Sickle Cell DiseaseNCT01959958
Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population. This increased risk has been attributed to a decreased ability to concentrate urine caused by sickling-induced nephropathy. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Questionnaire/Interview
- How many people
- 250 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2013to Apr 2016
- 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 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Pediatric patients with sickle cell disease and their parents/guardians
Who may be able to join
- Child Characteristics:
- St. Jude Children's Research Hospital (SJCRH) patient with a primary diagnosis of sickle cell disease (HbSS, HbSC, HbSβ0thalassemia, HbSβ+thalassemia)
- Age 6.0-17.99 years at the time of enrollment
- Primary language is English
- Has the cognitive capacity to complete questionnaires
- Caregiver Characteristics:
Show all 9Show fewer
- Parent or guardian of SJCRH patient who meets above criteria
- Primary language is English
- Has the cognitive capacity to complete questionnaires
Who cannot join
- Child Characteristics:
- Experiencing current acute complications of sickle cell disease requiring hospitalization or an acute care visit (e.g., pain crises, acute chest syndrome, acute cerebrovascular events/stroke or active infection/fever).
- Caregiver Characteristics:
- Has a child experiencing current acute complication of sickle cell disease, such as pain crisis, acute chest syndrome, stroke, or infection.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population. This increased risk has been attributed to a decreased ability to concentrate urine caused by sickling-induced nephropathy. The sociodemographic, psychosocial, and medical factors associated with nocturnal enuresis are not well defined. In addition, the impact of these behaviors on emotional and behavioral functioning, along with health-related quality of life are not clear. Despite the availability of evidence-based interventions for nocturnal enuresis, very few families with a child with sickle cell disease have utilized these methods. The reasons for this underutilization of interventions are not clear.
Read the detailed description
PRIMARY OBJECTIVE: To examine differences between those who do and those who do not exhibit nocturnal enuresis in regard to health-related quality of life, emotional and behavioral functioning, family functioning, stressful life events, sociodemographic factors and medical factors.
SECONDARY OBJECTIVE: To identify barriers to intervention implementation for nocturnal enuresis.
In this study, children and adolescents with sickle cell disease ages 6-18 years and their parents/guardians will each complete an interview with questionnaires assessing nocturnal enuresis, health-related quality of life, emotional and behavioral functioning, family functioning, and stressful life events. Participants who report nocturnal enuresis will identify interventions they have used in the past, along with barriers to intervention implementation.
The future goal will be to use this information to develop an effective intervention strategy for nocturnal enuresis specific to the needs of children and adolescents with sickle cell disease.
- Conditions
- Sickle Cell Disease
- Being tested
- Questionnaire/Interview
- Keywords
- Nocturnal enuresis, Barriers to treatment
- Medical subject headings
- Anemia, Sickle Cell, Nocturnal Enuresis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Questionnaire/InterviewOther
Participants will each complete an interview with questionnaires assessing nocturnal enuresis, health-related quality of life, emotional and behavioral functioning, family functioning, and stressful life events. Participants who report nocturnal enuresis will also be asked to identify interventions they have used in the past, along with barriers to intervention implementation.
| Study group | Role | What they receive |
|---|---|---|
| Sickle cell disease | In this study, children and adolescents with sickle cell disease ages 6-18 years and their parents/guardians will complete a questionnaire/interview. Receives: Other: Questionnaire/Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Descriptive group characteristics of sickle cell disease participants with and without nocturnal enuresis
Health-related quality of life, emotional and behavioral functioning, family functioning, stressful life events, sociodemographic factors and medical factors will be examined from questionnaire responses. Classification will be based on the criteria outlined in the Diagnostic and Statistical Manual of Mental Disorders, 4th edition, text revision (DSM-IV-TR). Data will be stratified by age (6-11 years and 12-18 years) and genotype (HbSS/ HbSβ0 thalassemia and HbSC/ HbSβ+ thalassemia). The sample will be dichotomized using the binary response (by nocturnal enuresis/no nocturnal enuresis). Pearson's chi-square or Fisher's Exact test for categorical data and the two-sample t-test for continuous data will be performed to examine the group differences. Logistic regression will be performed using the binary response to examine the effect of these factors with covariates selected by Bayesian information criterion (BIC) with age group indicator and genotype included in the regression.
Measured: Once at or near enrollment
Secondary outcomes (2)
Frequency of behavioral interventions
The total number of interventions used and the frequencies of the type interventions used will be calculated. A summary will also be prepared for each age group (6-11 years and 12-18 years).
