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A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion (Cardinal Study)

Primary Purpose

Agglutinin Disease, Cold

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

BIVV009

About this trial

This is an interventional treatment trial for Agglutinin Disease, Cold

Eligibility Criteria

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

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; e) Immunoglobulin G (IgG) DAT less than or equal to (<=) 1+, and f) No overt malignant disease.
History of at least one documented blood transfusion within 6 months of enrollment.
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.
Clinically relevant infection of any kind within the month preceding enrollment (e.g., 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 adjudicated on a case-by-case basis during the Confirmatory Review of Patient Eligibility.
Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at Screening.
Positive human immunodeficiency virus (HIV) antibody at screening.
Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (e.g., with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BIVV009

Arm Description

Participants with primary CAD who had a recent history of transfusion (defined as at least 1 transfusion during the last 6 months prior to screening) received an intravenous (IV) infusion of BIVV009 6.5 grams (g) (if body weight was less than [<] 75 kilograms [kg]) or BIVV009 7.5 g (if body weight was 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) could continue to receive BIVV009 in Part B, every 2 weeks starting at Week 27 for up to an additional 149 weeks. All participants who completed Part A elected to continue in Part B.

Outcomes

Primary Outcome Measures

Part A: Percentage of Participants With Response to Treatment

A participant was considered a responder: if he or she did not receive a blood transfusion from Week 5 through Week 26 (end of treatment in Part A) and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, the participant's hemoglobin (Hgb) level must meet either of the following criteria: Hgb level >= 12 grams per deciliter (g/dL) at the treatment assessment endpoint (defined as the average of the values from the Week 23, 25, and 26 visits), or Hgb increased >= 2 g/dL from baseline (defined as the last Hgb value before administration of the first dose of study drug) at treatment assessment endpoint. Percentage of responders was calculated together with a 95% exact Clopper-Pearson confidence interval (CI).

Part B: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

An Adverse Event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily have to have a causal relationship with the treatment. 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 a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent (TE) period (from the first investigational medicinal product [IMP] administration in Part B to the last IMP administration + 9 weeks follow up period).

Secondary Outcome Measures

Part A: Mean Change From Baseline in Bilirubin Levels at the Treatment Assessment Timepoint

Mean change from baseline in bilirubin levels at the treatment assessment timepoint was 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.

Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Treatment Assessment Timepoint

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 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.

Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) at the Treatment Assessment Timepoint

Mean change from baseline 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. LS mean and 95% CI was assessed by MMRM approach using 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.

Part A: Number of Blood Transfusions Per Participant

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. Number of transfusions after the first 5 weeks of study drug administration and up to Week 26 are reported in this outcome measure.

Part A: Number of Blood Units Transfused Per Participant

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. Number of blood units transfused after the first 5 weeks of study drug administration and up to Week 26 are reported in this outcome measure.

Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint

Mean change from baseline (Week 0) in Hgb at 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. LS mean and 95% CI was assessed by MMRM approach using 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.

Part B: Change From Baseline in Hemoglobin (Hgb) Level at Each Specified Time Points

Change From Hgb level from baseline (Week 0) at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175 and early termination/safety follow up [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 185).

Part B: Change From Baseline in Bilirubin Levels at Each Specified Time Points

Change from baseline (Week 0) in bilirubin levels at each specified time point (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 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 185).

Part B: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and Early Termination (ET) Visit/Safety Follow-up Visit

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 185).

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

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 for which, score ranged 0 (poor health) to 100 (better health). Higher scores = better HRQOL. Baseline (Week 0) was defined as the last non-missing value prior to 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 185). Change from baseline in SF-12 PCS and MCS scores is reported in this outcome measure.

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 Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and ET Visit/SFU Visit

EQ-5D-5L:standardized, participant-rated questionnaire to assess health-related quality of life. EQ-5D-5L includes 2 components: EQ-5D-5L 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 problems, slight problems, moderate problems, severe problems, and extreme problems measured with Likert scale. EQ-5D-5L responses relating to 5 dimensions are converted into a single index utility score between 0 to 1, where higher score=better health state. The EQ-5D-5L VAS rated participant's current health state on a scale from 0=worst imaginable health state to 100 =best imaginable health state. Baseline (Week 0): last non-missing value prior to 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 185).

Part B: Number of Participants With Response to Participant's Global Impression of (Fatigue) Severity (PGIS) Questionnaire at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU Visit

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 185).

Part B: Number of Participants With Response to Participant's Global Impression of Change (PGIC) Questionnaire at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU Visit

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 185).

