Stem Cell Transplant During ChildhoodNCT01701492
Cognitive, Academic and Psychosocial Functioning in Long-Term Survivors of Pediatric Stem Cell Transplantation
An observational study of Stem Cell Transplant During Childhood and Allogeneic Bone Marrow Transplant During Childhood, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
This is an observational study to collect information by use of performance-based measures and survey questionnaires. It does not include interventions aimed at altering patient outcome. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Stem Cell Transplant During Childhood, Allogeneic Bone Marrow Transplant During Childhood
- What is being tested?
- Questionnaire and Neurocognitive Evaluation
- How many people
- 133 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Oct 2012to 23 Jan 2018
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Survivors of SCT who are > 5 years since transplant and are returning for a transplant clinic outpatient follow-up visit will be recruited to participate on study. We will recruit patients beginning at age 8 years, with no upper age limit, although we anticipate that most patients will be between 8 and 25 years at the time of assessment. For patients in the age range 8-21 we will obtain data by both self- and parent-report. However, if patients age 18-21 present to clinic without a parent, they will still be eligible for study, and for these patients, as well as those ≤ 21 years, we will obtain performance based and self-report measures only.
Who may be able to join
- Treated with allogeneic bone marrow or stem cell transplant at St. Jude Children's Research Hospital (SJCRH)
- ≤ 21 years at time of transplant
- > 5 years from date of last transplant
- Currently at least 8 years of age
- English as primary language
Who cannot join
- Transplanted for metabolic storage disorder, osteogenesis imperfecta, osteopetrosis, or dyskeratosis congenita.
- History of central nervous system (CNS) injury/disease predating or unrelated to reason for SCT
- Major sensory or motor impairment that would preclude valid cognitive assessment. However, we will not exclude participants with mild cognitive impairments or special educational needs.
- Prior participation in BMTPE protocol at St. Jude Children's Research Hospital.
- Control Group Inclusion Criteria
- Age at least 8 years
Show all 11Show fewer
- No known history of serious illness
- Demographic match to a St. Jude stem cell transplant patient
- Control Group Exclusion Criteria
- Major sensory or motor impairment that would preclude valid cognitive assessment.
- History of specific CNS disease/injury (e.g., Down Syndrome, traumatic brain injury). However, we will not exclude participants with mild cognitive impairments or special educational needs.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
This is an observational study to collect information by use of performance-based measures and survey questionnaires. It does not include interventions aimed at altering patient outcome.
Advances in pediatric hematopoietic stem cell transplantation (SCT) have resulted in improved survival and prompted increased attention to the potential adverse late effects of this procedure. Survivors of SCT are thought to be at risk for neurocognitive deficits as a result of their exposure to a number of potentially neurotoxic agents. Prior studies done by our group and others have demonstrated generally stable cognitive function in the first 5 years following transplant, with little evidence of significant declines. However, there has been almost no research to date on the status of very long-term (> 5 years post-transplant) survivors. In this study, we will evaluate a large sample of long-term survivors of allogeneic SCT using measures of intelligence, academic achievement, and specific cognitive functions such as attention, working memory and processing speed. We will also obtain measures of behavioral functioning and quality of life. We will examine how this group of survivors are functioning relative to normative expectations, and in comparison to community controls without a history of serious illness, matched on age, gender, race/ethnicity, and socioeconomic status. We will also examine the relationship between cognitive function and psychosocial function and quality of life in this population.
Read the detailed description
This study will involve a one-time assessment using both performance based measures as well as both self- and parent-report questionnaires. During their yearly visit to St. Jude, eligible patients who agree to participate will undergo a single neurocognitive evaluation. This evaluation will take place in the Psychology Clinic. The administration time for the assessment battery is estimated at 2.5-3.5 hours, with variability depending on the age of the patient and the speed of completion of the tasks. All measures selected for the study have well-established psychometric properties, including acceptable reliability and validity. All measures were standardized on large, representative samples of children and adults and have age-specific norms.
PRIMARY OBJECTIVE:
To examine and describe psychological outcomes in long-term (> 5 years) survivors of allogeneic bone marrow or stem cell transplantation (SCT) in childhood. This will be conducted in a one-time cross-sectional assessment using both performance based measures and survey questionnaires. Comparisons will be made to established normative data on well validated standardized instruments and to control participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status.
- To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement.
- To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions.
- To examine psychosocial function and quality of life among long-term SCT survivors using both self- and parent-report.
- Being tested
- Questionnaire, Neurocognitive Evaluation
- Keywords
- pediatric, hematopoietic stem cell transplantation, allogeneic bone marrow transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnaireOther
A one-time assessment using both performance-based measures as well as both self- and parent-report questionnaires.
Neurocognitive EvaluationOther
A one-time neurocognitive evaluation conducted in the Psychology Clinic
| Study group | Role | What they receive |
|---|---|---|
| Stem Cell Transplant Survivors | All participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation. Receives: Other: Questionnaire; Other: Neurocognitive Evaluation | |
| Normal control participants | Control participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status. They will complete a questionnaire and undergo neurocognitive evaluation. Receives: Other: Questionnaire; Other: Neurocognitive Evaluation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Intelligence and academic achievement
To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement when compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Measured: 1 day
Performance in specific neuropsychologic domains.
