Clinical trial NCT05791708: Cold Agglutinin Disease Real World Evidence Registry
Official title: Cold Agglutinin Disease Real World Evidence Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cold Agglutinin Disease (CAD)
Cold Agglutinin Syndrome (CAS)
Lead sponsor: Recordati Rare Diseases
Sponsor class: INDUSTRY
First posted: 2023-03-30
Enrollment: 400
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with CAD or CAS will be eligible, regardless of the type of CAD or CAS-specific therapy they are receiving. In addition, the Cadence registry will include a sutimlimab cohort study whose objective is to assess the safety and the effectiveness of sutimlimab in patients with CAD in a real-world setting.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Patterns of CAD and CAS disease characteristics
timeFrame: Up to 6 years
measure: Number of participants with CAD or CAS complications
timeFrame: Up to 6 years
measure: Patterns of use of CAD and CAS treatments
timeFrame: Up to 6 years
measure: Number of participants with health-resource utilization
timeFrame: Up to 6 years
measure: Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ)
timeFrame: Up to 6 years
measure: Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue)
timeFrame: Up to 6 years
measure: 36-Item Short Form Survey Instrument (SF-36) v2
timeFrame: Up to 6 years
measure: Sutimlimab cohort: Sutimlimab treatment patterns
timeFrame: Up to 6 years
measure: 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)
timeFrame: Up to 6 years
measure: Sutimlimab cohort: change from baseline in hemoglobin
timeFrame: From baseline up to 6 years
measure: Sutimlimab cohort: change from baseline in bilirubin
timeFrame: From baseline up to 6 years
measure: Sutimlimab cohort: change from baseline in Lactate dehydrogenase (LDH)
timeFrame: From baseline up to 6 years
measure: Sutimlimab cohort: number of blood cells transfusions
timeFrame: Up to 6 years
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham Site Number : 1230
state: Alabama
city: San Luis Obispo
country: United States
facility: SLO Oncology and Hematology Site Number : 1235
state: California
city: Santa Maria
country: United States
facility: Mission Hope Medical Oncology Site Number : 1241
state: California
city: Washington
country: United States
facility: MedStar Georgetown University Hospital / Lombardi Comprehensive Cancer Center Site Number : 1208
state: District of Columbia
city: Atlanta
country: United States
facility: Piedmont Cancer Institute, P.C Site Number : 1226
state: Georgia
city: Skokie
country: United States
facility: Orchard Heathcare Research Inc. Site Number : 1202
state: Illinois
city: Sioux City
country: United States
facility: Siouxland Regional Cancer Center dba June E. Nylen Cancer Center Site Number : 1220
state: Iowa
city: Worcester
country: United States
facility: Reliant Medical Group Site Number : 1205
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Hospital Site Number : 1232
state: Michigan
city: Lake Success
country: United States
facility: 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
state: New York
city: New York
country: United States
facility: Center for Blood Disorders Site Number : 1214
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University Site Number : 1221
state: New York
city: Greenville
country: United States
facility: Brody School of Medicine at East Carolina University Site Number : 1203
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Site Number : 1207
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Site Number : 1236
state: Ohio
city: Medford
country: United States
facility: Hematology Oncology Associates, PC Site Number : 1211
state: Oregon
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center-UPMC Hillman Cancer Center Site Number : 1210
state: Pennsylvania
city: Knoxville
country: United States
facility: UT Medical Center Cancer Institute Site Number : 1237
state: Tennessee
city: Seattle
country: United States
facility: University of Washingtion Seattle Cancer Care Alliance Site Number : 1238
state: Washington
city: Salzburg
country: Austria
facility: Investigational Site Number : 3101
city: Wien
country: Austria
facility: Investigational Site Number : 3102
city: Amiens
country: France
facility: Investigational Site Number : 3222
city: Angers
country: France
facility: Investigational Site Number : 3214
city: Cesson Sevigne
country: France
facility: Investigational Site Number : 3204
city: CHALON SUR SAONE Cedex
country: France
facility: Investigational Site Number : 3211
city: Corbeil-Essonnes
country: France
facility: Investigational Site Number : 3206
city: Creteil
country: France
facility: Investigational Site Number : 3201
city: Dijon
country: France
facility: Investigational Site Number : 3219
city: Epagny-Metz-Tessy
country: France
facility: Investigational Site Number : 3221
city: Paris
country: France
facility: Investigational Site Number : 3217
city: Pau
country: France
facility: Investigational Site Number : 3213
city: Pessac
country: France
facility: Investigational Site Number : 3203
city: Poitiers
country: France
facility: Investigational Site Number : 3216
city: Quimper
country: France
facility: Investigational Site Number : 3202
city: RENNES Cedex 09
country: France
facility: Investigational Site Number : 3220
city: Saint Priest En Jarez
country: France
facility: Investigational Site Number : 3205
city: TOULOUSE Cedex 9
country: France
facility: Investigational Site Number : 3218
city: Essen
country: Germany
facility: Investigational Site Number : 3301
city: Hannover
country: Germany
facility: Investigational Site Number : 3306
city: Landshut
country: Germany
facility: Investigational Site Number : 3302
city: Brescia
country: Italy
facility: Investigational Site Number : 3413
state: Lombardia
city: Campobasso
country: Italy
facility: Investigational Site Number : 3410
city: Novara
country: Italy
facility: Investigational Site Number : 3403
city: Palermo
country: Italy
facility: Investigational Site Number : 3411
city: Pavia
country: Italy
facility: Investigational Site Number : 3415
city: Ravenna
country: Italy
facility: Investigational Site Number : 3412
city: Reggio Calabria
country: Italy
facility: Investigational Site Number : 3402
city: Roma
country: Italy
facility: Investigational Site Number : 3404
city: Terni
country: Italy
facility: Investigational Site Number : 3405
city: Fukushima-shi
country: Japan
facility: Investigational Site Number : 2203
state: Fukushima
city: Himeji-shi
country: Japan
facility: Investigational Site Number : 2204
state: Hyogo
city: Barcelona
country: Spain
facility: Investigational Site Number : 3701
state: Barcelona [Barcelona]
city: Madrid
country: Spain
facility: Investigational Site Number : 3712
city: Truro
country: United Kingdom
facility: Investigational Site Number : 3603
state: Cornwall
city: Carlisle
country: United Kingdom
facility: Investigational Site Number : 3617
state: Cumbria
city: Gillingham
country: United Kingdom
facility: Investigational Site Number : 3608
state: Kent
city: London
country: United Kingdom
facility: Investigational Site Number : 3605
state: London, City Of
city: Salford
country: United Kingdom
facility: Investigational Site Number : 3602
state: Manchester
city: Airdrie
country: United Kingdom
facility: Investigational Site Number : 3623
state: North Lanarkshire
city: Newcastle Upon Tyne
country: United Kingdom
facility: Investigational Site Number : 3601
state: North Tyneside
city: Middlesbrough
country: United Kingdom
facility: Investigational Site Number : 3613
state: North Yorkshire
city: Oxford
country: United Kingdom
facility: Investigational Site Number : 3611
state: Oxfordshire
city: Taunton
country: United Kingdom
facility: Investigational Site Number : 3606
state: Somerset
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 3618
city: Harrow
country: United Kingdom
facility: Investigational Site Number : 3620
city: Leeds
country: United Kingdom
facility: Investigational Site Number : 3612
city: Liverpool
country: United Kingdom
facility: Investigational Site Number : 3607
city: London
country: United Kingdom
facility: Investigational Site Number : 3621
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: