MedPath

Cold Agglutinin Disease Real World Evidence Registry

Recruiting
Conditions
Cold Agglutinin Disease (CAD)
Cold Agglutinin Syndrome (CAS)
Interventions
Registration Number
NCT05791708
Lead Sponsor
Recordati Rare Diseases
Brief Summary

This is a multinational, multi-center, observational, prospective, longitudinal disease registry designed to collect data on participants with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS). Among them, a minimum of 30 patients with CAD treated with sutimlimab are expected to take part in the sutimlimab cohort study. Patients with CAD who have been enrolled in previous sutimlimab clinical trials (e.g., BIVV009-01/LTS16214 \[NCT02502903,CAD patients\], BIVV009-03/EFC16215 \[NCT03347396\], and BIVV009-04/EFC16216 \[NCT03347422\]) and who either completed or discontinued the corresponding clinical trial are eligible to participate in the registry.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
400
Inclusion Criteria
  1. Patient aged ≥18 years
  2. Patient able to understand the purpose of the study and who (or whose legally authorized representative) provided signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations
  3. Patient with a diagnosis of CAD or CAS as per Investigator judgment based on the diagnosis criteria listed in study protocol
Exclusion Criteria
  1. Patient with mixed warm and cold autoimmune hemolytic anemia, or warm autoimmune hemolytic anemia
  2. Patient actively participating in a CAD or CAS interventional clinical trial. After a patient completes participation in said trial, he/she may be eligible for enrollment in this registry.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
cold agglutinin disease (CAD)SutimlimabPatient with a diagnosis of CAD as per investigator judgment based on the diagnosis criteria listed in study protocol. In addition, this group includes a cohort of CAD patients treated with sutimlimab
Primary Outcome Measures
NameTimeMethod
36-Item Short Form Survey Instrument (SF-36) v2Up to 6 years
Sutimlimab cohort: number of blood cells transfusionsUp to 6 years
Patterns of use of CAD and CAS treatmentsUp to 6 years

CAD or CAS treatments received before enrollment, at enrollment, and during the follow up period will be summarized. CAD or CAS symptoms and complications, autoimmune disorders or lymphoproliferative disorders, physician assessment of treatment status, and changes in laboratory results will be described by CAD or CAS treatments.

Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ)Up to 6 years
Patterns of CAD and CAS disease characteristicsUp to 6 years

CAD or CAS characteristics at diagnosis, enrollment, and during follow up (e.g., criteria used for diagnosis, symptoms, laboratory results) will be described.

Number of participants with health-resource utilizationUp to 6 years

Health resource utilization may include hospitalizations (including length of stay), outpatient visits, emergency room visits, specific transfusion visits, nursing home/rehabilitation center stays, and home healthcare visits during registry follow-up period

Sutimlimab cohort: Sutimlimab treatment patternsUp to 6 years

Sutimlimab dosing information, reasons for dose adjustments, reasons for discontinuation will be described.

Number of participants with CAD or CAS complicationsUp to 6 years

Thromboembolic events (venous, arterial or cerebral), deaths related to CAD or CAS, other CAD/CAS complications (e.g., acute hemolytic crisis, hemolysis-related complications), and other comorbid conditions occurring before enrollment, at enrollment, and during follow up will be described.

Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue)Up to 6 years
Sutimlimab cohort: Number of participants with adverse events, serious adverse events, adverse events of special interests (AESIs), other events of interests (e.g., Treatment emergent)Up to 6 years
Sutimlimab cohort: change from baseline in hemoglobinFrom baseline up to 6 years
Sutimlimab cohort: change from baseline in Lactate dehydrogenase (LDH)From baseline up to 6 years
Sutimlimab cohort: change from baseline in bilirubinFrom baseline up to 6 years
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (68)

Investigational Site Number : 3405

🇮🇹

Terni, Italy

Investigational Site Number : 3203

🇫🇷

Pessac, France

Investigational Site Number : 3412

🇮🇹

Ravenna, Italy

University of Alabama at Birmingham Site Number : 1230

🇺🇸

Birmingham, Alabama, United States

SLO Oncology and Hematology Site Number : 1235

🇺🇸

San Luis Obispo, California, United States

Mission Hope Medical Oncology Site Number : 1241

🇺🇸

Santa Maria, California, United States

MedStar Georgetown University Hospital / Lombardi Comprehensive Cancer Center Site Number : 1208

🇺🇸

Washington, District of Columbia, United States

Piedmont Cancer Institute, P.C Site Number : 1226

🇺🇸

Atlanta, Georgia, United States

Orchard Heathcare Research Inc. Site Number : 1202

🇺🇸

Skokie, Illinois, United States

Siouxland Regional Cancer Center dba June E. Nylen Cancer Center Site Number : 1220

🇺🇸

Sioux City, Iowa, United States

Reliant Medical Group Site Number : 1205

🇺🇸

Worcester, Massachusetts, United States

Henry Ford Hospital Site Number : 1232

🇺🇸

Detroit, Michigan, United States

Northwell Health-Monter Cancer Center-North Shore University Hospital and Steven and Alexandra Cohen Children's Medical Center of New York (Schneider Children's Hospital)-North Shore Long Island Jewis Site Number : 1231

