A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Without A Recent History of Blood Transfusion (Cadenza)

• ClinicalTrials.gov Identifier: NCT03347422
• Recruitment Status: Completed
• First Posted: November 20, 2017
• Results First Posted: December 23, 2022
• Last Update Posted: December 23, 2022
• Sponsor: Bioverativ, a Sanofi company
• Information provided by (Responsible Party): Sanofi ( Bioverativ, a Sanofi company )

Study Description

Brief Summary:

The purpose of Part A was to determine whether sutimlimab administration resulted in a greater than or equal to (>=)1.5 grams per deciliter (g/dL) increase in hemoglobin (Hgb) level and avoidance of transfusion in participants with primary cold agglutinin disease (CAD) without a recent history of blood transfusion. The purpose of Part B was to evaluate the long-term safety and tolerability of sutimlimab in participants with primary CAD.

Condition or disease	Intervention/treatment	Phase
Cold Agglutinin Disease	Drug: sutimlimab (BIVV009); Drug: placebo	Phase 3

Detailed Description:

The planned total study duration per participant was approximately 1.5 to 2.5 years.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 42 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Sutimlimab in Patients With Primary Cold Agglutinin Disease Without a Recent History of Blood Transfusion
• Actual Study Start Date: March 17, 2018
• Actual Primary Completion Date: December 3, 2021
• Actual Study Completion Date: December 3, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: BIVV009/BIVV009; Participants with primary CAD and without a recent history of blood transfusion during the last 6 months prior to enrollment in this study, received an intravenous (IV) infusion of BIVV009 6.5 g (for participants less than [<]75 kilograms [kg]) or 7.5 g dose (for participants greater than or equal to [>=]75 kg) on Day 0 and Day 7 and every 14 days thereafter in Part A up to Week 25. Participants who completed Part A per protocol through the end of treatment visit (Week 26), received placebo on Week 26 and continued to receive BIVV009 6.5 or 7.5 g in Part B, every 2 weeks starting at Week 27 for up to an additional 149 weeks (for 6.5 g) or 121 weeks (for 7.5 g). All participants who completed Part A elected to continue in Part B.	Drug: sutimlimab (BIVV009); Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)
Experimental: Placebo/BIVV009; Participants with primary CAD and without a recent history of blood transfusion during the last 6 months prior to enrollment in this study, received an IV infusion of placebo matched to BIVV009 on Day 0 and Day 7 and every 14 days thereafter in Part A up to Week 25. Participants who completed Part A per protocol through the end of treatment visit (Week 26) received BIVV009 6.5 (if <75 kg) or 7.5 g (if >=75 kg) in Part B, on Week 26 and Week 27 and every 2 weeks thereafter for up to an additional 123 weeks (for 6.5 g) or 137 weeks (for 7.5 g). All participants who completed Part A elected to continue in Part B.	Drug: sutimlimab (BIVV009); Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.); Drug: placebo; Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)

Outcome Measures

Primary Outcome Measures :

1. Part A: Percentage of Participants With Response to Treatment [ Time Frame: From Week 5 through Week 26 ]
   A participant was considered a responder: if he or she did not receive blood transfusion from Week 5 through Week 26 (end of treatment) and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, participant's hemoglobin (Hgb) level must have increased to >=1.5 grams per deciliter (g/dL) from baseline (defined as last Hgb value before administration of first dose of study drug) at treatment assessment timepoint (defined as average of values from the Week 23, 25, and 26 visits). Percentage of responders was calculated together with 95% exact Clopper-Pearson confidence interval (CI).
2. Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: Part B, 6.5 g cohort: From first dose (Week 26) up to 149 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 184); Part B, 7.5 g cohort: From first dose (Week 26) up to 137 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 172) ]
   Adverse Event (AE): any untoward medical occurrence in a participant who received study drug and did not necessarily have to have a causal relationship with the treatment. Treatment emergent serious adverse events (TESAEs) was defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect, was medically important event. Treatment emergent adverse events (TEAEs): AEs that developed, worsened or became serious during the treatment-emergent (TE) period (from first investigational medicinal product [IMP] administration in Part B to last IMP administration + 9 weeks follow-up period).

