Sickle Cell DiseaseNCT03431935
Predictors and Outcomes in Patients With Sickle Cell Disease
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 2 sites in United States.
Trial summary
Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea. Despite greater longevity in SCD, the period following the transition from pediatric to adult care is critical; youth aged 18-30 years are at high risk… Read more about this study
- Who is it for?
- Ages 16 to 20 years
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 94 participants
- Where
- 2 sitesUnited States
- Study dates
- 31 Jul 2018to 26 May 2023
- 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 16 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants who meet eligibility criteria and have a pre-existing relationship as a patient at St. Jude Children's Research Hospital.
Who may be able to join
- Diagnosis of sickle cell disease (all genotypes).
- Age 16.0 - 20.99 years at initial assessment.
- Primary language is English.
Who cannot join
- Participant unable to complete the questionnaires due to refusal, current acute illness (e.g., pain crisis), or significant cognitive impairment as judged by health care providers at the SCD clinic.
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Methodist Adult Comprehensive Sickle Cell Center
Memphis, Tennessee, 38104
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Longitudinal Examination of Predictors and Outcomes of Sickle Cell Disease Healthcare Transition
Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea. Despite greater longevity in SCD, the period following the transition from pediatric to adult care is critical; youth aged 18-30 years are at high risk for mortality and have high rates of healthcare utilization, leading to high healthcare costs. As such, health care transition (HCT) programs have been created to prepare patients for adult-centered care and subsequently, improve health outcomes. However, very few programs have been evaluated for effectiveness in achieving optimal health outcomes in SCD. This paucity of program evaluation is attributed to a lack of identifiable predictors and outcomes.
Researchers at St. Jude Children's Research Hospital want to identify factors and patterns of successful HCT. This information will be used to develop approaches to best evaluate HCT interventions and identify areas of improvement of HCT programming.
Read more
PRIMARY OBJECTIVE: Describe hospital utilization, treatment adherence, and health-related quality of life in a cohort of patients with sickle cell disease (SCD) who will transfer to adult care during the study period.
SECONDARY OBJECTIVE: Examine the associations between various factors and health care transition (HCT) outcomes.
Read the detailed description
Participants will be asked to complete a set of questions during an outpatient clinic visit at St. Jude or Methodist Adult Comprehensive Sickle Cell Center. The questions ask about sickle cell disease knowledge and self-management skills, access to care, general adjustment, quality of life, and stress. These questions will take about 60 minutes to complete.
Participants will answer questionnaires on a password protected laptop or tablet using a computer program called Audio Computer-Assisted Self-Interviews (ACASI). The laptop or tablet will be kept by the study team. A study team member will be available during this time to address any technical issues or answer any questions. Participants will answer one of the questionnaires verbally, and the study team member will record responses from the questionnaire with paper and pen. Questionnaires given verbally will be audio-recorded and reviewed by the lead researcher or other study team member. The recordings will be destroyed immediately after review. No transcript or written record of the recordings will be made. Study members will meet five times with each participant during regularly-scheduled clinical visits over a two year period.
Two institutions will collaborate in the proposed project. St. Jude Children's Research Hospital (St. Jude) and the Methodist Comprehensive Sickle Cell Center will be the primary source of participants. Faculty from the University of Memphis, Department of Psychology, will be involved in methodological considerations and analyzing the data.
