Sickle Cell Disease Clinical Trial
— SPOTLIGHTOfficial title:
Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India (SPOTLIGHT)
| Verified date | January 2024 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
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.
| Status | Terminated |
| Enrollment | 44 |
| Est. completion date | August 31, 2023 |
| Est. primary completion date | August 31, 2023 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 16 Years to 99 Years |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| Bahrain | Novartis Investigative Site | Manama | |
| Kuwait | Novartis Investigative Site | Al Ahmadi | |
| Qatar | Novartis Investigative Site | Doha | |
| Saudi Arabia | Novartis Investigative Site | Dammam | |
| Saudi Arabia | Novartis Investigative Site | Jazan | |
| United Arab Emirates | Novartis Investigative Site | Al Ain Abu Dhabi |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
Bahrain, Kuwait, Qatar, Saudi Arabia, United Arab Emirates,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit | 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. |
12 months | |
| Secondary | Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home. | Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home. | Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Duration of use of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home. | 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 | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Number of healthcare visit and home-managed (VOCs) leading to hospitalization | Number of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Type of healthcare visit and home-managed (VOCs) leading to hospitalization | Type of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Duration of healthcare visit and home-managed (VOCs) leading to hospitalization | Duration of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization | Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected. | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Number of home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S. | 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 |
Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Annualized rate home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S. | 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 |
month 12 and month 24 | |
| Secondary | Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit | Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Percentage of patients with acute and chronic complications/ end organ damage related to SCD | Percentage of patients with acute and chronic complications/ end organ damage related to SCD to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Frequency of patients on blood transfusions | Frequency of patients on blood transfusions to be collected. Blood transfusions: simple/ exchange/ chronic/ episodic | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Frequency of patients with SCD-related Healthcare Resource Utilization (HRU) | Frequency of patients with SCD-related Healthcare Resource Utilization (HRU) to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | |
| Secondary | Number of patients with clinical laboratory parameters abnormalities | Number of patients with clinical laboratory parameters abnormalities to be collected | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months |
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