Secondary Outcome Measures :

1. Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint [ Time Frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26) ]
   Mean change from baseline (Week 0) in Hemoglobin (Hgb) at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Least squares (LS) mean and 95 % confidence interval (CI) was assessed by Mixed Model for Repeated Measures (MMRM) approach using heterogeneous Toeplitz (TOEPH) covariance matrix with change from baseline as the dependent variable and baseline value and visits as independent variables. Baseline was defined as the last non-missing value prior to the first administration of study drug.
2. Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at the Treatment Assessment Timepoint [ Time Frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26) ]
   FACIT-Fatigue scale consists of 13 questions 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 were added to obtain a total score. Total score ranged from 0 to 52, with higher score indicating more fatigue. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. LS mean and 95% CI was assessed by MMRM approach using TOEPH covariance matrix with change from baseline (Week 0) as the dependent variable and baseline value and visits as independent variables. Baseline was defined as the last non-missing value prior to the first administration of study drug.
3. Part A: Mean Change From Baseline in Total Bilirubin Levels at the Treatment Assessment Timepoint [ Time Frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26) ]
   Mean change from baseline (Week 0) in total bilirubin at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Baseline was defined as the last non-missing value prior to the first administration of study drug.
4. Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) at the Treatment Assessment Timepoint [ Time Frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26) ]
   Mean change from baseline (Week 0) in LDH at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Baseline was defined as the last non-missing value prior to the first administration of study drug.
5. Part A: Percentage of Participants With Solicited Symptomatic Anemia at Week 26 [ Time Frame: Week 26 ]
   Symptomatic anemia was defined as having following symptoms: i. Fatigue; ii. Weakness; iii. Shortness of breath; iv. Palpitations, fast heartbeat; v. Light headedness and/or vi. Chest pain. Percentage of participants with solicited symptomatic anemia symptoms was reported in this outcome measure.
6. Part B: Change From Baseline in Hemoglobin (Hgb) Level at Each Specified Time Points [ Time Frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184) ]
   Change from baseline (Week 0) in Hgb levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43,45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83,85,87, 89,91, 93, 95, 97, 99,101,103, 105, 107,109, 111,113,115,117,119, 121,123, 125, 127,129,131,133,135,137,139,141,143,145,147,149,151,153, 155,157,159,161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. Early Termination (ET) visit/safety follow up (SFU) visit was 9 weeks after administration of last dose (i.e., up to Week 184). Here, "0" in the number analyzed field signifies that none of participants were available for assessment at the specified timepoints.
7. Part B: Change From Baseline in Total Bilirubin Levels at Each Specified Time Points [ Time Frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184) ]
   Change from baseline (Week 0) in total bilirubin levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65,67,69,71,73,75, 77, 79, 81,83, 85, 87, 89, 91, 93, 95, 97, 99,101,103, 105, 107,109, 111,113,115,117,119, 121, 123, 125, 127,129,131,133,135,137,139,141,143, 145,147,149,151,153,155,157,159, 161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184). Here, "0" in the number analyzed field signifies that none of participants were available for assessment at the specified timepoints.
8. Part B: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Each Specified Time Points [ Time Frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184) ]
   FACIT-Fatigue scale consists of 13 questions 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 were added to obtain a total score. The Total score ranged from 0 to 52, with higher score indicating more fatigue. Baseline (Week 0) was defined as the last non-missing value prior to the first administration of study drug in Part A. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
9. Part B: Change From Baseline in 12-Item Short-Form Survey (SF-12) Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Each Specified Time Points [ Time Frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184) ]
   SF-12: 12 item-questionnaire assessed health-related quality of life (HRQOL), contained 12 items, categorized into 8 domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health). Higher scores = good health condition. These 8 domains were further summarized into 2 summary scores, PCS and MCS that ranged from 0 (poor health) to 100 (better health). Higher scores = better HRQOL. Baseline (Week 0): last non-missing value prior to first administration of study drug in Part A. ET visit/SFU visit: 9 weeks after administration of last dose (i.e., up to Week 184).
10. Part B: Change From Baseline in 5-level European Quality of Life 5- Dimensions 5-Level Questionnaire (EQ-5D-5L) Health State Utility Index and VAS Scores at Each Specified Time Points [ Time Frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184) ]
    EQ-5D-5L included 2 components: health state utility index (descriptive system) and Visual Analog Scale (VAS). EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 response option: no problem, slight problem, moderate problem, severe problem and extreme problems measured with Likert scale. EQ-5D-5L responses converted into single index utility score between 0 to 1. Higher score=better health. EQ-5D-5L VAS rated participant's current health state on scale from 0 (worst imaginable health) to 100 (best imaginable health). Baseline (Week 0): last non-missing value prior to first administration of study drug in Part A. ET visit/SFU visit: 9 weeks after administration of last dose (i.e., up to Week 184).
11. Part B: Number of Participants With Response to Participant's Global Impression of (Fatigue) Severity (PGIS) Questionnaire at Each Specified Time Points [ Time Frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184) ]
    The PGIS is a self-reported scale. The PGIS is a 1-item questionnaire designed to assess participant's impression of disease severity using a 5-point scale ranging from 1 to 5, where 1=none, 2=mild, 3=moderate, 4=severe, 5=very severe. Higher scores indicated greater severity. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
12. Part B: Number of Participants With Response to Participant's Global Impression of Change (PGIC) Questionnaire at Each Specified Time Points [ Time Frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184) ]
    PGIC is a self-administered questionnaire to evaluate the improvement or worsening compared to the start of the study. PGIC was assessed on a 7-point Likert scale ranged from 1 (greatly improved) to 7 (greatly worsened). Categories were defined based on the PGIC scores as follows: 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse and 7=very much worsen. Higher scores indicated greater severity. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
13. Part B: Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level at Each Specified Time Points [ Time Frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184) ]
    Change from baseline (Week 0) in LDH levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97, 99, 101, 103, 105, 107,109, 111, 113, 115, 117, 119, 121,123, 125, 127,129,131,133,135,137,139,141,143,145,147, 149, 151,153,155,157,159,161,163,165,167,169, 171, 173, 175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
14. Part B: Number of Blood Transfusions Per Participant [ Time Frame: From Week 27 up to 149 weeks of treatment (i.e., up to Week 176) ]
    A participant was to receive a transfusion if his or her Hgb level met either of the following criteria: Hgb was <9 g/dL and the participant had symptoms of anemia or Hgb was <7 g/dL and the participant was asymptomatic.
15. Part B: Mean Change From Baseline in Haptoglobin Values at Each Specified Time Points [ Time Frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184) ]
    Change from baseline (Week 0) in haptoglobin values at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97,99,101,103, 105, 107,109, 111,113,115,117,119, 121,123, 125, 127, 129,131,133,135,137,139,141,143, 145,147,149,151,153,155,157,159, 161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184). Haptoglobin values <0.2 were imputed as 0.2.
16. Part B: Number of Healthcare Visits by Type [ Time Frame: From Week 27 up to 149 weeks of treatment (i.e., up to Week 176) ]
    In this outcome measure, number of healthcare visits which included non-study healthcare resource utilization visit (consisted mainly of extra visits to the office of the study doctor, visit to a generalist doctor or visit to a specialist doctor), hospitalization visit and visit to hospital emergency is reported.

