Clinical trial NCT05020873: Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India
Official title: Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India (SPOTLIGHT)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Sickle Cell Disease
Keywords: NIS
sickle cell disease
SCD
crizanlizumab
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-08-25
Enrollment: 44
Sex: ALL
Minimum age (years): 16
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: patients with SCD initiated on treatment with commercially available crizanlizumab at sites

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.

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

1. Patients who did not provide informed consent.
2. Patients who received a stem cell transplant at time of enrollment.
3. 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.
4. 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.

Primary outcomes:
measure: Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit
timeFrame: 12 months
Secondary outcomes:
measure: Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Duration of use of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Number of healthcare visit and home-managed (VOCs) leading to hospitalization
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Type of healthcare visit and home-managed (VOCs) leading to hospitalization
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Duration of healthcare visit and home-managed (VOCs) leading to hospitalization
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Number of home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S.
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Annualized rate home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S.
timeFrame: month 12 and month 24
measure: Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Percentage of patients with acute and chronic complications/ end organ damage related to SCD
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Frequency of patients on blood transfusions
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Frequency of patients with SCD-related Healthcare Resource Utilization (HRU)
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
measure: Number of patients with clinical laboratory parameters abnormalities
timeFrame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Other outcomes:
Locations:
city: Manama
country: Bahrain
facility: Novartis Investigative Site
city: Al Ahmadi
country: Kuwait
facility: Novartis Investigative Site
city: Doha
country: Qatar
facility: Novartis Investigative Site
city: Dammam
country: Saudi Arabia
facility: Novartis Investigative Site
city: Jazan
country: Saudi Arabia
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: