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

NCT01369160 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 33

Last updated 2014-05-28

No results posted yet for this study

Summary

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.

Conditions

Interventions

BEHAVIORAL

Quality of Life measures

measuring QOL with different therapies

Sponsors & Collaborators

  • Emory University

    lead OTHER

Principal Investigators

  • Ann Haight, MD · Children's Healthcare of Atlanta

Eligibility

Min Age
3 Years
Max Age
23 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-05-31
Primary Completion
2007-05-31
Completion
2014-03-31

Countries

  • United States

Study Locations

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Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01369160 on ClinicalTrials.gov