Part B: Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level at Each Specified Time Points

Mean change from baseline in LDH levels at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175 and ET/SFU Visit) is reported in this outcome measure. 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 185).

Part B: Number of Blood Transfusions Per Participant

Part B: Number of Blood Units Transfused Per Participant

Part B: Change From Baseline in Haptoglobin Values at Each Specified Time Points

Change in Haptoglobin values from baseline at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 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 185). Haptoglobin values <0.2 were imputed as 0.2.

Part B: Number of Healthcare Visits by Type

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, or visit to a generalist doctor, or visit to a specialist doctor) and hospitalization visit and visit to hospital emergency is reported.

Full Information

NCT ID

NCT03347396

First Posted

November 16, 2017

Last Updated

October 4, 2022

Sponsor

Bioverativ, a Sanofi company

1. Study Identification

Unique Protocol Identification Number

NCT03347396

Brief Title

A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion (Cardinal Study)

Official Title

A Phase 3, Pivotal, Open-label, Multicenter Study to Assess the Efficacy and Safety of Sutimlimab in Patients With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Completed

Study Start Date

March 5, 2018 (Actual)

Primary Completion Date

October 5, 2021 (Actual)

Study Completion Date

October 5, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bioverativ, a Sanofi company

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

The purpose of Part A was to determine whether sutimlimab administration resulted in a greater than or equal to (>=) 2 grams per deciliter (g/dL) increase in hemoglobin (Hgb) levels or increased Hgb to >= 12 g/dL and obviated the need for blood transfusion during treatment in participants with primary cold agglutinin disease (CAD) who had 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 CAD.

6. Conditions and Keywords

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

Agglutinin Disease, Cold

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

24 (Actual)

8. Arms, Groups, and Interventions

Arm Title

BIVV009

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

BIVV009

Other Intervention Name(s)

Sutimlimab

Intervention Description

Sutimlimab was administered as intravenous (IV) infusion.

Primary Outcome Measure Information:

Title

Part A: Percentage of Participants With Response to Treatment

Description

Time Frame

From Week 5 through Week 26

Title

Part B: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

Description

Time Frame

Part B, 6.5 g cohort: From first dose (Week 27) up to 143 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 179); Part B, 7.5 g cohort: From first dose (Week 27) up to 149 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 185)

Secondary Outcome Measure Information:

Title

Part A: Mean Change From Baseline in Bilirubin Levels at the Treatment Assessment Timepoint

Description

Time Frame

Baseline, treatment assessment timepoint (i.e., average of Week 23, 25 and 26)

Title

Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Treatment Assessment Timepoint

Description

Time Frame

Baseline, treatment assessment timepoint (i.e., average of Week 23, 25 and 26)

Title

Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) at the Treatment Assessment Timepoint

Description

Time Frame

Baseline, treatment assessment timepoint (i.e., average of Week 23, 25 and 26)

Title

Part A: Number of Blood Transfusions Per Participant

Description

Time Frame

From Week 5 up to Week 26

Title

Part A: Number of Blood Units Transfused Per Participant

Description

Time Frame

From Week 5 up to Week 26

Title

Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint

Description

Time Frame

Baseline, treatment assessment timepoint (i.e., average of Week 23, 25 and 26)

Title

Part B: Change From Baseline in Hemoglobin (Hgb) Level at Each Specified Time Points

Description

Time Frame

Baseline, Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175, ET/SFU (up to Week 185)

Title

Part B: Change From Baseline in Bilirubin Levels at Each Specified Time Points

Description

Time Frame

Title

Description

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 185).

Time Frame

Baseline, Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and ET Visit/SFU visit (i.e., up to Week 185)

Title

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

Description

Time Frame

Baseline, Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135 and ET Visit/SFU visit (i.e., up to Week 185)

Title

Description

Time Frame

Baseline, Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and ET Visit/SFU visit (i.e., up to Week 185)

Title

Description

Time Frame

At Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU visit (i.e., up to Week 185)

Title

Part B: Number of Participants With Response to Participant's Global Impression of Change (PGIC) Questionnaire at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU Visit

Description

Time Frame

At Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU visit (i.e., up to Week 185)

Title

Part B: Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level at Each Specified Time Points

Description

Time Frame

Title

Part B: Number of Blood Transfusions Per Participant

Description

Time Frame

From Week 27 up to 149 weeks of treatment (i.e., up to Week 176)

Title

Part B: Number of Blood Units Transfused Per Participant

Description

Time Frame

From Week 27 up to 149 weeks of treatment (i.e., up to Week 176)

