Sickle Cell DiseaseNCT04388241
Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD
A study testing The Balance Program in people with Sickle Cell Disease and Pain, run by Children's National Research Institute at 1 site in United States.
Trial summary
Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain. SCD-related pain is associated with significant functional impairment, spanning poor school attendance, decreased quality of life, and stress and mood difficulties. Read more about this study
- Who is it for?
- Ages 8 to 17 years
- Conditions the study is for
- Sickle Cell Disease, Pain
- What is being tested?
- The Balance Program
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Sep 2020to 4 Aug 2022
- 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 8 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children and adolescents with SCD (HbSS, HbSC, HbS-beta0 thalassemia, or HbS-beta+ thalassemia) between the ages of 8 and 17 years old with pain-related disability and who are currently prescribed short- or long-acting opioid medications.
- Participants will meet at least one of the following criteria for pain-related disability: 1) Have had at least 3 pain crises in the last year, 2) Have had at least one admission for pain in the last year, or 3) Have missed at least one week of school (5 days) in the last year
- Current prescription of opioids will be confirmed by participants' primary hematologist, review of the electronic medical record, and discussion with the family.
Who cannot join
- Patients and caregivers with limited English proficiency, a neurodevelopmental delay, or a visual or motor impairment that would interfere with their ability to complete the assessments and intervention.
- Documented history of major depressive disorder in medical record and/or through discussion with their primary hematology suggesting that patients may need a specific and higher level of therapeutic care.
- Patients have regularly scheduled outpatient therapy sessions outside of the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: Preliminary Feasibility and Efficacy of The Balance Program to Reduce the Impact of Pain on Daily Functioning in Pediatric Sickle Cell Disease
Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain. SCD-related pain is associated with significant functional impairment, spanning poor school attendance, decreased quality of life, and stress and mood difficulties. Pharmacological approaches are the first-line treatment for SCD-related pain, but these can be costly and have unwanted side effects. Given limitations from pharmacological approaches and the influence that poor behavioral responses have on disease management and health outcomes suggest a critical need for alternative and adjunctive treatments. Due to gaps in available behavioral treatments specifically designed for addressing common challenges associated with pain management in pediatric SCD, the investigators developed a manualized behavioral therapy protocol by tailoring existing evidence-based treatments. The overall goal of the intervention is to reduce the impact of pain on daily functioning in pediatric SCD. This study will empirically test the feasibility and preliminary efficacy of this intervention for youth with SCD. Children and adolescents with SCD between the ages of 8 and 17 years old (n=20) will be recruited to complete the treatment protocol. Feasibility will be assessed by examining participation and program completion rates, as well as feedback from a treatment acceptability questionnaire and qualitative interview. Participants will complete baseline assessments, weekly questionnaires, and post-treatment assessments (post-intervention assessment, follow-up time points: 1-month following the intervention, and 3-months following the intervention).
- Conditions
- Sickle Cell Disease, Pain
- Being tested
- The Balance Program
- Medical subject headings
- Anemia, Sickle Cell, Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
The Balance ProgramBehavioural
The Balance Program is a four-week behavioral intervention (each session lasting 60 minutes) developed specifically for the pediatric SCD population based on existing evidence-based treatments to target the unique presentation of pain-related disability in SCD.
| Study group | Role | What they receive |
|---|---|---|
| Behavioral Intervention | Experimental | Children and adolescents with SCD between the ages of 8 and 17 years old (n=20) will be recruited to complete a four-week behavioral intervention designed to reduce pain-related impairment in SCD. Receives: Behavioral: The Balance Program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility assessed by program completion rates
Feasibility will be determined by examining the proportion of participants (e.g., 80%) that complete all four sessions.
Measured: Following the completion of the intervention (5 weeks from baseline)
Acceptability assessed by satisfaction ratings and report
Acceptability will be determined by examining the satisfaction with the treatment on the Treatment Evaluation Inventory (TEI) (i.e., >80% reporting at least a "moderate" acceptability on the TEI) as well as through analysis of qualitative interviews.
Measured: Following the completion of the intervention (5 weeks from baseline)
Preliminary efficacy of the intervention in changing pain-related outcomes
Efficacy will be determined by examining self- and parent-reported changes in pain-related disability and behaviors at post-intervention and one- and three- month follow-up compared to baseline
Measured: Following the completion of the intervention (5 weeks from baseline, 1-month follow up, 3-month follow up))
Preliminary efficacy of the intervention in changing pain-related outcomes as measured by medication use
Efficacy will also be determined by examining changes in opioid use, as measured by number of prescriptions filled and self-reported use, at post-intervention and one- and three- month follow-up compared to baseline.
