Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India (SPOTLIGHT)

Novartis Pharmaceuticals1 site in 1 country44 target enrollmentNovember 4, 2021

ConditionsSickle Cell Disease

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Sickle Cell Disease
Sponsor: Novartis Pharmaceuticals
Enrollment: 44
Locations: 1
Primary Endpoint: Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit
Status: Terminated
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

This is a multicenter, prospective, single-arm observational non-interventional study (NIS), which will be conducted in various countries in the Middle East and India.

Detailed Description

The study is designed to collect information on the utilization and effectiveness of crizanlizumab treatment in SCD patients under routine clinical practice conditions, to which the physician has made an independent decision to prescribe crizanlizumab.

Registry

clinicaltrials.gov

Start Date

November 4, 2021

End Date

August 31, 2023

Last Updated

last year

Study Type

Observational

Sex

All

Investigators

Sponsor

Novartis Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male and female patients with clinical diagnosis of SCD (based on laboratory parameters) of any genotype.
•Patients newly initiated on treatment with locally approved crizanlizumab.
•Patients aged 16 years or older at crizanlizumab initiation.

Exclusion Criteria

•Patients who did not provide informed consent.
•Patients who received a stem cell transplant at time of enrollment.
•Patients who participated in or are participating in a clinical trial at time of enrollment or in the 12 months prior to starting commercial crizanlizumab.
•According to the investigator's opinion, the patient is an unlikely candidate to provide an accurate medical history and/or to obtain long-term follow-up information for any reasons such as unavailability or severe concomitant illnesses.

Outcomes

Primary Outcomes

Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit

Time Frame: 12 months

The annualized rate of VOCs is defined as the total number of pain crises for a patient occurring from the date of initial infusion with crizanlizumab (commercially available) to the last contact date of the study end date × 365 divided by the number of days during that same time period. This calculation accounts for early dropouts or lost to follow-up by extrapolating the VOC rate of every patient to 1 year.

Secondary Outcomes

Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Duration of healthcare visit and home-managed (VOCs) leading to hospitalization(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Frequency of patients on blood transfusions(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Type of healthcare visit and home-managed (VOCs) leading to hospitalization(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Annualized rate home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S.(month 12 and month 24)
Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Number of home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S.(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Duration of use of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Percentage of patients with acute and chronic complications/ end organ damage related to SCD(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Frequency of patients with SCD-related Healthcare Resource Utilization (HRU)(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Number of healthcare visit and home-managed (VOCs) leading to hospitalization(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)
Number of patients with clinical laboratory parameters abnormalities(Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months)