Cold Agglutinin Disease Real World Evidence Registry

Condition(s) studied

Investigational drug(s) / intervention(s)

Sutimlimab: CAD patients in Sutimlimab cohort must be treated according to routine clinical practice

Study 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

Primary outcome measure(s)

• Patterns of CAD and CAS disease characteristics — Up 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 CAD or CAS complications — Up 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.
• Patterns of use of CAD and CAS treatments — Up 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.
• Number of participants with health-resource utilization — Up 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
• Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ) — Up to 6 years
• Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) — Up to 6 years
• 36-Item Short Form Survey Instrument (SF-36) v2 — Up to 6 years
• Sutimlimab cohort: Sutimlimab treatment patterns — Up to 6 years
  Sutimlimab dosing information, reasons for dose adjustments, reasons for discontinuation will be described.
• 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 hemoglobin — From baseline up to 6 years
• Sutimlimab cohort: change from baseline in bilirubin — From baseline up to 6 years
• Sutimlimab cohort: change from baseline in Lactate dehydrogenase (LDH) — From baseline up to 6 years
• Sutimlimab cohort: number of blood cells transfusions — Up to 6 years

Trial sites (68)

+ 28 more sites — see the full list on the official registry below.