Eligibility Criteria

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

Criteria

Inclusion criteria:

• Body weight of >=39 kg at screening.
• Confirmed diagnosis of primary CAD based on the following criteria: a) Chronic hemolysis, b) Polyspecific direct antiglobulin test (DAT) positive, c) Monospecific DAT strongly positive for C3d, d) Cold agglutinin titer >= 64 at 4 degree Celsius, and e) Immunoglobulin G DAT less than or equal to (<=) 1+, and, f) No overt malignant disease.
• Hemoglobin level <= 10.0 g/dL.
• Bilirubin level above the normal reference range, including participants with Gilbert's Syndrome.

Exclusion criteria:

• Cold agglutinin syndrome secondary to infection, rheumatologic disease, or active hematologic malignancy.
• History of blood transfusion within 6 months of screening, or history of more than one blood transfusion within 12 months of screening.
• Clinically relevant infection of any kind within the month preceding enrollment (example, active hepatitis C, pneumonia).
• Clinical diagnosis of systemic lupus erythematosus; or other autoimmune disorders with anti-nuclear antibodies at screening. Anti-nuclear antibodies of long-standing duration without associated clinical symptoms would be adjudicated on a case-by-case basis during the confirmatory review of participant eligibility.
• Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at screening.
• Positive human immunodeficiency virus antibody at screening.
• Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (example, with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment.

