Sickle Cell Clinical Research and Intervention Program
Sickle Cell Clinical Research and Intervention Program
Sponsors
Lead 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
, 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) 












Source
St. Jude Children's Research Hospital
Oversight Info
Has Dmc
No
Is Fda Regulated Drug
No
Is Fda Regulated Device
No
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.
St. Jude Children's Research Hospital and the Methodist Adult Comprehensive Sickle Cell
Disease Center, in Memphis, TN serve as enrolling centers for the SCCRIP protocol.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), and adulthood (ages 18 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.
- To explore the long-term alterations of prolonged antibiotic exposure on the microbial
community composition among people living with SCD through the collection of swabs as
guided by the Human Microbiome Project (HMP) Manual of Procedures.
Other Pre-Specified Objective:
- Define the drug-exposure to clinical response relationship of HU therapy in children
with SCD.
Overall Status
Recruiting
Start Date
2014-04-15
Completion Date
2044-12-01
Primary Completion Date
2044-12-01
Study Type
Observational [Patient Registry]
Primary Outcome
Measure |
Time Frame |
Relationship between treatment plan and health outcomes in participants with sickle cell disease (SCD) |
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 |
Relationship between genetic properties of biological samples and health outcomes in participants with sickle cell disease |
Collected every 6 years from newborn until end-of life, up until December 2044 |
Enrollment
10000
Condition
Eligibility
Study Pop
Participants with a diagnosis of sickle cell disease of any genotype.
Sampling Method
Non-Probability Sample
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.
Gender
All
Minimum Age
N/A
Maximum Age
N/A
Healthy Volunteers
No
Overall Official
Last Name |
Role |
Affiliation |
Jane Hankins, MD, MS |
Principal Investigator |
St. Jude Children's Research Hospital |
Overall Contact
Location
Facility |
Status |
Contact |
Investigator |
Children's Hospital of Illinois at OSF-Saint Francis Medical Center Peoria Illinois 61637 United States |
Recruiting |
Last Name: Kay Saving, MD Phone: 309-624-4945 |
Last Name: Kay Saving, MD Role: Principal Investigator |
Our Lady of the Lake Regional Medical Center Baton Rouge Louisiana 70808 United States |
Recruiting |
Last Name: Jeff Deyo, MD, PhD Phone: 225-763-6337 |
Last Name: Jeff Deyo, MD, PhD Role: Principal Investigator |
Novant Health Hemby Children's Hospital Charlotte North Carolina 28204 United States |
Recruiting |
Last Name: Paulette Bryant, MD Phone: 704-384-1900 |
Last Name: Paulette Bryant, MD Role: Principal Investigator |
Regional One Health, Diggs-Kraus Sickle Cell Center Memphis Tennessee 38103 United States |
Withdrawn | ||
Methodist Adult Comprehensive Sickle Cell Center Memphis Tennessee 38104 United States |
Recruiting |
Last Name: Kenneth Ataga, MBBS Phone: 901-516-8182 |
Last Name: Kenneth Ataga, MBBS Role: Principal Investigator |
St. Jude Children's Research Hospital Memphis Tennessee 38105 United States |
Recruiting |
Last Name: Jane Hankins, MD, MS Role: Principal Investigator |
Location Countries
Country
United States
Verification Date
2019-08-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
Has Expanded Access
No
Condition Browse
Secondary Id
1U01HL133996
UTHSC-MRC Sub
Firstreceived Results Date
N/A
Biospec Retention
Samples With DNA
Biospec Descr
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. In addition, buccal,
nasal, and rectal swabs and stool samples will be collected from participants and stored in
the Infectious Disease repository 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.
Target Duration
99 Years
Firstreceived Results Disposition Date
N/A
Study Design Info
Observational Model
Cohort
Time Perspective
Prospective
Study First Submitted
March 21, 2014
Study First Submitted Qc
March 25, 2014
Study First Posted
March 28, 2014
Last Update Submitted
August 5, 2019
Last Update Submitted Qc
August 5, 2019
Last Update Posted
August 7, 2019
ClinicalTrials.gov processed this data on December 09, 2019
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.