Observational program for sickle cell disease management and outcomes

Sickle Cell Clinical Research and Intervention Program

This study is tracking people with sickle cell disease from childhood to adulthood to see how their condition changes over time and how treatments like hydroxyurea affect their health.

Quick facts

• Study type: Observational
• Enrollment: 10000 (estimated)
• Sex: All
• Sponsor: St. Jude Children's Research Hospital (Academic / other)
• Locations: 6 sites (Peoria, Illinois and 5 other locations)
• Trial ID: NCT02098863 on ClinicalTrials.gov

What this trial studies

This observational program aims to systematically collect data on individuals with sickle cell disease (SCD) from birth to end of life, focusing on the progression of chronic end-organ dysfunction and the impact of disease-modifying therapies like hydroxyurea. Participants will be followed through their transition from pediatric to adult care at designated centers in Memphis, TN, and affiliated locations. The study will involve regular clinic visits, both in-person and remote, to monitor disease progression and complications. By understanding the long-term implications of pediatric sickle cell care, the program seeks to fill critical knowledge gaps in SCD management.

Who should consider this trial

Good fit: Ideal candidates include individuals diagnosed with sickle cell disease of any genotype, particularly those transitioning from pediatric to adult care.

Not a fit: Patients with medical or social conditions that would make participation ill-advised, or those unable to complete required procedures, may not benefit from this program.

Why it matters

Potential benefit: If successful, this program could lead to improved management strategies and better long-term outcomes for patients with sickle cell disease.

How similar studies have performed: Previous studies on sickle cell disease have shown success in understanding disease progression and treatment impacts, making this approach a continuation of established research.

Eligibility criteria

SCCRIP Inclusion Criteria:

* A diagnosis of sickle cell disease of any genotype.
* PK Sub-study Inclusion Criteria:

  * Participants at St. Jude Children's Research Hospital who are consented to the parent protocol (SCCRIP, Amendment 6.1 or above).
  * Participants currently completing a hydroxyurea (HU) regimen, who have achieved maximum tolerated dose and have maintained that dose for a minimum of 90 days prior to enrollment.

SCCRIP Exclusion Criteria:

* Any medical or social reason, which, in the opinion of the principal investigators would make the participation of the subject ill-advised.
* PK Sub-study Exclusion Criteria:

  * Participants unable to complete the blood draws required for PK sampling.
  * Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
  * Any medical or social reason, which, in the opinion of the principal investigators would make the participation of the subject ill-advised.

Where this trial is running

Peoria, Illinois and 5 other locations

• Children's Hospital of Illinois at OSF-Saint Francis Medical Center — Peoria, Illinois, United States (Recruiting)
• Our Lady of the Lake Regional Medical Center — Baton Rouge, Louisiana, United States (Completed)
• Novant Health Hemby Children's Hospital — Charlotte, North Carolina, United States (Recruiting)
• Regional One Health, Diggs-Kraus Sickle Cell Center — Memphis, Tennessee, United States (Recruiting)
• Methodist Adult Comprehensive Sickle Cell Center — Memphis, Tennessee, United States (Recruiting)
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)

Study contacts

• Principal investigator: Clifford Takemoto, MD — St. Jude Children's Research Hospital
• Study coordinator: Clifford Takemoto, MD

Conditions Sickle Cell Disease; Sickle Cell Anemia; Survival; Mortality; End-Order Dysfunction