Sickle Cell DiseaseNCT06662812
Characteristics and Treatment Patterns of Patients With Sickle Cell Disease in Globin Research Network for Data and Discovery Registry
An observational study of Sickle Cell Disease, run by Novartis at 1 site in United States.
Trial summary
This was a retrospective observational cohort study. This study was a secondary analysis of individuals with sickle cell disease (SCD) enrolled in the GRNDaD registry. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 1,665 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Oct 2021to 1 Dec 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This was a retrospective, noninterventional cohort study.
Who may be able to join
- Diagnosis of SCD.
- Signed consent to participate in GRNDaD registry.
Who cannot join
- None identified at this time.
Trial locations and contacts
1 site in 1 country.
United States1 site
Novartis Pharmaceuticals
East Hanover, New Jersey, 07936
About this study
Official title: Disease Characteristics and Treatment Patterns of Patients With Sickle Cell Disease in Globin Research Network for Data and Discovery (GRNDaD) Registry
This was a retrospective observational cohort study. This study was a secondary analysis of individuals with sickle cell disease (SCD) enrolled in the GRNDaD registry.
- Conditions
- Sickle Cell Disease
- 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.
| Study group | Role | What they receive |
|---|---|---|
| GRNDaD Cohort | Patients with sickle cell disease (SCD) who were included in the GRNDaD registry and received a disease modifying therapy (DMT). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients in the Globin Research Network for Data and Discovery (GRNDaD) Registry who Received a Disease Modifying Therapy (DMT)
DMTs included: hydroxyurea, voxelotor, crizanlizumab, l-glutamine, and chronic red cell transfusion.
Measured: Up to 2 years
Secondary outcomes (19)
Number of Patients in the Globin Research Network for Data and Discovery (GRNDaD) Registry Using Hydroxyurea Categorized by Gender
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Hydroxyurea Categorized by Genotype
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Hydroxyurea Categorized by Age
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Gender
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Genotype
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Age
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Crizanlizumab Categorized by Gender
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Crizanlizumab Categorized by Genotype
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Crizanlizumab Categorized by Age
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using L-glutamine Categorized by Gender
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using L-glutamine Categorized by Genotype
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using L-glutamine Categorized by Age
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Chronic Transfusion Categorized by Gender
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Chronic Transfusion Categorized by Genotype
Measured: Up to 2 years
Number of Patients in the GRNDaD Registry Using Chronic Transfusion Categorized by Age
Measured: Up to 2 years
Number of Hospital Admissions Categorized by Age
Measured: Up to 2 years
Mean Number of Hospital Admissions
Measured: Up to 2 years
Number of Emergency Department Visits Categorized by Age
Measured: Up to 2 years
Mean Number of Emergency Department Visits
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2024
- Study start6 Oct 2021
- Primary completion1 Dec 2023
- Study completion1 Dec 2023
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.
- 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
- 1,665 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06662812
- Sponsor's protocol ID
- CSEG101AUS19
- Status last verified
- Oct 2024
- First submitted
- 27 Oct 2024
- Last updated
- 29 Oct 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Disease Characteristics and Treatment Patterns of Patients With Sickle Cell Disease in Globin Research Network for Data and Discovery (GRNDaD) Registry
- ClinicalTrials.gov ID
- NCT06662812
- Acronym / sponsor ID
- CSEG101AUS19
- Conditions
- Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,665 participants (actual)
- Lead sponsor
- NovartisIndustry (pharma, biotech, devices)
- Study start
- 6 Oct 2021 (actual)
- Primary completion
- 1 Dec 2023 (actual)
- Study completion
- 1 Dec 2023 (actual)
- First posted
- 29 Oct 2024 (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 observational cohort study. This study was a secondary analysis of individuals with sickle cell disease (SCD) enrolled in the GRNDaD registry.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GRNDaD Cohort | – | –Patients with sickle cell disease (SCD) who were included in the GRNDaD registry and received a disease modifying therapy (DMT). |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This was a retrospective, noninterventional cohort study.
Key inclusion criteria (2 listed)
- Diagnosis of SCD.
- Signed consent to participate in GRNDaD registry.
Key exclusion criteria (1 listed)
- None identified at this time.
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
Number of Patients in the Globin Research Network for Data and Discovery (GRNDaD) Registry who Received a Disease Modifying Therapy (DMT)
Time frame: Up to 2 years
DMTs included: hydroxyurea, voxelotor, crizanlizumab, l-glutamine, and chronic red cell transfusion.
19 secondary outcomes
Number of Patients in the Globin Research Network for Data and Discovery (GRNDaD) Registry Using Hydroxyurea Categorized by Gender
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Hydroxyurea Categorized by Genotype
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Hydroxyurea Categorized by Age
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Gender
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Genotype
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Age
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Crizanlizumab Categorized by Gender
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Crizanlizumab Categorized by Genotype
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Crizanlizumab Categorized by Age
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using L-glutamine Categorized by Gender
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using L-glutamine Categorized by Genotype
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using L-glutamine Categorized by Age
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Chronic Transfusion Categorized by Gender
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Chronic Transfusion Categorized by Genotype
Time frame: Up to 2 years
Number of Patients in the GRNDaD Registry Using Chronic Transfusion Categorized by Age
Time frame: Up to 2 years
Number of Hospital Admissions Categorized by Age
Time frame: Up to 2 years
Mean Number of Hospital Admissions
Time frame: Up to 2 years
Number of Emergency Department Visits Categorized by Age
Time frame: Up to 2 years
Mean Number of Emergency Department Visits
Time frame: Up to 2 years
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 29 Oct 2024.
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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
1 site 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 at 1 site 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 2 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 29 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Number of Patients in the Globin Research Network for Data and Discovery (GRNDaD) Registry who Received a Disease Modifying Therapy (DMT) (time frame: Up to 2 years). 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 29 Oct 2024.