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Sickle Cell Clinical Research and Intervention Program

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ClinicalTrials.gov Identifier: NCT02098863
Recruitment Status : Recruiting
First Posted : March 28, 2014
Last Update Posted : October 25, 2018
Sponsor:
Collaborators:
Methodist Healthcare
University of Memphis School of Public Health
Le Bonheur Children's Hospital
Methodist Adult Comprehensive Sickle Cell Center, Memphis, TN
University of Alabama at Birmingham
Washington University School of Medicine
Regional One Health, Diggs-Kraus Sickle Cell Center, Memphis
UTHSC-ORNL Center in Biomedical Informatics
University of Washington Seattle Cancer Care Alliance
Medical College of Wisconsin
University of Tennessee Health Science Center
Children's Hospital of Philadelphia
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Tracking Information
First Submitted Date March 21, 2014
First Posted Date March 28, 2014
Last Update Posted Date October 25, 2018
Actual Study Start Date April 15, 2014
Estimated Primary Completion Date December 2044   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 19, 2018)
  • Relationship between treatment plan and health outcomes in participants with sickle cell disease (SCD) [ Time Frame: Every 2 years from newborn to ≤ 30 years of age, and every 6 years after age 30 until end-of-life, up until December 2044 ]
    As described in the Detailed Description, standard of care data will be collected from participants every two years during participants' annual clinic visits until study participation is discontinued or until participants reach death/end of life, whichever occurs last. This collection of observational data will be entered into a study database and will serve as a research resource to facilitate evaluation of health outcomes in participants with SCD from pediatric care into adulthood.
  • Relationship between genetic properties of biological samples and health outcomes in participants with sickle cell disease [ Time Frame: Collected every 6 years from newborn until end-of life, up until December 2044 ]
    A repository of biological samples from participants with sickle cell disease will be established for future retrospective studies investigating genetic and epigenetic contributions to disease severity, response to treatment, and morbidity and mortality.
Original Primary Outcome Measures
 (submitted: March 25, 2014)
  • Relationship between treatment plan and health outcomes in participants with sickle cell disease (SCD) [ Time Frame: From newborn to ≤ 25 years of age ]
    As described in the Detailed Description, standard of care data will be collected from participants every two years during participants' annual clinic visits until study participation is discontinued or until participants reach 25 years of age, whichever occurs last. This collection of observational data will be entered into a study database and will serve as a research resource to facilitate evaluation of health outcomes in participants with SCD from pediatric care into adulthood.
  • Relationship between genetic properties of biological samples and health outcomes in participants with sickle cell disease [ Time Frame: Collected at newborn and ages 6, 12, 18 and 24 ]
    A repository of biological samples from participants with sickle cell disease will be established for future retrospective studies investigating genetic and epigenetic contributions to disease severity, response to treatment, and morbidity and mortality.
Change History Complete list of historical versions of study NCT02098863 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Sickle Cell Clinical Research and Intervention Program
Official Title Sickle Cell Clinical Research and Intervention Program
Brief Summary

Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation. An important knowledge deficit is the slow but progressive process of chronic end-organ dysfunction. The majority of organ dysfunction becomes apparent in the young adult years, but comprehensive assessment of adults and understanding of predictors of adulthood organ dysfunction are insufficient. Similarly, the role of disease-modifying therapies, such as hydroxyurea, in preventing organ dysfunction later in life is not clear. Extended follow-up of patients through the transition into adulthood is imperative to understand the long-term implications of pediatric sickle cell care.

This observational study will collect data in a systematic fashion at participants' regular clinic visits to answer the objectives described below.

In addition to primary study objectives, SCCRIP participants will be eligible to participate in a sub-study, which will investigate genetically determined responses to Hydroxyurea (HU) via a pharmacokinetic study (PK). This one time study will involve blood collection at timed intervals proceeding a dose of HU. Defining the basis for this inter-individual variability will allow the identification of poor HU responders prior to initiation of therapy and the seeking of alternative treatments which seek to optimize disease treatment by accounting for individual variability in genes, environment, and lifestyle.

Detailed Description

The St. Jude Pediatric SCD Program has developed a comprehensive plan of care that spans the ages of 0 to 25, and provides the structure for screening and monitoring disease progression and complications in infancy, childhood, and young adulthood. From age 0 to 18, SCD patients are followed at St. Jude Children's Research Hospital. At age 18, their care is typically transferred to either the Methodist Adult Comprehensive Sickle Cell Disease Center in Memphis, TN, or the Regional One Health, Diggs-Kraus Sickle Cell Center in Memphis, TN, where they are routinely followed from age 18 to 25 years. After age 25, participants will be followed and invited to return to St. Jude every 6 years for study related tests until participants elect to come off study or until death. Three St. Jude Affiliate locations will also be sites of enrollment for this protocol for patients age 0 to 18 years. These include St. Jude Affiliate sites located in: Baton Rouge, Louisiana; Peoria, Illinois; and Charlotte, North Carolina. Approximately 500 additional participants are expected to be enrolled from these affiliate sites. This protocol will collect data on SCD participants from birth to end of life.

