NCT06662812

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,665

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Oct 2021

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 6, 2021

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2023

Completed
11 months until next milestone

First Submitted

Initial submission to the registry

October 27, 2024

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 29, 2024

Completed
Last Updated

October 29, 2024

Status Verified

October 1, 2024

Enrollment Period

2.2 years

First QC Date

October 27, 2024

Last Update Submit

October 27, 2024

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

  • 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.

    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

    Up to 2 years

  • Number of Patients in the GRNDaD Registry Using Hydroxyurea Categorized by Genotype

    Up to 2 years

  • Number of Patients in the GRNDaD Registry Using Hydroxyurea Categorized by Age

    Up to 2 years

  • Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Gender

    Up to 2 years

  • Number of Patients in the GRNDaD Registry Using Voxelotor Categorized by Genotype

    Up to 2 years

  • +14 more secondary outcomes

Study Arms (1)

GRNDaD Cohort

Patients with sickle cell disease (SCD) who were included in the GRNDaD registry and received a disease modifying therapy (DMT).

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

This was a retrospective, noninterventional cohort study.

You may qualify if:

  • Diagnosis of SCD.
  • Signed consent to participate in GRNDaD registry.

You may not qualify if:

  • None identified at this time.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Novartis Pharmaceuticals

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 27, 2024

First Posted

October 29, 2024

Study Start

October 6, 2021

Primary Completion

December 1, 2023

Study Completion

December 1, 2023

Last Updated

October 29, 2024

Record last verified: 2024-10

Locations

US(1)