The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Contacts and Locations

Locations

United States, Arizona

Arizona Oncology Associates PC

Tucson, Arizona, United States, 85711

United States, California

USC/Keck School of Medicine

Los Angeles, California, United States, 90033

United States, District of Columbia

Georgetown University Medical Center

Georgetown, District of Columbia, United States, 20007

United States, Florida

Cleveland Clinic Florida

Weston, Florida, United States, 33331

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

United States, Missouri

Washington University School of Medicine

Saint Louis, Missouri, United States, 63110

United States, New York

Montefiore Medical Center

New York, New York, United States, 10461

New York Medical College at Westchester Medical Center

Valhalla, New York, United States, 10595

United States, North Carolina

East Carolina University

Greenville, North Carolina, United States, 27834

United States, Ohio

Cleveland Clinic

Cleveland, Ohio, United States, 44195

United States, Pennsylvania

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States, 15232

United States, Wisconsin

UW Hospitals and Clinics

Madison, Wisconsin, United States, 53792

Australia, Queensland

USC Health Clinics

Buderim, Queensland, Australia, 4556

Australia, Victoria

Ballarat Oncology & Haematology

Ballarat, Victoria, Australia, 3350

Monash Medical Centre

Clayton, Victoria, Australia, 3168

Australia, Western Australia

Perth Blood Institute

West Perth, Western Australia, Australia, 6005

Austria

Medical University of Vienna

Vienna, Austria, 1090

Belgium

ZNA Stuivenberg

Antwerpen, Belgium, 2060

University Hospitals Leuven

Leuven, Belgium, 3000

Canada, Ontario

St. Michael's Hospital

Toronto, Ontario, Canada, M5B1W8

Canada, Quebec

McGill University Health Center

Montréal, Quebec, Canada, H4A3J1

France

CHU d'Angers

Angers Cedex 9, France, 49933

Hôpital de Caen

Caen, France, 14033

Centre Hospitalier Henri Mondor

Créteil, France, 94000

Centre Hospitalier Lyon Sud

Pierre-Bénite, France, 69495

Germany

Gemeinschaftspraxis Hämatologie-Onkologie

Dresden, Germany, 1307

Universitätsklinikum Essen

Essen, Germany, 45147

Univ Ulm, Inst Klin. Transfusions. Immungen

Ulm, Germany, 89081

Israel

Hadassah Medical Center

Jerusalem, Israel, 91120

Laniado Hospital

Netanya, Israel, 4244916

Italy

A. O. Spedali Civili di Brescia

Brescia, Italy, 25123

Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico

Milan, Italy, 20122

U.O.C. Ematologia- Policlinico "A. Gemelli"

Rome, Italy, 00168

U.O.C. Ematologia Ospedale San Bortolo

Vicenza, Italy, 36100

Japan

Japanese Red Cross Society Himeji Hospital

Himeji, Hyogo, Japan, 670-8540

Ishikawa Prefectural Central Hospital

Kanazawa, Ishikawa-ken, Japan, 9208530

Tokai University Hospital

Isehara, Kanagawa, Japan, 259-1193

Osaka University Hospital

Suita, Osaka, Japan, 565-0871

Saitama Medical University Hospital

Iruma-gun, Saitama-Ken, Japan, 350-0495

Aichi Medical University Hospital

Nagakute, Japan, 480-1195

Netherlands

Academisch Medisch Centrum

Amsterdam, Netherlands, 1105

Leids Universitair Medisch Centrum

Leiden, Netherlands, 2333

Norway

Haukeland University Hospital

Bergen, Norway, 5053

St Olavs Hospital, Avdeling for blodsykdommer

Trondheim, Norway, 7030

Spain

Hospital Universitario Puerta de Hierro

Majadahonda, Madrid, Spain, 28222

Hospital Clinci i Provincial de Barcelona

Barcelona, Spain, 08036

Hospital Universitario Virgen del Rocio

Sevilla, Spain, 41013

Hospital Universitario Dr. Peset

Valencia, Spain, 46017

United Kingdom

St James Hospital, Leeds

Leeds, United Kingdom, LS9 7TF

Imperial College Healthcare NHS Trust, Hammersmith Hospital

London, United Kingdom, W12 0HS

University College London

London, United Kingdom, WC1E 6AG

Sponsors and Collaborators

Bioverativ, a Sanofi company

Investigators

• Study Director: Clinical Sciences & Operations; Sanofi

  Study Documents (Full-Text)

Documents provided by Sanofi ( Bioverativ, a Sanofi company ):

Study Protocol  [PDF] November 4, 2020

Statistical Analysis Plan  [PDF] October 2, 2020

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Roth A, Berentsen S, Barcellini W, D'Sa S, Jilma B, Michel M, Weitz IC, Yamaguchi M, Nishimura JI, Vos JMI, Storek M, Wong N, Patel P, Jiang X, Vagge DS, Wardecki M, Shafer F, Lee M, Broome CM. Sutimlimab in patients with cold agglutinin disease: results of the randomized placebo-controlled phase 3 CADENZA trial. Blood. 2022 Sep 1;140(9):980-991. doi: 10.1182/blood.2021014955.

• Responsible Party: Bioverativ, a Sanofi company
• ClinicalTrials.gov Identifier: NCT03347422
• Other Study ID Numbers: EFC16216; 2017-003539-12 ( EudraCT Number ); BIVV009-04 ( Other Identifier: Bioverativ Therapeutics Inc. )
• First Posted: November 20, 2017
• Results First Posted: December 23, 2022
• Last Update Posted: December 23, 2022
• Last Verified: December 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Anemia, Hemolytic, Autoimmune; Anemia, Hemolytic; Anemia; Hematologic Diseases; Autoimmune Diseases; Immune System Diseases