Title

Part B: Change From Baseline in Haptoglobin Values at Each Specified Time Points

Description

Time Frame

Baseline, Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175, ET/SFU (up to Week 185)

Title

Part B: Number of Healthcare Visits by Type

Description

Time Frame

From Week 27 up to 149 weeks of treatment (i.e., up to Week 176)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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; e) Immunoglobulin G (IgG) DAT less than or equal to (<=) 1+, and f) No overt malignant disease. History of at least one documented blood transfusion within 6 months of enrollment. 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. Clinically relevant infection of any kind within the month preceding enrollment (e.g., 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 adjudicated on a case-by-case basis during the Confirmatory Review of Patient Eligibility. Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at Screening. Positive human immunodeficiency virus (HIV) antibody at screening. Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (e.g., with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Sciences & Operations

Organizational Affiliation

Sanofi

Official's Role

Study Director

Facility Information:

Facility Name

Arizona Oncology Associates PC

City

Tucson

State/Province

Arizona

ZIP/Postal Code

85711

Country

United States

Facility Name

USC/Keck School of Medicine

City

Los Angeles

State/Province

California

ZIP/Postal Code

90033

Country

United States

Facility Name

The Oncology Institute of Hope and Innovation

City

Whittier

State/Province

California

ZIP/Postal Code

90603

Country

United States

Facility Name

Georgetown University Medical Center

City

Georgetown

State/Province

District of Columbia

ZIP/Postal Code

20007

Country

United States

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

Facility Name

Brigham and Women's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Montefiore Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10461

