Ignite Creation Date:
2025-12-25 @ 2:11 AM
Ignite Modification Date:
2025-12-31 @ 10:49 AM
Study NCT ID:
NCT01369160
Status:
COMPLETED
Last Update Posted:
2014-05-28
First Post:
2009-04-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease
Sponsor:
Emory University
Organization:
Study Overview
Official Title:
Curative vs Disease-Modifying Therapies in Children With Severe Sickle Cell Disease: A Pilot, Cross-Sectional Study
Status:
COMPLETED
Status Verified Date:
2014-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SCD_Cross
Brief 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.
Detailed Description:
sickle cell disease (SCD), but a significant proportion experience clinically severe disease requiring more aggressive intervention. Widely applicable curative therapy with a favorable toxicity profile remains elusive for such patients.
Three distinct intervention strategies are currently available for children with severe sickle cell disease (SCD): oral hydroxyurea (HU), chronic blood transfusions (CT), and allogeneic hematopoietic stem cell transplantation (SCT) from an HLA-matched sibling donor (MSD). Each intervention has distinct advantages and disadvantages. Many patients do not receive specific intervention, and continue standard comprehensive care (SCC).
Though indications for these therapies overlap, to date there are no comparative outcomes data, leaving families and physicians without adequate information upon which to base therapeutic decisions. The gold standard for obtaining such data would be a randomized, prospective study comparing each intervention, though this may or may not be feasible to conduct. Before such a trial is considered, a large cross-sectional trial should be conducted to establish comparisons among the four therapeutic groups (HU, SCT, CT, SCC) with respect to the outcomes that clinicians and families deem most important.
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.
Three distinct intervention strategies are currently available for children with severe sickle cell disease (SCD): oral hydroxyurea (HU), chronic blood transfusions (CT), and allogeneic hematopoietic stem cell transplantation (SCT) from an HLA-matched sibling donor (MSD). Each intervention has distinct advantages and disadvantages. Many patients do not receive specific intervention, and continue standard comprehensive care (SCC).
Though indications for these therapies overlap, to date there are no comparative outcomes data, leaving families and physicians without adequate information upon which to base therapeutic decisions. The gold standard for obtaining such data would be a randomized, prospective study comparing each intervention, though this may or may not be feasible to conduct. Before such a trial is considered, a large cross-sectional trial should be conducted to establish comparisons among the four therapeutic groups (HU, SCT, CT, SCC) with respect to the outcomes that clinicians and families deem most important.
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: