Sickle Cell DiseaseSCD_CrossNCT01369160
Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease
An observational study of Sickle Cell Disease, run by Emory University at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 to 23 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Quality of Life measures
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 33 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2005to Mar 2014
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 23 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- Inadequate medical records to support eligibility criteria
- Patients less than 1 year from start of therapy (CT, HU, MSD-BMT or SCC).
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, 30322
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: Curative vs Disease-Modifying Therapies in Children With Severe Sickle Cell Disease: A Pilot, Cross-Sectional Study
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Quality of Life measures
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Quality of Life measuresBehavioural
measuring QOL with different therapies
| Study group | Role | What they receive |
|---|---|---|
| 1 | Chronic Transfusion Receives: Behavioral: Quality of Life measures | |
| 2 | hydroxyurea Receives: Behavioral: Quality of Life measures | |
| 3 | matched sibling donor stem cell transplantation (MSD-SCT) Receives: Behavioral: Quality of Life measures | |
| 4 | standard comprehensive care (SCC, control) Receives: Behavioral: Quality of Life measures |
What is being measured
The primary outcome is the main question the study is designed to answer.
quality of life
Measured: 5 years after last patient enrolled
neuropsychiatric testing
Measured: 1 year after last patient enrolled
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2011 (estimated)
- Study startMay 2005
- Primary completionMay 2007
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ann E. Haight, Emory University
- Investigators
- Ann Haight, MD, Principal investigator, Children's Healthcare of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01369160
- Sponsor's protocol ID
- 261-2005
- Acronym
- SCD_Cross
- Status last verified
- May 2014
- First submitted
- 22 Apr 2009
- Last updated
- 28 May 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Curative vs Disease-Modifying Therapies in Children With Severe Sickle Cell Disease: A Pilot, Cross-Sectional Study
- ClinicalTrials.gov ID
- NCT01369160
- Acronym / sponsor ID
- SCD_Cross / 261-2005
- Conditions
- Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- May 2005
- Primary completion
- May 2007 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 8 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1 | – | Behavioral: Quality of Life measuresChronic Transfusion |
| 2 | – | Behavioral: Quality of Life measureshydroxyurea |
| 3 | – | Behavioral: Quality of Life measuresmatched sibling donor stem cell transplantation (MSD-SCT) |
| 4 | – | Behavioral: Quality of Life measuresstandard comprehensive care (SCC, control) |
Who may qualify
- Ages
- 3 to 23 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (2 listed)
- Inadequate medical records to support eligibility criteria
- Patients less than 1 year from start of therapy (CT, HU, MSD-BMT or SCC).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Quality of Life measuresmeasuring QOL with different therapies | Behavioural | 1, 2, 3, 4 |
Primary outcome
quality of life
Time frame: 5 years after last patient enrolled
neuropsychiatric testing
Time frame: 1 year after last patient enrolled
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ann Haight, MD principal investigator
Children's Healthcare of Atlanta
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Emory University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
quality of life (time frame: 5 years after last patient enrolled). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2014.