- Conditions
- Sickle Cell Disease
- Keywords
- Health Care Transition, Adolescent, Young Adult
- Medical subject headings
- Anemia, Sickle Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of emergency room/hospital admissions in the previous 12 months
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Measured: Once, at age 19
Percent of clinic visits completed in the previous 12 months
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Measured: Once, at age 19
Percent hydroxyurea prescription refill rate
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Measured: Once, at age 19
Health-related quality of life as assessed by the PedsQL
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Measured: Once, at age 19
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2018
- Study start31 Jul 2018
- Primary completion31 Jan 2022
- Study completion26 May 2023
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
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Methodist Comprehensive Sickle Cell Center, University of Memphis, National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Jerlym Porter, PhD, MPH, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03431935
- Sponsor's protocol ID
- LEAPS
- Other IDs
- K01HL125495-01A1
- Status last verified
- Feb 2026
- First submitted
- 7 Feb 2018
- Last updated
- 6 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Examination of Predictors and Outcomes of Sickle Cell Disease Healthcare Transition
- ClinicalTrials.gov ID
- NCT03431935
- Acronym / sponsor ID
- LEAPS
- Conditions
- Sickle Cell Disease
- Keywords
- Health Care Transition, Adolescent, Young Adult
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Methodist Comprehensive Sickle Cell Center, University of Memphis, National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 31 Jul 2018 (actual)
- Primary completion
- 31 Jan 2022 (actual)
- Study completion
- 26 May 2023 (actual)
- First posted
- 13 Feb 2018 (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.
Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea. Despite greater longevity in SCD, the period following the transition from pediatric to adult care is critical; youth aged 18-30 years are at high risk for mortality and have high rates of healthcare utilization, leading to high healthcare costs. As such, health care transition (HCT) programs have been created to prepare patients for adult-centered care and subsequently, improve health outcomes. However, very few programs have been evaluated for effectiveness in achieving optimal health outcomes in SCD. This paucity of program evaluation is attributed to a lack of identifiable predictors and outcomes.
Researchers at St. Jude Children's Research Hospital want to identify factors and patterns of successful HCT. This information will be used to develop approaches to best evaluate HCT interventions and identify areas of improvement of HCT programming.
PRIMARY OBJECTIVE: Describe hospital utilization, treatment adherence, and health-related quality of life in a cohort of patients with sickle cell disease (SCD) who will transfer to adult care during the study period.
SECONDARY OBJECTIVE: Examine the associations between various factors and health care transition (HCT) outcomes.
Detailed description
Participants will be asked to complete a set of questions during an outpatient clinic visit at St. Jude or Methodist Adult Comprehensive Sickle Cell Center. The questions ask about sickle cell disease knowledge and self-management skills, access to care, general adjustment, quality of life, and stress. These questions will take about 60 minutes to complete.
Participants will answer questionnaires on a password protected laptop or tablet using a computer program called Audio Computer-Assisted Self-Interviews (ACASI). The laptop or tablet will be kept by the study team. A study team member will be available during this time to address any technical issues or answer any questions. Participants will answer one of the questionnaires verbally, and the study team member will record responses from the questionnaire with paper and pen. Questionnaires given verbally will be audio-recorded and reviewed by the lead researcher or other study team member. The recordings will be destroyed immediately after review. No transcript or written record of the recordings will be made. Study members will meet five times with each participant during regularly-scheduled clinical visits over a two year period.
Two institutions will collaborate in the proposed project. St. Jude Children's Research Hospital (St. Jude) and the Methodist Comprehensive Sickle Cell Center will be the primary source of participants. Faculty from the University of Memphis, Department of Psychology, will be involved in methodological considerations and analyzing the data.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 16 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants who meet eligibility criteria and have a pre-existing relationship as a patient at St. Jude Children's Research Hospital.
Key inclusion criteria (3 listed)
- Diagnosis of sickle cell disease (all genotypes).
- Age 16.0 - 20.99 years at initial assessment.
- Primary language is English.
Key exclusion criteria (2 listed)
- Participant unable to complete the questionnaires due to refusal, current acute illness (e.g., pain crisis), or significant cognitive impairment as judged by health care providers at the SCD clinic.
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of emergency room/hospital admissions in the previous 12 months
Time frame: Once, at age 19
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Percent of clinic visits completed in the previous 12 months
Time frame: Once, at age 19
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Percent hydroxyurea prescription refill rate
Time frame: Once, at age 19
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
Health-related quality of life as assessed by the PedsQL
Time frame: Once, at age 19
The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
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 Feb 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- Methodist Comprehensive Sickle Cell CenterLink 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
2 sites 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 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 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: finished, as of the sponsor's last update on 6 Feb 2026. 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. 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?
Number of emergency room/hospital admissions in the previous 12 months (time frame: Once, at age 19). 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 Feb 2026.