Sickle Cell DiseaseNCT05833022
Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
An observational study of Sickle Cell Disease, run by Novartis Pharmaceuticals at 11 sites in United States.
Trial summary
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab. Patients who were prescribed crizanlizumab were included in the cohort. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 297 participants
- Where
- 11 sitesUnited States
- Study dates
- 30 Jul 2021to 18 Apr 2022
- 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 years and older
- All sexes
- Child, Adult, Older adult
Study population: This was a retrospective, noninterventional cohort study
Who may be able to join
- Any patient with diagnosis of SCD based on high-performance liquid chromatography (HPLC) or hemoglobin electrophoresis at treating institution
- Treatment order for crizanlizumab; Index date was defined as the date for the first crizanlizumab treatment
- At least 12 months of available electronic medical record (EMR) data pre-index date (baseline)
- Patients must have received at least one dose of crizanlizumab
- For inclusion in pre/post analysis of effectiveness, patients must have received at least 6 doses of medication. The rates could be annualized if total post-index time was <12 months.
Who cannot join
- None specified
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Johns Hopkins University
Baltimore, Maryland, 21205
University of Alabama Birmingham
Birmingham, Alabama, 35233
University of North Carolina
Chapel Hill, North Carolina, 27514
University Hospital at Cleveland Medical Center
Cleveland, Ohio, 44106
Ohio State University
Columbus, Ohio, 43203
Beth Israel Hospital
East Hanover, New Jersey, 07936
University of Texas (Houston)
Houston, Texas, 77030
Montefiore
New York, New York, 10461
Washington University of St. Louis
St Louis, Missouri, 63110
All Children's Hospital
St. Petersburg, Florida, 33701
Cristiana Care
Wilmington, Delaware, 19801
About this study
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab. Patients who were prescribed crizanlizumab were included in the cohort.
- Conditions
- Sickle Cell Disease
- Medical subject headings
- Anemia, Sickle Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) prior to treatment with crizanlizumab
Measured: Up to 12 months
Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) during treatment with crizanlizumab
Measured: Up to 12 months
Secondary outcomes (5)
Number of patients who discontinued treatment with crizanlizumab
Measured: Up to 24 months
Number of doses of crizanlizumab for patients who discontinued treatment with crizanlizumab
Measured: Up to 24 months
Reasons for early discontinuation of crizanlizumab treatment
Measured: Up to 24 months
Number of sickle cell disease (SCD)-related hospitalizations
Measured: Up to 24 months
Number of SCD-related emergency department (ED) visits
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2023
- Study start30 Jul 2021
- Primary completion18 Apr 2022
- Study completion18 Apr 2022
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
- Retrospective
- Enrolment
- 297 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
- NCT05833022
- Sponsor's protocol ID
- CSEG101AUS16
- Status last verified
- Apr 2023
- First submitted
- 17 Apr 2023
- Last updated
- 27 Apr 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
- ClinicalTrials.gov ID
- NCT05833022
- Acronym / sponsor ID
- CSEG101AUS16
- Conditions
- Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 297 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Jul 2021 (actual)
- Primary completion
- 18 Apr 2022 (actual)
- Study completion
- 18 Apr 2022 (actual)
- First posted
- 27 Apr 2023 (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 was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab. Patients who were prescribed crizanlizumab were included in the cohort.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- This was a retrospective, noninterventional cohort study
Key inclusion criteria (5 listed)
- Any patient with diagnosis of SCD based on high-performance liquid chromatography (HPLC) or hemoglobin electrophoresis at treating institution
- Treatment order for crizanlizumab; Index date was defined as the date for the first crizanlizumab treatment
- At least 12 months of available electronic medical record (EMR) data pre-index date (baseline)
- Patients must have received at least one dose of crizanlizumab
- For inclusion in pre/post analysis of effectiveness, patients must have received at least 6 doses of medication. The rates could be annualized if total post-index time was <12 months.
Key exclusion criteria (1 listed)
- None specified
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) prior to treatment with crizanlizumab
Time frame: Up to 12 months
Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) during treatment with crizanlizumab
Time frame: Up to 12 months
5 secondary outcomes
Number of patients who discontinued treatment with crizanlizumab
Time frame: Up to 24 months
Number of doses of crizanlizumab for patients who discontinued treatment with crizanlizumab
Time frame: Up to 24 months
Reasons for early discontinuation of crizanlizumab treatment
Time frame: Up to 24 months
Number of sickle cell disease (SCD)-related hospitalizations
Time frame: Up to 24 months
Number of SCD-related emergency department (ED) visits
Time frame: Up to 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2023.
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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
11 sites in 1 country: 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 11 sites in United States.
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 5 inclusion and 1 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: finished, as of the sponsor's last update on 27 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) prior to treatment with crizanlizumab (time frame: Up to 12 months). The study lists 2 primary outcomes in all. 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 27 Apr 2023.