Country

United States

Facility Name

New York Medical College at Westchester Medical Center

City

Valhalla

State/Province

New York

ZIP/Postal Code

10595

Country

United States

Facility Name

East Carolina University

City

Greenville

State/Province

North Carolina

ZIP/Postal Code

27834

Country

United States

Facility Name

Cleveland Clinic

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44195

Country

United States

Facility Name

Hospital of the University of Pennsylvania

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

University of Pittsburgh Medical Center

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15232

Country

United States

Facility Name

UW Hospitals and Clinics

City

Madison

State/Province

Wisconsin

ZIP/Postal Code

53792

Country

United States

Facility Name

USC Health Clinics

City

Buderim

State/Province

Queensland

ZIP/Postal Code

4556

Country

Australia

Facility Name

Ballarat Oncology & Haematology

City

Ballarat

State/Province

Victoria

ZIP/Postal Code

3350

Country

Australia

Facility Name

Monash Medical Centre

City

Clayton

State/Province

Victoria

ZIP/Postal Code

3168

Country

Australia

Facility Name

Medical University of Vienna

City

Vienna

ZIP/Postal Code

1090

Country

Austria

Facility Name

ZNA Stuivenberg

City

Antwerpen

ZIP/Postal Code

2060

Country

Belgium

Facility Name

Centre Hospitalier Jolimont

City

La Louvière

ZIP/Postal Code

7100

Country

Belgium

Facility Name

University Hospitals Leuven

City

Leuven

ZIP/Postal Code

3000

Country

Belgium

Facility Name

St. Michael's Hospital

City

Toronto

State/Province

Ontario

ZIP/Postal Code

M5B1W8

Country

Canada

Facility Name

McGill University Health Center

City

Montréal

State/Province

Quebec

ZIP/Postal Code

H4A3J1

Country

Canada

Facility Name

University of Alberta

City

Edmonton

ZIP/Postal Code

T6G1Z1

Country

Canada

Facility Name

CHU de Caen

City

Caen

ZIP/Postal Code

14033

Country

France

Facility Name

Centre Hospitalier Henri Mondor

City

Créteil

ZIP/Postal Code

94000

Country

France

Facility Name

Centre Hospitalier Lyon Sud

City

Lyon

ZIP/Postal Code

69495

Country

France

Facility Name

Gemeinschaftspraxis Hämatologie-Onkologie

City

Dresden

ZIP/Postal Code

1307

Country

Germany

Facility Name

Universitätsklinikum Essen

City

Essen

ZIP/Postal Code

45147

Country

Germany

Facility Name

Univ Ulm, Inst Klin. Transfusions. Immungen

City

Ulm

ZIP/Postal Code

89081

Country

Germany

Facility Name

Hadassah Medical Center

City

Jerusalem

ZIP/Postal Code

91120

Country

Israel

Facility Name

Laniado Hospital

City

Netanya

ZIP/Postal Code

4244916

Country

Israel

Facility Name

Tel Aviv Sourasky Medical Center

City

Tel Aviv

ZIP/Postal Code

64239

Country

Israel

Facility Name

A. O. Spedali Civili di Brescia

City

Brescia

ZIP/Postal Code

25123

Country

Italy

Facility Name

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

City

Milan

ZIP/Postal Code

20122

Country

Italy

Facility Name

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

City

Rome

ZIP/Postal Code

00168

Country

Italy

Facility Name

U.O.C. Ematologia Ospedale San Bortolo

City

Vicenza

ZIP/Postal Code

36100

Country

Italy

Facility Name

Tokai University Hospital

City

Isehara

State/Province

Kanagawa

ZIP/Postal Code

259-1193

Country

Japan

Facility Name

Saitama Medical University Hospital

City

Iruma-gun

State/Province

Saitama-Ken

ZIP/Postal Code

350-0495

Country

Japan

Facility Name

Juntendo University Nerima Hospital

City

Tokyo

State/Province

Tokyo-To

ZIP/Postal Code

177-8521

Country

Japan

Facility Name

Academisch Medisch Centrum

City

Amsterdam

ZIP/Postal Code

1105

Country

Netherlands

Facility Name

Haukeland University Hospital

City

Bergen

ZIP/Postal Code

5053

Country

Norway

Facility Name

Oslo University Hospital

City

Oslo

ZIP/Postal Code

0372

Country

Norway

Facility Name

St Olavs Hospital, Avdeling for blodsykdommer

City

Trondheim

ZIP/Postal Code

7030

Country

Norway

Facility Name

Hospital Universitario Puerta de Hierro

City

Majadahonda

State/Province

Madrid

ZIP/Postal Code

28222

Country

Spain

Facility Name

Hospital Clinci i Provincial de Barcelona

City

Barcelona

ZIP/Postal Code

08036

Country

Spain

Facility Name

Hospital Universitario Virgen del Rocio

City

Sevilla

ZIP/Postal Code

41013

Country

Spain

Facility Name

Hospital Universitario Dr. Peset

City

Valencia

ZIP/Postal Code

46017

Country

Spain

Facility Name

St James Hospital, Leeds

City

Leeds

ZIP/Postal Code

LS9 7TF

Country

United Kingdom

Facility Name

University College London

City

London

ZIP/Postal Code

WC1E 6AG

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing 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

Citations:

Citation

Alexander Röth, Wilma Barcellini, Shirley D'Sa, Yoshitaka Miyakawa, Catherine M Broome, Marc Michel, David J. Kuter, Bernd Jilma, Tor Henrik Anderson Tvedt, Stella Lin, Xiaoyu Jiang, Caroline Reuter, William Hobbs, Sigbjørn Berentsen; Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase 3 Cardinal Study. Blood 2019; 134 (Supplement_2): LBA-2. doi: https://doi.org/10.1182/blood-2019-132490

Results Reference

result

PubMed Identifier

35999387

Citation

Roth A, Barcellini W, Tvedt THA, Miyakawa Y, Kuter DJ, Su J, Jiang X, Hobbs W, Arias JM, Shafer F, Weitz IC. Sutimlimab improves quality of life in patients with cold agglutinin disease: results of patient-reported outcomes from the CARDINAL study. Ann Hematol. 2022 Oct;101(10):2169-2177. doi: 10.1007/s00277-022-04948-y. Epub 2022 Aug 23.

Results Reference

derived

PubMed Identifier

34778998

Citation

Tvedt THA, Steien E, Ovrebo B, Haaverstad R, Hobbs W, Wardecki M, Tjonnfjord GE, Berentsen SA. Sutimlimab, an investigational C1s inhibitor, effectively prevents exacerbation of hemolytic anemia in a patient with cold agglutinin disease undergoing major surgery. Am J Hematol. 2022 Feb 1;97(2):E51-E54. doi: 10.1002/ajh.26409. Epub 2021 Dec 11. No abstract available.

Results Reference

derived

PubMed Identifier

33826820

Citation

Roth A, Barcellini W, D'Sa S, Miyakawa Y, Broome CM, Michel M, Kuter DJ, Jilma B, Tvedt THA, Fruebis J, Jiang X, Lin S, Reuter C, Morales-Arias J, Hobbs W, Berentsen S. Sutimlimab in Cold Agglutinin Disease. N Engl J Med. 2021 Apr 8;384(14):1323-1334. doi: 10.1056/NEJMoa2027760.

Results Reference

derived

Links:

URL

https://doi.org/10.1182/blood-2019-132490

Description

Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase 3 Cardinal Study

Learn more about this trial

A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion (Cardinal Study)

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A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion (Cardinal Study)

Agglutinin Disease, Cold

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial