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

Terminated

• Conditions: Sickle Cell Disease
• Interventions: Other: Crizanlizumab

• Registration Number: NCT05020873
• Lead Sponsor: Novartis Pharmaceuticals

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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2021-08-19
• Study First Submitted QC: 2021-08-19
• Study First Posted: 2021-08-25
• Study Start: 2021-11-04
• Primary Completion: 2023-08-31
• Study Completion: 2023-08-31
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-21
• Last Update Posted: 2024-06-24

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 44

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.

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Crizanlizumab	Crizanlizumab	Patients initiated on treatment with commercially available crizanlizumab

Primary Outcome Measures

Name	Time	Method
Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit	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 Outcome Measures

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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇸🇦 Jazan, Saudi Arabia