Sickle Cell DiseaseNCT02098863
Sickle Cell Clinical Research and Intervention Program
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 10,000 participants
- Where
- 6 sitesUnited States
- Study dates
- 15 Apr 2014to Dec 2044
- How long will I take part?
- 99 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with a diagnosis of sickle cell disease of any genotype.
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, Amendment 6.1 or above).
- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 5 recruiting
- Completed
Our Lady of the Lake Regional Medical Center
Baton Rouge, Louisiana, 70808
- Recruiting
Novant Health Hemby Children's Hospital
Charlotte, North Carolina, 28204
Felipe Bautista Otanez, MD, 704-384-1900
Felipe B. Otanez, MD
- Recruiting
Regional One Health, Diggs-Kraus Sickle Cell Center
Memphis, Tennessee, 38103
Ugochi Ogu, MD, 901-545-8535
Kenneth Ataga, MD
- Recruiting
Methodist Adult Comprehensive Sickle Cell Center
Memphis, Tennessee, 38104
Marquita D. Nelson, MD, 901-516-8182
Kenneth Ataga, MD
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Deepa Manwani, MD, 888-226-4343, referralinfo@stjude.org
Deepa Manwani, MD
- Recruiting
Children's Hospital of Illinois at OSF-Saint Francis Medical Center
Peoria, Illinois, 61637
Kay Saving, MD, 309-624-4945
Kay Saving, MD
About this study
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 (in-person or remote) 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.
Read the 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, the Methodist Adult Comprehensive Sickle Cell Disease Center and the Regional One Health Diggs-Kraus Sickle Cell Center, in Memphis, TN serve as enrolling centers for the SCCRIP protocol. Two 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: Peoria, Illinois and Charlotte, North Carolina. Approximately 300 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.
Participants will be administered a developmental evaluation at approximately 1 year of age. The evaluation will utilize standardized performance-based measures as well as parent rating scales lasting approximately 2 hours. Domains assessed will include cognitive, motor, language, and adaptive development.
A Neuropsychological Screener may be completed with participants within the Young Adult and Adult cohorts, every 6 years. The screening will include a series of verbal and nonverbal problem-solving activities, pencil \& paper tasks, and will include a computerized component.
Quality of Life evaluations (Pediatric Quality of Life Inventory (PedsQL™) will be offered.
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 5.9 months), the infant/pre-school stage (ages 6 months to 5.9 years), the early school stage (ages 6 to 11.9 years), the adolescent stage (ages 12 to 17.9 years), young adulthood (ages 18 to 24.9 years) and mature adulthood (ages 25 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.
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Anemia, Sickle Cell Disease, Survival, Mortality, End-Order Dysfunction
- Medical subject headings
- Anemia, Sickle Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
Relationship between treatment plan and health outcomes in participants with sickle cell disease (SCD)
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.
Measured: 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
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.
Measured: Collected every 6 years from newborn until end-of life, up until December 2044
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2014 (estimated)
- Study start15 Apr 2014
- Primary completionDec 2044 (estimated)
- Study completionDec 2044 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies 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
- 10,000 participants (estimated)
- Target duration
- 99 Years
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- University of Memphis School of Public Health, Le Bonheur Children's Hospital, University of Alabama at Birmingham, Washington University School of Medicine, UTHSC-ORNL Center in Biomedical Informatics, University of Washington, Medical College of Wisconsin, University of Tennessee, Children's Hospital of Philadelphia, National Heart, Lung, and Blood Institute (NHLBI), Vanderbilt University School of Medicine, Baylor College of Medicine
- Responsible party
- Sponsor
- Investigators
- Deepa Manwani, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02098863
- Sponsor's protocol ID
- SCCRIP
- Other IDs
- 1U01HL133996, UTHSC-MRC Sub
- Status last verified
- Jun 2026
- First submitted
- 21 Mar 2014
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Rashkin SR, Kang G, Takemoto CM, Weiss MJ, Ataga KI, Saraf SL, Lebensburger J, Zahr RS. The Longitudinal Effect of APOL1 Risk Alleles on Sickle Cell Anemia-Associated Kidney Function. Am J Hematol. 2026 Jun;101(6):1341-1350. doi: 10.1002/ajh.70290. Epub 2026 Mar 23.
PubMed 41870384Zahr RS, Kang G, Zhang X, Rashkin SR, Kovesdy CP, Takemoto C, Weiss M, Lebensburger J, Ataga KI, Saraf SL. Development of Polygenic Risk Score for Persistent Albuminuria in Children and Adults With Sickle Cell Anemia. Am J Hematol. 2025 Jun;100(6):1019-1028. doi: 10.1002/ajh.27678. Epub 2025 Apr 5.
