Sickle Cell AnemiaNCT00519701
Hydroxyurea in Young Children With Sickle Cell Anemia
A study testing hydroxyurea in people with Sickle Cell Anemia, run by Duke University at 1 site in United States.
Trial summary
The purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage. Read more about this study
- Who is it for?
- Ages 18 months to 5 years
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2002to Feb 2007
- Phase
- Interventional
- 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 18 months to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medican Center
Durham, North Carolina, 27710
About this study
Official title: Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia
The purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage.
Read the detailed description
Previous studies have shown that hydroxyurea therapy in adults and older children with SCA improves laboratory parameters and ameliorates the clinical severity of disease. Little is known, however, about the effects of hydroxyurea on the chronic organ damage that occurs in patients with SCA and leads to significant morbidity and mortality in young adults. The objectives of this study are to assess the safety and efficacy of HU in young children with SCA and to determine whether HU preserves renal function, reduces transcranial doppler ultrasound (TCD) values, and prevents development of brain ischemia as evidenced by MRI/MRA imaging. In addition, we will evaluate the effects of hydroxyurea on quality of life
- Conditions
- Sickle Cell Anemia
- Being tested
- hydroxyurea
- Keywords
- sickle cell anemia, hydroxyurea
- 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.
hydroxyureaDrug
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | hydroxyurea Receives: Drug: hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transcranial doppler ultrasound velocity
Measured: 2 years
Magnetic resonance imaging/angiography
Measured: 2 years
Glomerular Filtration Rate
Measured: 2 years
Quality of Life
Measured: 2 years
Neurocognitive outcomes
Measured: 2 years
Secondary outcomes (2)
Growth parameters
Measured: 2 years
Hematological parameters
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2007 (estimated)
- Study startApr 2002
- Study completionFeb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by Duke 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Investigators
- Courtney D Thornburg, MD mS, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00519701
- Sponsor's protocol ID
- 3297
- Status last verified
- Aug 2007
- First submitted
- 21 Aug 2007
- Last updated
- 23 Aug 2007
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT00519701
- Acronym / sponsor ID
- 3297
- Conditions
- Sickle Cell Anemia
- Keywords
- sickle cell anemia, hydroxyurea
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- Apr 2002
- Study completion
- Feb 2007 (actual)
- First posted
- 23 Aug 2007 (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 purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage.
Detailed description
Previous studies have shown that hydroxyurea therapy in adults and older children with SCA improves laboratory parameters and ameliorates the clinical severity of disease. Little is known, however, about the effects of hydroxyurea on the chronic organ damage that occurs in patients with SCA and leads to significant morbidity and mortality in young adults. The objectives of this study are to assess the safety and efficacy of HU in young children with SCA and to determine whether HU preserves renal function, reduces transcranial doppler ultrasound (TCD) values, and prevents development of brain ischemia as evidenced by MRI/MRA imaging. In addition, we will evaluate the effects of hydroxyurea on quality of life
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: hydroxyureahydroxyurea |
Who may qualify
- Ages
- 18 months to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| hydroxyurea | Drug | 1 |
Primary outcome
Transcranial doppler ultrasound velocity
Time frame: 2 years
Magnetic resonance imaging/angiography
Time frame: 2 years
Glomerular Filtration Rate
Time frame: 2 years
Quality of Life
Time frame: 2 years
Neurocognitive outcomes
Time frame: 2 years
2 secondary outcomes
Growth parameters
Time frame: 2 years
Hematological parameters
Time frame: 2 years
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 23 Aug 2007.
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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.
Courtney D Thornburg, MD mS principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing hydroxyurea in people with Sickle Cell Anemia, run by Duke University at 1 site 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 1 inclusion and 0 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 23 Aug 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Transcranial doppler ultrasound velocity (time frame: 2 years). The study lists 5 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 23 Aug 2007.