Sickle Cell Clinical Research and Intervention Program
| Status: | Recruiting |
|---|---|
| Conditions: | Anemia |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 10/27/2018 |
| Start Date: | April 15, 2014 |
| End Date: | December 2044 |
| Contact: | Jane Hankins, MD, MS |
| Email: | referralinfo@stjude.org |
| Phone: | 866-278-5833 |
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.
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.
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.
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.
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.
We found this trial at
6
sites
Baton Rouge, Louisiana
Principal Investigator: Jeff Deyo, MD, PhD
Phone: 225-763-6337
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262 Danny Thomas Pl
Memphis, Tennessee 38105
Memphis, Tennessee 38105
(901) 495-3300
Principal Investigator: Jane Hankins, MD, MS
Phone: 866-278-5833
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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Charlotte, North Carolina 28204
Principal Investigator: Paulette Bryant, MD
Phone: 704-384-1900
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880 Madison Avenue
Memphis, Tennessee 38103
Memphis, Tennessee 38103
Principal Investigator: Patricia Adams-Graves, MD
Phone: 901-545-8535
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1265 Union Avenue
Memphis, Tennessee 38104
Memphis, Tennessee 38104
Principal Investigator: Curtis Owens, MD
Phone: 901-516-8182
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Peoria, Illinois 61637
Principal Investigator: Kay Saving, MD
Phone: 309-624-4945
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