Cure Sickle Cell Initiative recommendations on common data elements for sickle cell disease gene therapy trials.

Area of Science:

• Hematology
• Gene Therapy
• Clinical Research Informatics

Background:

• Sickle cell disease (SCD) gene therapy research requires standardized data for effective analysis.
• Lack of interoperability hinders meta-analysis and comparison of clinical trial outcomes.
• The National Heart, Lung, and Blood Institute (NHLBI) established the Cure Sickle Cell Initiative (CureSCi) to address this gap.

Purpose of the Study:

• To develop standardized Common Data Elements (CDEs) for sickle cell disease (SCD) gene therapy clinical trials.
• To improve data interoperability, meta-analysis, and facilitate the development of safe and effective gene therapies for SCD.
• To provide a comprehensive resource for researchers and clinicians involved in SCD gene therapy studies.

Main Methods:

• Five working groups (WGs) involving patients, stakeholders, clinicians, and researchers convened.
• Existing data definitions from major organizations were reviewed and refined.
• A comprehensive list of CDEs was developed, incorporating public feedback over a 60-day review period.

Main Results:

• A standardized set of SCD Common Data Elements (CDEs) was created.
• Recommendations include 42 instruments and 49 template case report forms for various study stages.
• The SCD CDEs and associated resources are publicly available on the CureSCi and NIH Repository websites.

Conclusions:

• The CureSCi CDEs standardize data collection, enhancing comparability and quality across SCD gene therapy trials.
• These recommendations facilitate multi-center research and support the development of accessible gene therapies.
• The initiative aims to accelerate research and educate new investigators in the field of SCD gene therapy.