To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Measured: 1 day
Psychosocial function
To examine psychosocial function among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Measured: 1 day
Quality of life
To examine quality of life among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2012 (estimated)
- Study start19 Oct 2012
- Primary completion23 Jan 2018
- Study completion23 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stem Cell Transplant During ChildhoodStudies 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
- 133 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- St. Baldrick's Foundation
- Responsible party
- Sponsor
- Investigators
- Sean Phipps, PhD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01701492
- Sponsor's protocol ID
- XPD12-077 BMTPE3
- Status last verified
- May 2018
- First submitted
- 3 Oct 2012
- Last updated
- 18 May 2018
- 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 18 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cognitive, Academic and Psychosocial Functioning in Long-Term Survivors of Pediatric Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01701492
- Acronym / sponsor ID
- XPD12-077 BMTPE3
- Keywords
- pediatric, hematopoietic stem cell transplantation, allogeneic bone marrow transplantation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 133 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- St. Baldrick's Foundation
- Study start
- 19 Oct 2012 (actual)
- Primary completion
- 23 Jan 2018 (actual)
- Study completion
- 23 Jan 2018 (actual)
- First posted
- 5 Oct 2012 (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.
This is an observational study to collect information by use of performance-based measures and survey questionnaires. It does not include interventions aimed at altering patient outcome.
Advances in pediatric hematopoietic stem cell transplantation (SCT) have resulted in improved survival and prompted increased attention to the potential adverse late effects of this procedure. Survivors of SCT are thought to be at risk for neurocognitive deficits as a result of their exposure to a number of potentially neurotoxic agents. Prior studies done by our group and others have demonstrated generally stable cognitive function in the first 5 years following transplant, with little evidence of significant declines. However, there has been almost no research to date on the status of very long-term (> 5 years post-transplant) survivors. In this study, we will evaluate a large sample of long-term survivors of allogeneic SCT using measures of intelligence, academic achievement, and specific cognitive functions such as attention, working memory and processing speed. We will also obtain measures of behavioral functioning and quality of life. We will examine how this group of survivors are functioning relative to normative expectations, and in comparison to community controls without a history of serious illness, matched on age, gender, race/ethnicity, and socioeconomic status. We will also examine the relationship between cognitive function and psychosocial function and quality of life in this population.
Detailed description
This study will involve a one-time assessment using both performance based measures as well as both self- and parent-report questionnaires. During their yearly visit to St. Jude, eligible patients who agree to participate will undergo a single neurocognitive evaluation. This evaluation will take place in the Psychology Clinic. The administration time for the assessment battery is estimated at 2.5-3.5 hours, with variability depending on the age of the patient and the speed of completion of the tasks. All measures selected for the study have well-established psychometric properties, including acceptable reliability and validity. All measures were standardized on large, representative samples of children and adults and have age-specific norms.
PRIMARY OBJECTIVE:
To examine and describe psychological outcomes in long-term (> 5 years) survivors of allogeneic bone marrow or stem cell transplantation (SCT) in childhood. This will be conducted in a one-time cross-sectional assessment using both performance based measures and survey questionnaires. Comparisons will be made to established normative data on well validated standardized instruments and to control participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status.
- To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement.
- To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions.
- To examine psychosocial function and quality of life among long-term SCT survivors using both self- and parent-report.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stem Cell Transplant Survivors | – | Other: Questionnaire, Other: Neurocognitive EvaluationAll participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation. |
| Normal control participants | – | Other: Questionnaire, Other: Neurocognitive EvaluationControl participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status. They will complete a questionnaire and undergo neurocognitive evaluation. |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Survivors of SCT who are > 5 years since transplant and are returning for a transplant clinic outpatient follow-up visit will be recruited to participate on study. We will recruit patients beginning at age 8 years, with no upper age limit, although we anticipate that most patients will be between 8 and 25 years at the time of assessment. For patients in the age range 8-21 we will obtain data by both self- and parent-report. However, if patients age 18-21 present to clinic without a parent, they will still be eligible for study, and for these patients, as well as those ≤ 21 years, we will obtain performance based and self-report measures only.
Key inclusion criteria (5 listed)
- Treated with allogeneic bone marrow or stem cell transplant at St. Jude Children's Research Hospital (SJCRH)
- ≤ 21 years at time of transplant
- > 5 years from date of last transplant
- Currently at least 8 years of age
- English as primary language
Key exclusion criteria (11 listed)
- Transplanted for metabolic storage disorder, osteogenesis imperfecta, osteopetrosis, or dyskeratosis congenita.
- History of central nervous system (CNS) injury/disease predating or unrelated to reason for SCT
- Major sensory or motor impairment that would preclude valid cognitive assessment. However, we will not exclude participants with mild cognitive impairments or special educational needs.
- Prior participation in BMTPE protocol at St. Jude Children's Research Hospital.
- Control Group Inclusion Criteria
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 |
|---|---|---|
| QuestionnaireA one-time assessment using both performance-based measures as well as both self- and parent-report questionnaires. | Other | Normal control participants, Stem Cell Transplant Survivors |
| Neurocognitive EvaluationA one-time neurocognitive evaluation conducted in the Psychology Clinic | Other | Normal control participants, Stem Cell Transplant Survivors |
Primary outcome
Intelligence and academic achievement
Time frame: 1 day
To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement when compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Performance in specific neuropsychologic domains.
Time frame: 1 day
To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Psychosocial function
Time frame: 1 day
To examine psychosocial function among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.
Quality of life
Time frame: 1 day
To examine quality of life among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.
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 18 May 2018.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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.
Sean Phipps, PhD 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 Stem Cell Transplant During Childhood and Allogeneic Bone Marrow Transplant During Childhood, 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 5 inclusion and 11 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 18 May 2018. 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?
Intelligence and academic achievement (time frame: 1 day). 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 18 May 2018.