🇺🇸

Lake Success, New York, United States

Center for Blood Disorders Site Number : 1214

🇺🇸

New York, New York, United States

SUNY Upstate Medical University Site Number : 1221

🇺🇸

Syracuse, New York, United States

Brody School of Medicine at East Carolina University Site Number : 1203

🇺🇸

Greenville, North Carolina, United States

Cleveland Clinic Foundation Site Number : 1207

🇺🇸

Cleveland, Ohio, United States

The Ohio State University Site Number : 1236

🇺🇸

Columbus, Ohio, United States

Hematology Oncology Associates, PC Site Number : 1211

🇺🇸

Medford, Oregon, United States

University of Pittsburgh Medical Center-UPMC Hillman Cancer Center Site Number : 1210

🇺🇸

Pittsburgh, Pennsylvania, United States

UT Medical Center Cancer Institute Site Number : 1237

🇺🇸

Knoxville, Tennessee, United States

University of Washingtion Seattle Cancer Care Alliance Site Number : 1238

🇺🇸

Seattle, Washington, United States

Investigational Site Number : 3101

🇦🇹

Salzburg, Austria

Investigational Site Number : 3102

🇦🇹

Wien, Austria

Investigational Site Number : 3222

🇫🇷

Amiens, France

Investigational Site Number : 3214

🇫🇷

Angers, France

Investigational Site Number : 3204

🇫🇷

Cesson Sevigne, France

Investigational Site Number : 3211

🇫🇷

CHALON SUR SAONE Cedex, France

Investigational Site Number : 3206

🇫🇷

Corbeil-Essonnes, France

Investigational Site Number : 3201

🇫🇷

Creteil, France

Investigational Site Number : 3219

🇫🇷

Dijon, France

Investigational Site Number : 3221

🇫🇷

Epagny-Metz-Tessy, France

Investigational Site Number : 3217

🇫🇷

Paris, France

Investigational Site Number : 3213

🇫🇷

Pau, France

Investigational Site Number : 3216

🇫🇷

Poitiers, France

Investigational Site Number : 3202

🇫🇷

Quimper, France

Investigational Site Number : 3220

🇫🇷

RENNES Cedex 09, France

Investigational Site Number : 3205

🇫🇷

Saint Priest En Jarez, France

Investigational Site Number : 3218

🇫🇷

TOULOUSE Cedex 9, France

Investigational Site Number : 3301

🇩🇪

Essen, Germany

Investigational Site Number : 3306

🇩🇪

Hannover, Germany

Investigational Site Number : 3302

🇩🇪

Landshut, Germany

Investigational Site Number : 3413

🇮🇹

Brescia, Lombardia, Italy

Investigational Site Number : 3410

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Campobasso, Italy

Investigational Site Number : 3403

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Novara, Italy

Investigational Site Number : 3411

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Palermo, Italy

Investigational Site Number : 3415

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Pavia, Italy

Investigational Site Number : 3402

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Reggio Calabria, Italy

Investigational Site Number : 3404

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Roma, Italy

Investigational Site Number : 2203

🇯🇵

Fukushima-shi, Fukushima, Japan

Investigational Site Number : 2204

🇯🇵

Himeji-shi, Hyogo, Japan

Investigational Site Number : 3701

🇪🇸

Barcelona, Barcelona [Barcelona], Spain

Investigational Site Number : 3712

🇪🇸

Madrid, Spain

Investigational Site Number : 3603

🇬🇧

Truro, Cornwall, United Kingdom

Investigational Site Number : 3617

🇬🇧

Carlisle, Cumbria, United Kingdom

Investigational Site Number : 3608

🇬🇧

Gillingham, Kent, United Kingdom

Investigational Site Number : 3605

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London, London, City Of, United Kingdom

Investigational Site Number : 3602

🇬🇧

Salford, Manchester, United Kingdom

Investigational Site Number : 3623

🇬🇧

Airdrie, North Lanarkshire, United Kingdom

Investigational Site Number : 3601

🇬🇧

Newcastle Upon Tyne, North Tyneside, United Kingdom

Investigational Site Number : 3613

🇬🇧

Middlesbrough, North Yorkshire, United Kingdom

Investigational Site Number : 3611

🇬🇧

Oxford, Oxfordshire, United Kingdom

Investigational Site Number : 3606

🇬🇧

Taunton, Somerset, United Kingdom

Investigational Site Number : 3618

🇬🇧

Birmingham, United Kingdom

Investigational Site Number : 3620

🇬🇧

Harrow, United Kingdom

Investigational Site Number : 3612

🇬🇧

Leeds, United Kingdom

Investigational Site Number : 3607

🇬🇧

Liverpool, United Kingdom

Investigational Site Number : 3621

🇬🇧

London, United Kingdom

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