Sickle Cell DiseaseSPOTLIGHTNCT05020873
Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India
An observational study of Sickle Cell Disease, run by Novartis Pharmaceuticals at 5 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 99 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Crizanlizumab
- How many people
- 44 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 4 Nov 2021to 31 Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patients with SCD initiated on treatment with commercially available crizanlizumab at sites
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
Saudi Arabia2 sites
Novartis Investigative Site
Dammam, 32263
Novartis Investigative Site
Jizan, 82943
About this study
Official title: Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India (SPOTLIGHT)
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Crizanlizumab
- Keywords
- NIS, sickle cell disease, SCD, crizanlizumab
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CrizanlizumabOther
Prospective observational study. There is no treatment allocation. Patients administered crizanlizumab, that have started before inclusion of the patient into the study will be enrolled.
| Study group | Role | What they receive |
|---|---|---|
| Crizanlizumab | Patients initiated on treatment with commercially available crizanlizumab Receives: Other: Crizanlizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months
Secondary outcomes (14)
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
Measured: 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.
Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home to be collected
Measured: 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.
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
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
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
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
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
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
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
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
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.
Measured: 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.
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
Measured: 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.
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
Measured: month 12 and month 24
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
Measured: 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
Percentage of patients with acute and chronic complications/ end organ damage related to SCD to be collected
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Frequency of patients on blood transfusions
Frequency of patients on blood transfusions to be collected. Blood transfusions: simple/ exchange/ chronic/ episodic
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Frequency of patients with SCD-related Healthcare Resource Utilization (HRU)
Frequency of patients with SCD-related Healthcare Resource Utilization (HRU) to be collected
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Number of patients with clinical laboratory parameters abnormalities
Number of patients with clinical laboratory parameters abnormalities to be collected
Measured: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2021
- Study start4 Nov 2021
- Primary completion31 Aug 2023
- Study completion31 Aug 2023
Why it stopped: sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05020873
- Sponsor's protocol ID
- CSEG101AIC05
- Acronym
- SPOTLIGHT
- Status last verified
- Jun 2024
- First submitted
- 19 Aug 2021
- Last updated
- 24 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India (SPOTLIGHT)
- ClinicalTrials.gov ID
- NCT05020873
- Acronym / sponsor ID
- SPOTLIGHT / CSEG101AIC05
- Conditions
- Sickle Cell Disease
- Keywords
- NIS, sickle cell disease, SCD, crizanlizumab
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: sponsor decision
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 4 Nov 2021 (actual)
- Primary completion
- 31 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 25 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Crizanlizumab | – | Other: CrizanlizumabPatients initiated on treatment with commercially available crizanlizumab |
Who may qualify
- Ages
- 16 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients with SCD initiated on treatment with commercially available crizanlizumab at sites
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CrizanlizumabProspective observational study. There is no treatment allocation. Patients administered crizanlizumab, that have started before inclusion of the patient into the study will be enrolled. | Other | Crizanlizumab |
Primary outcome
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.
14 secondary outcomes
Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Number of healthcare visit and home-managed (VOCs) leading to hospitalization
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Type of healthcare visit and home-managed (VOCs) leading to hospitalization
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Duration of healthcare visit and home-managed (VOCs) leading to hospitalization
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization
Time frame: 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.
Time frame: 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.
Time frame: month 12 and month 24
Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit
Time frame: 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
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Frequency of patients on blood transfusions
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Frequency of patients with SCD-related Healthcare Resource Utilization (HRU)
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Number of patients with clinical laboratory parameters abnormalities
Time frame: Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months
Results and safety
The study was stopped early (reason given: sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Novartis Pharmaceuticals at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Saudi Arabia, Bahrain, Kuwait, Qatar. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: sponsor decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2024.