Sickle Cell AnemiaPhase 2NCT02090296
Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Anemia, run by Children's National Research Institute at 1 site in United States.
Trial summary
Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications… Read more about this study
- Who is it for?
- Ages 6 to 12 months
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- Hydroxyurea
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2014to Jun 2015
- Phase
- Phase 2
- 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 6 to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- ages 6-12 months
- Sickle cell anemia (HbSS)
- steady state absolute reticulocyte count between 2-6 months is available in the medical record
Who cannot join
- receiving hydroxyurea or chronic monthly blood transfusions
- patient enrolled in preliminary study
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: Reticulocyte as Risk Marker: Targeted Therapy for Infants With Sickle Cell Anemia
Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications, while others have few serious complications. If SCA severity could be predicted early in life, those patients at greatest risk for complications could receive treatment prior to the onset of organ damage. No general SCA severity predictor or one that can be informative early in life exists. The investigators preliminary research has identified the absolute reticulocyte count (ARC) as a potential early predictive risk marker for SCA complications in pediatric patients. A higher ARC between ages 2 and 6 months of age is associated with an increased risk of hospitalization in the first 3 years of life; the mean ARC for the 36 patients who were hospitalized for SCA complications was significantly higher than that of the remaining 23 in those who were not hospitalized. Moreover, total hospitalizations were nearly three times higher by age 2 years in those infants who had an ARC of > 200 than for those infants whose ARC was <200. The proposed study will determine if ARC can be used as a risk-stratifier in asymptomatic infants with SCA and ascertain its value in targeting hydroxyurea therapy to those infants at highest risk of SCA sequelae.
- Conditions
- Sickle Cell Anemia
- Being tested
- Hydroxyurea, Placebo
- 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
Also called Hydrea
PlaceboOther
Also called Sugar water
| Study group | Role | What they receive |
|---|---|---|
| Sugar water | Placebo comparator | Placebo arm Receives: Other: Placebo |
| Hydroxyurea | Active comparator | Treatment Arm Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months)
Participants will be have a study visit at baseline and then every month for the 18 month duration of the study. Study visits will include a physical exam, laboratory monitoring and interval history to assess the change (if any) every 4 weeks of sickle cell complications.
Measured: Every 4 weeks for the 18 months study duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2014 (estimated)
- Study startFeb 2014
- Primary completionJun 2015
- Study completionJun 2015
Why it stopped: Participant withdrew consent
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.
- 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Emily Riehm Meier, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02090296
- Sponsor's protocol ID
- RBTSCA
- Status last verified
- Oct 2017
- First submitted
- 4 Feb 2014
- Last updated
- 18 Oct 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reticulocyte as Risk Marker: Targeted Therapy for Infants With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT02090296
- Acronym / sponsor ID
- RBTSCA
- Conditions
- Sickle Cell Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Participant withdrew consent
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- Feb 2014
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 18 Mar 2014 (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.
Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications, while others have few serious complications. If SCA severity could be predicted early in life, those patients at greatest risk for complications could receive treatment prior to the onset of organ damage. No general SCA severity predictor or one that can be informative early in life exists. The investigators preliminary research has identified the absolute reticulocyte count (ARC) as a potential early predictive risk marker for SCA complications in pediatric patients. A higher ARC between ages 2 and 6 months of age is associated with an increased risk of hospitalization in the first 3 years of life; the mean ARC for the 36 patients who were hospitalized for SCA complications was significantly higher than that of the remaining 23 in those who were not hospitalized. Moreover, total hospitalizations were nearly three times higher by age 2 years in those infants who had an ARC of > 200 than for those infants whose ARC was <200. The proposed study will determine if ARC can be used as a risk-stratifier in asymptomatic infants with SCA and ascertain its value in targeting hydroxyurea therapy to those infants at highest risk of SCA sequelae.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sugar water | Placebo comparator | Other: PlaceboPlacebo arm |
| Hydroxyurea | Active comparator | Drug: HydroxyureaTreatment Arm |
Who may qualify
- Ages
- 6 to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- ages 6-12 months
- Sickle cell anemia (HbSS)
- steady state absolute reticulocyte count between 2-6 months is available in the medical record
Key exclusion criteria (2 listed)
- receiving hydroxyurea or chronic monthly blood transfusions
- patient enrolled in preliminary study
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 |
|---|---|---|
| HydroxyureaAlso: Hydrea | Drug | Hydroxyurea |
| PlaceboAlso: Sugar water | Other | Sugar water |
Primary outcome
Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months)
Time frame: Every 4 weeks for the 18 months study duration
Participants will be have a study visit at baseline and then every month for the 18 month duration of the study. Study visits will include a physical exam, laboratory monitoring and interval history to assess the change (if any) every 4 weeks of sickle cell complications.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2017.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Anemia, run by Children's National Research Institute 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 3 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: withdrawn, as of the sponsor's last update on 18 Oct 2017. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months) (time frame: Every 4 weeks for the 18 months study duration). 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 18 Oct 2017; AzTrials last refreshed its copy on 28 Oct 2026.