Measured: Once at or near enrollment
Barriers to the use of interventions to prevent nocturnal enuresis
The number and frequencies of barriers to implementation of interventions will be assessed by a checklist of items (i.e., time, inconvenience, forgetfulness) and open-ended responses provided by participants. A summary will also be prepared for each age group (6-11 years and 12-18 years).
Measured: Once at or near enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2013 (estimated)
- Study startOct 2013
- Primary completionApr 2016
- Study completionApr 2016
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 St. Jude Children's Research HospitalThe 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jerlym Porter, PhD, MPH, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01959958
- Sponsor's protocol ID
- PEESC
- Status last verified
- Apr 2016
- First submitted
- 8 Oct 2013
- Last updated
- 11 May 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01959958
- Acronym / sponsor ID
- PEESC
- Conditions
- Sickle Cell Disease
- Keywords
- Nocturnal enuresis, Barriers to treatment
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 250 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Oct 2013
- Primary completion
- Apr 2016 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 10 Oct 2013 (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.
Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population. This increased risk has been attributed to a decreased ability to concentrate urine caused by sickling-induced nephropathy. The sociodemographic, psychosocial, and medical factors associated with nocturnal enuresis are not well defined. In addition, the impact of these behaviors on emotional and behavioral functioning, along with health-related quality of life are not clear. Despite the availability of evidence-based interventions for nocturnal enuresis, very few families with a child with sickle cell disease have utilized these methods. The reasons for this underutilization of interventions are not clear.
Detailed description
PRIMARY OBJECTIVE: To examine differences between those who do and those who do not exhibit nocturnal enuresis in regard to health-related quality of life, emotional and behavioral functioning, family functioning, stressful life events, sociodemographic factors and medical factors.
SECONDARY OBJECTIVE: To identify barriers to intervention implementation for nocturnal enuresis.
In this study, children and adolescents with sickle cell disease ages 6-18 years and their parents/guardians will each complete an interview with questionnaires assessing nocturnal enuresis, health-related quality of life, emotional and behavioral functioning, family functioning, and stressful life events. Participants who report nocturnal enuresis will identify interventions they have used in the past, along with barriers to intervention implementation.
The future goal will be to use this information to develop an effective intervention strategy for nocturnal enuresis specific to the needs of children and adolescents with sickle cell disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sickle cell disease | – | Other: Questionnaire/InterviewIn this study, children and adolescents with sickle cell disease ages 6-18 years and their parents/guardians will complete a questionnaire/interview. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Pediatric patients with sickle cell disease and their parents/guardians
Key inclusion criteria (9 listed)
- Child Characteristics:
- Caregiver Characteristics:
Key exclusion criteria (4 listed)
- Child Characteristics:
- Caregiver Characteristics:
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 |
|---|---|---|
| Questionnaire/InterviewParticipants will each complete an interview with questionnaires assessing nocturnal enuresis, health-related quality of life, emotional and behavioral functioning, family functioning, and stressful life events. Participants who report nocturnal enuresis will also be asked to identify interventions they have used in the past, along with barriers to intervention implementation. | Other | Sickle cell disease |
Primary outcome
Descriptive group characteristics of sickle cell disease participants with and without nocturnal enuresis
Time frame: Once at or near enrollment
Health-related quality of life, emotional and behavioral functioning, family functioning, stressful life events, sociodemographic factors and medical factors will be examined from questionnaire responses. Classification will be based on the criteria outlined in the Diagnostic and Statistical Manual of Mental Disorders, 4th edition, text revision (DSM-IV-TR). Data will be stratified by age (6-11 years and 12-18 years) and genotype (HbSS/ HbSβ0 thalassemia and HbSC/ HbSβ+ thalassemia). The sample will be dichotomized using the binary response (by nocturnal enuresis/no nocturnal enuresis). Pearson's chi-square or Fisher's Exact test for categorical data and the two-sample t-test for continuous data will be performed to examine the group differences. Logistic regression will be performed using the binary response to examine the effect of these factors with covariates selected by Bayesian information criterion (BIC) with age group indicator and genotype included in the regression.
2 secondary outcomes
Frequency of behavioral interventions
Time frame: Once at or near enrollment
Barriers to the use of interventions to prevent nocturnal enuresis
Time frame: Once at or near enrollment
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 11 May 2016.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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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.
Jerlym Porter, PhD, MPH principal investigator
St. Jude Children's Research Hospital
1 site 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 St. Jude Children's Research Hospital 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 9 inclusion and 4 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 11 May 2016. 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?
Descriptive group characteristics of sickle cell disease participants with and without nocturnal enuresis (time frame: Once at or near enrollment). 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 11 May 2016.