Measured: Following the completion of the intervention (5 weeks from baseline, 1-month follow up, 3-month follow up))
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2020
- Study start23 Sep 2020
- Primary completion22 Jun 2022
- Study completion4 Aug 2022
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 Children's National Research InstituteThe 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Megan Connolly, Children's National Research Institute
- Investigators
- Megan Connolly, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04388241
- Sponsor's protocol ID
- Pro00013663
- Status last verified
- Sep 2022
- First submitted
- 11 May 2020
- Last updated
- 6 Sep 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Preliminary Feasibility and Efficacy of The Balance Program to Reduce the Impact of Pain on Daily Functioning in Pediatric Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT04388241
- Acronym / sponsor ID
- Pro00013663
- Conditions
- Sickle Cell Disease, Pain
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 23 Sep 2020 (actual)
- Primary completion
- 22 Jun 2022 (actual)
- Study completion
- 4 Aug 2022 (actual)
- First posted
- 14 May 2020 (actual)
- 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.
Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain. SCD-related pain is associated with significant functional impairment, spanning poor school attendance, decreased quality of life, and stress and mood difficulties. Pharmacological approaches are the first-line treatment for SCD-related pain, but these can be costly and have unwanted side effects. Given limitations from pharmacological approaches and the influence that poor behavioral responses have on disease management and health outcomes suggest a critical need for alternative and adjunctive treatments. Due to gaps in available behavioral treatments specifically designed for addressing common challenges associated with pain management in pediatric SCD, the investigators developed a manualized behavioral therapy protocol by tailoring existing evidence-based treatments. The overall goal of the intervention is to reduce the impact of pain on daily functioning in pediatric SCD. This study will empirically test the feasibility and preliminary efficacy of this intervention for youth with SCD. Children and adolescents with SCD between the ages of 8 and 17 years old (n=20) will be recruited to complete the treatment protocol. Feasibility will be assessed by examining participation and program completion rates, as well as feedback from a treatment acceptability questionnaire and qualitative interview. Participants will complete baseline assessments, weekly questionnaires, and post-treatment assessments (post-intervention assessment, follow-up time points: 1-month following the intervention, and 3-months following the intervention).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Behavioral Intervention | Experimental | Behavioral: The Balance ProgramChildren and adolescents with SCD between the ages of 8 and 17 years old (n=20) will be recruited to complete a four-week behavioral intervention designed to reduce pain-related impairment in SCD. |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children and adolescents with SCD (HbSS, HbSC, HbS-beta0 thalassemia, or HbS-beta+ thalassemia) between the ages of 8 and 17 years old with pain-related disability and who are currently prescribed short- or long-acting opioid medications.
Key exclusion criteria (3 listed)
- Patients and caregivers with limited English proficiency, a neurodevelopmental delay, or a visual or motor impairment that would interfere with their ability to complete the assessments and intervention.
- Documented history of major depressive disorder in medical record and/or through discussion with their primary hematology suggesting that patients may need a specific and higher level of therapeutic care.
- Patients have regularly scheduled outpatient therapy sessions outside of the 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 |
|---|---|---|
| The Balance ProgramThe Balance Program is a four-week behavioral intervention (each session lasting 60 minutes) developed specifically for the pediatric SCD population based on existing evidence-based treatments to target the unique presentation of pain-related disability in SCD. | Behavioural | Behavioral Intervention |
Primary outcome
Feasibility assessed by program completion rates
Time frame: Following the completion of the intervention (5 weeks from baseline)
Feasibility will be determined by examining the proportion of participants (e.g., 80%) that complete all four sessions.
Acceptability assessed by satisfaction ratings and report
Time frame: Following the completion of the intervention (5 weeks from baseline)
Acceptability will be determined by examining the satisfaction with the treatment on the Treatment Evaluation Inventory (TEI) (i.e., >80% reporting at least a "moderate" acceptability on the TEI) as well as through analysis of qualitative interviews.
Preliminary efficacy of the intervention in changing pain-related outcomes
Time frame: Following the completion of the intervention (5 weeks from baseline, 1-month follow up, 3-month follow up))
Efficacy will be determined by examining self- and parent-reported changes in pain-related disability and behaviors at post-intervention and one- and three- month follow-up compared to baseline
Preliminary efficacy of the intervention in changing pain-related outcomes as measured by medication use
Time frame: Following the completion of the intervention (5 weeks from baseline, 1-month follow up, 3-month follow up))
Efficacy will also be determined by examining changes in opioid use, as measured by number of prescriptions filled and self-reported use, at post-intervention and one- and three- month follow-up compared to baseline.
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 6 Sep 2022.
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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.
Megan Connolly principal investigator
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing The Balance Program in people with Sickle Cell Disease and Pain, 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 3 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 6 Sep 2022. 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?
Feasibility assessed by program completion rates (time frame: Following the completion of the intervention (5 weeks from baseline)). The study lists 4 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 6 Sep 2022.