Sickle Cell DiseasePhase 3NCT01411280
Ameliorating Attention Problems in Children With Sickle Cell Disease (SCD)
A Phase 3 study testing methylphenidate in people with Sickle Cell Disease, run by Temple University at 2 sites in United States.
Trial summary
The purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on laboratory-based measures of sustained attention, reaction time, and executive functions, and indirectly… Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- methylphenidate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 34 participants
- Where
- 2 sitesUnited States
- Study dates
- Jun 2006to May 2009
- Phase
- Phase 3
- 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 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed Consent can be obtained from parent or care-giver and Assent can be obtained from the child
- Children with sickle cell disease (HbSS or HbSC)
- Age range from 6 to 16 years inclusive
- English is the child's primary language
- T-score greater than or equal to 63 on either the Conners' Parent Rating Scale - Revised or the Conners' Teacher Rating
Who cannot join
- History of glaucoma for which methylphenidate is contraindicated
- Child or immediate family member has a history of a tic disorder or Tourette's syndrome
- Child is currently receiving antidepressant, anxiolytic, antipsychotic, or stimulant drug therapy
- Family history of substance abuse disorder due to potential for abuse of stimulants by caregivers or other family members
- Recent history of uncontrolled seizures (may be on anticonvulsants, provided seizures are under "reasonable" control and that the patient and family understand the risk of altered seizure control and potential interference with maintaining therapeutic levels of anticonvulsants)
- Hypothyroidism
Show all 9Show fewer
- Symptoms of affective and mood disorders
- Previously diagnosed with ADHD prior to the onset of neurological complications (e.g., stroke or silent infarct) as documented in the medical record or caregiver report.
- Mental retardation (FSIQ < 70 on WASI)
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Temple University
Philadelphia, Pennsylvania, 19140
About this study
Official title: Ameliorating Attention Problems in Children With SCD
The purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on laboratory-based measures of sustained attention, reaction time, and executive functions, and indirectly, verbal short-term and long-term memory.
Read the detailed description
Sickle cell disease (SCD) is a group of autosomal recessive disorders, affecting an estimated 1 in 400 African American newborns annually. The pathophysiology of this group of disorders involves the production of abnormal hemoglobin (HbS), which causes red blood cells to assume a rigid, sickled shape upon release of oxygen, thereby reducing their viability in circulation. Consequently, chronic anemia and system-wide ischemia result in acute painful episodes, organ system failure, and neurological complications. Among the most debilitating effects of SCD are neurological complications. Despite the mounting evidence for structural and functional involvement of the frontal systems in pediatric SCD, there have been no clinical trials designed to manage the cognitive and behavioral sequelae associated with pediatric SCD.
- Conditions
- Sickle Cell Disease
- Being tested
- methylphenidate
- 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.
methylphenidateDrug
Also called Ritalin
Ritalin 10mg, Ritalin 20mg
| Study group | Role | What they receive |
|---|---|---|
| Laboratory trial | Placebo comparator | Compare methylphenidate to placebo in an acute laboratory trial Receives: Drug: methylphenidate |
| Home/School trial | Experimental | Low dose and moderate dose methylphenidate are compared to placebo in a home and school trial Receives: Drug: methylphenidate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Conners Parent and teacher Rating Scale
Measured: 1 week
Secondary outcomes (1)
Childrens Verbal Learning Test
Measured: 4 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2011 (estimated)
- Study startJun 2006
- Primary completionMay 2009
- Study completionMay 2009
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.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Temple University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ronald T Brown, PhD, Principal investigator, Temple University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01411280
- Sponsor's protocol ID
- R21HD049244-01
- Status last verified
- Aug 2011
- First submitted
- 20 Jul 2006
- Last updated
- 30 Apr 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ameliorating Attention Problems in Children With SCD
- ClinicalTrials.gov ID
- NCT01411280
- Acronym / sponsor ID
- R21HD049244-01
- Conditions
- Sickle Cell Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Temple UniversityUniversity, hospital or other
- Study start
- Jun 2006
- Primary completion
- May 2009 (actual)
- Study completion
- May 2009 (actual)
- First posted
- 8 Aug 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 purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on laboratory-based measures of sustained attention, reaction time, and executive functions, and indirectly, verbal short-term and long-term memory.
Detailed description
Sickle cell disease (SCD) is a group of autosomal recessive disorders, affecting an estimated 1 in 400 African American newborns annually. The pathophysiology of this group of disorders involves the production of abnormal hemoglobin (HbS), which causes red blood cells to assume a rigid, sickled shape upon release of oxygen, thereby reducing their viability in circulation. Consequently, chronic anemia and system-wide ischemia result in acute painful episodes, organ system failure, and neurological complications. Among the most debilitating effects of SCD are neurological complications. Despite the mounting evidence for structural and functional involvement of the frontal systems in pediatric SCD, there have been no clinical trials designed to manage the cognitive and behavioral sequelae associated with pediatric SCD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Laboratory trial | Placebo comparator | Drug: methylphenidateCompare methylphenidate to placebo in an acute laboratory trial |
| Home/School trial | Experimental | Drug: methylphenidateLow dose and moderate dose methylphenidate are compared to placebo in a home and school trial |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Informed Consent can be obtained from parent or care-giver and Assent can be obtained from the child
- Children with sickle cell disease (HbSS or HbSC)
- Age range from 6 to 16 years inclusive
- English is the child's primary language
- T-score greater than or equal to 63 on either the Conners' Parent Rating Scale - Revised or the Conners' Teacher Rating
Key exclusion criteria (9 listed)
- History of glaucoma for which methylphenidate is contraindicated
- Child or immediate family member has a history of a tic disorder or Tourette's syndrome
- Child is currently receiving antidepressant, anxiolytic, antipsychotic, or stimulant drug therapy
- Family history of substance abuse disorder due to potential for abuse of stimulants by caregivers or other family members
- Recent history of uncontrolled seizures (may be on anticonvulsants, provided seizures are under "reasonable" control and that the patient and family understand the risk of altered seizure control and potential interference with maintaining therapeutic levels of anticonvulsants)
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 |
|---|---|---|
| methylphenidateAlso: RitalinRitalin 10mg, Ritalin 20mg | Drug | Home/School trial, Laboratory trial |
Primary outcome
Conners Parent and teacher Rating Scale
Time frame: 1 week
1 secondary outcome
Childrens Verbal Learning Test
Time frame: 4 hours
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 30 Apr 2015.
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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.
Ronald T Brown, PhD principal investigator
Temple University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing methylphenidate in people with Sickle Cell Disease, run by Temple 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 5 inclusion and 9 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 30 Apr 2015. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 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?
Conners Parent and teacher Rating Scale (time frame: 1 week). 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 30 Apr 2015.