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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

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.


Condition or disease
Sickle Cell Disease

  Show Detailed Description

Study Type : Observational [Patient Registry]
Estimated Enrollment : 1550 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 99 Years
Official Title: Sickle Cell Clinical Research and Intervention Program
Actual Study Start Date : April 15, 2014
Estimated Primary Completion Date : December 2044
Estimated Study Completion Date : December 2044

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 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.

  2. 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.


Biospecimen Retention:   Samples With DNA
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.


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants with a diagnosis of sickle cell disease of any genotype.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02098863


Contacts
Contact: Jane Hankins, MD, MS 866-278-5833 referralinfo@stjude.org

Locations
United States, Illinois
Children's Hospital of Illinois at OSF-Saint Francis Medical Center Recruiting
Peoria, Illinois, United States, 61637
Contact: Kay Saving, MD    309-624-4945      
Principal Investigator: Kay Saving, MD         
United States, Louisiana
Our Lady of the Lake Regional Medical Center Recruiting
Baton Rouge, Louisiana, United States, 70808
Contact: Jeff Deyo, MD, PhD    225-763-6337      
Principal Investigator: Jeff Deyo, MD, PhD         
United States, North Carolina
Novant Health Hemby Children's Hospital Recruiting
Charlotte, North Carolina, United States, 28204
Contact: Paulette Bryant, MD    704-384-1900      
Principal Investigator: Paulette Bryant, MD         
United States, Tennessee
Regional One Health, Diggs-Kraus Sickle Cell Center Not yet recruiting
Memphis, Tennessee, United States, 38103
Contact: Patricia Adams-Graves, MD    901-545-8535      
Principal Investigator: Patricia Adams-Graves, MD         
Methodist Adult Comprehensive Sickle Cell Center Recruiting
Memphis, Tennessee, United States, 38104
Contact: Curtis Owens, MD    901-516-8182      
Principal Investigator: Curtis Owens, MD         
St. Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
Contact: Jane Hankins, MD, MS    866-278-5833    referralinfo@stjude.org   
Principal Investigator: Jane Hankins, MD, MS         
Sponsors and Collaborators
St. Jude Children's Research Hospital
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

Additional Information:
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT02098863     History of Changes
Other Study ID Numbers: SCCRIP
1U01HL133996 ( U.S. NIH Grant/Contract )
UTHSC-MRC Sub ( Other Grant/Funding Number: Tennessee State )
First Posted: March 28, 2014    Key Record Dates
Last Update Posted: October 25, 2018
Last Verified: October 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by St. Jude Children's Research Hospital:
Sickle Cell Anemia
Sickle Cell Disease
Survival
Mortality
End-Order Dysfunction

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn