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

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Locations
United States
Study Type
Observational
Intervention
Quality of Life measures
Sponsored by
Emory University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an observational trial for Sickle Cell Disease

Eligibility Criteria

3 Years - 23 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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).

Exclusion Criteria:

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

Sites / Locations

  • Children's Healthcare of Atlanta/Emory University
  • Children's Healthcare of Atlanta

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Arm Label

1

2

3

4

Arm Description

Chronic Transfusion

hydroxyurea

matched sibling donor stem cell transplantation (MSD-SCT)

standard comprehensive care (SCC, control)

Outcomes

Primary Outcome Measures

quality of life
neuropsychiatric testing

Secondary Outcome Measures

Full Information

First Posted
April 22, 2009
Last Updated
May 23, 2014
Sponsor
Emory University
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1. Study Identification

Unique Protocol Identification Number
NCT01369160
Brief Title
Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease
Acronym
SCD_Cross
Official Title
Curative vs Disease-Modifying Therapies in Children With Severe Sickle Cell Disease: A Pilot, Cross-Sectional Study
Study Type
Observational

2. Study Status

Record Verification Date
May 2014
Overall Recruitment Status
Completed
Study Start Date
May 2005 (undefined)
Primary Completion Date
May 2007 (Actual)
Study Completion Date
March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Emory University

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Enrollment
33 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Description
Chronic Transfusion
Arm Title
2
Arm Description
hydroxyurea
Arm Title
3
Arm Description
matched sibling donor stem cell transplantation (MSD-SCT)
Arm Title
4
Arm Description
standard comprehensive care (SCC, control)
Intervention Type
Behavioral
Intervention Name(s)
Quality of Life measures
Intervention Description
measuring QOL with different therapies
Primary Outcome Measure Information:
Title
quality of life
Time Frame
5 years after last patient enrolled
Title
neuropsychiatric testing
Time Frame
1 year after last patient enrolled

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
23 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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). Exclusion Criteria: Inadequate medical records to support eligibility criteria Patients less than 1 year from start of therapy (CT, HU, MSD-BMT or SCC).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ann Haight, MD
Organizational Affiliation
Children's Healthcare of Atlanta
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Healthcare of Atlanta/Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States

12. IPD Sharing Statement

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

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