The SCD plan of care provides the specific sequence of laboratory and imaging studies that are performed according to the patient's age and expected course of illness. The following health outcomes are systematically monitored in patients with SCD: hematologic indices, pulmonary function, cardiac function, renal function, cognitive function, cerebral vasculopathy, vitamin D deficiency and bone health, parvovirus B19 immune status, ophthalmologic status, and splenic function. These tests are used to direct the patient's clinical management and initiate therapies when necessary.

In this study, the results of these tests will be collected and entered into the study database, providing longitudinal data that will inform health outcomes research regarding SCD and how the course is altered by disease-modifying therapy, in addition to facilitating future interventional projects.

Primary Objectives:

  • To establish a longitudinal clinical cohort of patients with sickle cell disease (SCD) to serve as a research resource to facilitate evaluation of health outcomes in SCD from pediatric care into adulthood.
  • To facilitate the collection of biological samples from patients with SCD to be used in future studies investigating genetic and epigenetic contributions to disease severity, response to treatment, and morbidity and mortality.

Secondary Objectives:

  • To determine the incidence, prevalence, and severity of SCD complications and adverse health conditions within the SCD cohort during five stages of development and adulthood: the newborn period (birth to 6 months), the infant/pre-school stage (ages 6 months to 6 years), the early school stage (ages 6 to 12 years), the adolescent stage (ages 12 to 18 years), the years of transition into young adulthood (ages 18 to 25 years), and adulthood (ages 26 years and above).
  • To identify and evaluate risk factors for premature mortality and long-term morbidity in patients with SCD, including those related to disease-modifying therapies, end-organ damage, genetics, neurocognitive deficits, psychosocial factors, and behavioral causes.
  • To investigate the long-term effects of hydroxyurea and other therapies on preservation of organ function, growth and development, and frequency and severity of disease complications, and their long-term medical, neurocognitive, and psychosocial toxicities.
  • To determine the functional aspects of the Transition to Adult Care Program within a clinical research cohort by evaluating disease specific health literacy and readiness in relation to healthcare utilization during adult care.

Other Pre-Specified Objective:

  • Define the drug-exposure to clinical response relationship of HU therapy in children with SCD.
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 99 Years
Biospecimen Retention:   Samples With DNA
Description:
All participants will be offered the option of having biological specimens collected and saved for future research. DNA, plasma, and urine will be collected from participants and stored at Tissue Resources at St. Jude Children's Research Hospital. This will facilitate future high quality genotype-phenotype studies and other genetic and proteomic studies related to variability in disease severity, treatment response, and health outcomes.
Sampling Method Non-Probability Sample
Study Population Participants with a diagnosis of sickle cell disease of any genotype.
Condition Sickle Cell Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: January 4, 2018)
1550
Original Estimated Enrollment
 (submitted: March 25, 2014)
1000
Estimated Study Completion Date December 2044
Estimated Primary Completion Date December 2044   (Final data collection date for primary outcome measure)
Eligibility Criteria

SCCRIP Inclusion Criteria:

  • A diagnosis of sickle cell disease of any genotype.
  • PK Sub-study Inclusion Criteria:

    • Participants at St. Jude Children's Research Hospital who are consented to the parent protocol (SCCRIP).
    • Participants currently completing a hydroxyurea (HU) regimen, who have achieved maximum tolerated dose and have maintained that dose for a minimum of 90 days prior to enrollment.

SCCRIP Exclusion Criteria:

  • Any medical or social reason, which, in the opinion of the principal investigators would make the participation of the subject ill-advised.
  • PK Sub-study Exclusion Criteria:

    • Participants unable to complete the blood draws required for PK sampling.
    • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
    • Any medical or social reason, which, in the opinion of the principal investigators would make the participation of the subject ill-advised.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Jane Hankins, MD, MS 866-278-5833 referralinfo@stjude.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02098863
Other Study ID Numbers SCCRIP
1U01HL133996 ( U.S. NIH Grant/Contract )
UTHSC-MRC Sub ( Other Grant/Funding Number: Tennessee State )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party St. Jude Children's Research Hospital
Study Sponsor St. Jude Children's Research Hospital
Collaborators
  • Methodist Healthcare
  • University of Memphis School of Public Health
  • Le Bonheur Children's Hospital
  • Methodist Adult Comprehensive Sickle Cell Center, Memphis, TN
  • University of Alabama at Birmingham
  • Washington University School of Medicine
  • Regional One Health, Diggs-Kraus Sickle Cell Center, Memphis
  • UTHSC-ORNL Center in Biomedical Informatics
  • University of Washington Seattle Cancer Care Alliance
  • Medical College of Wisconsin
  • University of Tennessee Health Science Center
  • Children's Hospital of Philadelphia
  • National Heart, Lung, and Blood Institute (NHLBI)
Investigators
Principal Investigator: Jane Hankins, MD, MS St. Jude Children's Research Hospital
PRS Account St. Jude Children's Research Hospital
Verification Date October 2018