time frame: 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.
32 secondary outcomes
Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs).
Time frame: Week 24
Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A
Time frame: 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)
Time frame: Week 24
Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A
Time frame: Week 24
Change From Baseline to Week 24 in LDH Levels-Part A in the Absence of Intercurrent Events (ICEs)
Time frame: Week 24
Change From Baseline to Week 24 in Haptoglobin Levels-Part A in the Absence of Intercurrent Events (ICEs)
Time frame: Week 24
Change From Baseline to Week 24 in Indirect Bilirubin-Part A in the Absence of Intercurrent Events (ICE)
Time frame: Week 24
Change From Baseline to Week 24 in ARC-Part A in the Absence of Intercurrent Events (ICEs)
Time frame: Week 24
Change From Baseline to Week 24 in D-dimer Levels-Part A in the Absence of Intercurrent Events (ICEs
Time frame: Week 24
Normalization of Markers of Hemolysis (LDH) at Week 24-Part A
Time frame: Week 24
Normalization of Markers of Hemolysis (Indirect Bilirubin) at Week 24-Part A
Time frame: Week 24
Normalization of Markers of Hemolysis (ARC) at Week 24-Part A
Time frame: Week 24
Normalization of Markers of Hemolysis (Haptoglobin) at Week 24-Part A
Time frame: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Haptoglobin Levels-Part A
Time frame: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Hemoglobin Levels-Part A
Time frame: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for LDH Levels-Part A
Time frame: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Indirect Bilirubin Levels-Part A
Time frame: Week 24
Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for ARC Levels-Part A
Time frame: Week 24
Number of Packed Red Blood Cell Units Transfused From Week 5 to Week 24-Part A
Time frame: Week 5 to Week 24
Change From Baseline to Week 24 in FACIT-F Subscale Score-Part A in the Absence of Intercurrent Events (ICEs)
Time frame: Week 24
Change From Baseline to Week 24 in SF-12-Part A in the Absence of Intercurrent Events (ICEs)
Time frame: Week 24
Change From Baseline to Week 24 in EQ-5D-5L Questionnaire -Part A in the Absence of Intercurrent Events (ICEs)
Time frame: Week 24
Change From Baseline to Week 48 in Hemoglobin (Hgb) Level-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in LDH Level-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in Haptoglobin Level-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in Indirect Bilirubin Level-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in ARC-Part B in the Absence of Intercurrent Events (ICEs)
Time frame: Week 48
Change From Baseline to Week 48 in D-dimer Levels-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in FACT-An Scale Score (Quality of Life)-Part B in the Absence of Intercurrent Events (ICEs)
Time frame: Week 48
Change From Baseline to Week 48 in FACIT-F Subscale Score-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in EQ-5D-5L-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Change From Baseline to Week 48 in SF-12-Part B in the Absence of Intercurrent Events (ICEs).
Time frame: Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study physician study director
Swedish Orphan Biovitrum
44 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pegcetacoplan in people with Cold Agglutinin Disease, run by Swedish Orphan Biovitrum at 44 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 44 sites in Spain, Italy, Japan, United States, United Kingdom and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Achieving a Response (R) at Week 24 (time frame: Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2025.