Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease

Completed

Conditions studied: Sickle Cell Disease

In brief

The research proposed is a pilot study of pediatric and adolescent/young adult patients who have received the curative intervention (MSD-SCT), disease-modifying interventions (HU or CT) or SCC (control), with respect to three clinically important outcomes: quality-of-life (QOL), neurocognitive function, and reproductive potential. Comparable cohorts will be identified for each of the groups, drawing from patients treated by the SCD program of Children's Healthcare of Atlanta (CHOA). QOL measures and neuropsychiatric testing and will be administered. Reproductive endocrine function markers (laboratory studies and pubertal staging), will be collected and analyzed. A tracking system of such patients will also be developed, gathering available retrospective data and setting up a mechanism for collection of new data.

Key facts

Study ID

NCT01369160

Run by

Emory University

People needed

33

Starts

2005-05-01

Expected to finish

2014-03-01

Last updated by the study team

2014-05-28

Who can join

Age: 3 and older, up to 23. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Greater than or equal to 3 years of age
• Homozygous hemoglobin S (HbSS)
• Severe disease, defined as having one or more of the following:
• recurrent (2 or more episodes per year) acute chest syndrome (ACS),
• frequent (3 or more episodes per year) vaso-occlusive pain events, defined as episode lasting 4 hours and requiring hospitalization or outpatient treatment with parenteral narcotics
• Any combination of 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above) yearly for 3 years.
• any stroke, defined as central nervous system (CNS) event lasting longer than 24 hours, plus objective imaging evidence of CNS vasculopathy, with or without residual neurologic findings
• At least one year has elapsed since start of therapy for severe disease (CT, HU, MSD-BMT or SCC).

You may not qualify if…

• Inadequate medical records to support eligibility criteria
• Patients less than 1 year from start of therapy (CT, HU, MSD-BMT or SCC).

Where it is running

• Children's Healthcare of Atlanta/Emory University — Atlanta, Georgia, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States

Full record on ClinicalTrials.gov

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