PubMed 40186439Chang TC, Yu J, Wang Z, Hankins JS, Weiss MJ, Wu G, Westhoff CM, Chou ST, Zheng Y. Machine learning to optimize automated RH genotyping using whole-exome sequencing data. Blood Adv. 2024 Jun 11;8(11):2651-2659. doi: 10.1182/bloodadvances.2023011660.
PubMed 38522094Rai P, Okhomina VI, Kang G, Martinez HR, Hankins JS, Joshi V. Longitudinal effect of disease-modifying therapy on left ventricular diastolic function in children with sickle cell anemia. Am J Hematol. 2023 Jun;98(6):838-847. doi: 10.1002/ajh.26911. Epub 2023 Mar 20.
PubMed 36890729Champlin G, Hwang SN, Heitzer A, Ding J, Jacola L, Estepp JH, Wang W, Ataga KI, Owens CL, Newman J, King AA, Davis R, Kang G, Hankins JS. Progression of central nervous system disease from pediatric to young adulthood in sickle cell anemia. Exp Biol Med (Maywood). 2021 Dec;246(23):2473-2479. doi: 10.1177/15353702211035778. Epub 2021 Aug 18.
PubMed 34407676
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sickle Cell Clinical Research and Intervention Program
- ClinicalTrials.gov ID
- NCT02098863
- Acronym / sponsor ID
- SCCRIP
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Anemia, Sickle Cell Disease, Survival, Mortality, End-Order Dysfunction
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10,000 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- University of Memphis School of Public Health, Le Bonheur Children's Hospital, University of Alabama at Birmingham, Washington University School of Medicine, UTHSC-ORNL Center in Biomedical Informatics, University of Washington, Medical College of Wisconsin, University of Tennessee, Children's Hospital of Philadelphia, National Heart, Lung, and Blood Institute (NHLBI), Vanderbilt University School of Medicine, Baylor College of Medicine
- Study start
- 15 Apr 2014 (actual)
- Primary completion
- Dec 2044 (estimated)
- Study completion
- Dec 2044 (estimated)
- First posted
- 28 Mar 2014 (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.
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 (in-person or remote) 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, the Methodist Adult Comprehensive Sickle Cell Disease Center and the Regional One Health Diggs-Kraus Sickle Cell Center, in Memphis, TN serve as enrolling centers for the SCCRIP protocol. Two 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: Peoria, Illinois and Charlotte, North Carolina. Approximately 300 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.
Participants will be administered a developmental evaluation at approximately 1 year of age. The evaluation will utilize standardized performance-based measures as well as parent rating scales lasting approximately 2 hours. Domains assessed will include cognitive, motor, language, and adaptive development.
A Neuropsychological Screener may be completed with participants within the Young Adult and Adult cohorts, every 6 years. The screening will include a series of verbal and nonverbal problem-solving activities, pencil \& paper tasks, and will include a computerized component.
Quality of Life evaluations (Pediatric Quality of Life Inventory (PedsQL™) will be offered.
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 5.9 months), the infant/pre-school stage (ages 6 months to 5.9 years), the early school stage (ages 6 to 11.9 years), the adolescent stage (ages 12 to 17.9 years), young adulthood (ages 18 to 24.9 years) and mature adulthood (ages 25 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 99 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with a diagnosis of sickle cell disease of any genotype.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Derived from this study: PubMed 41870384Rashkin SR, Kang G, Takemoto CM, Weiss MJ, Ataga KI, Saraf SL, Lebensburger J, Zahr RS. The Longitudinal Effect of APOL1 Risk Alleles on Sickle Cell Anemia-Associated Kidney Function. Am J Hematol. 2026 Jun;101(6):1341-1350. doi: 10.1002/ajh.70290. Epub 2026 Mar 23.
- Derived from this study: PubMed 40186439Zahr RS, Kang G, Zhang X, Rashkin SR, Kovesdy CP, Takemoto C, Weiss M, Lebensburger J, Ataga KI, Saraf SL. Development of Polygenic Risk Score for Persistent Albuminuria in Children and Adults With Sickle Cell Anemia. Am J Hematol. 2025 Jun;100(6):1019-1028. doi: 10.1002/ajh.27678. Epub 2025 Apr 5.
- Derived from this study: PubMed 38522094Chang TC, Yu J, Wang Z, Hankins JS, Weiss MJ, Wu G, Westhoff CM, Chou ST, Zheng Y. Machine learning to optimize automated RH genotyping using whole-exome sequencing data. Blood Adv. 2024 Jun 11;8(11):2651-2659. doi: 10.1182/bloodadvances.2023011660.
- Derived from this study: PubMed 36890729Rai P, Okhomina VI, Kang G, Martinez HR, Hankins JS, Joshi V. Longitudinal effect of disease-modifying therapy on left ventricular diastolic function in children with sickle cell anemia. Am J Hematol. 2023 Jun;98(6):838-847. doi: 10.1002/ajh.26911. Epub 2023 Mar 20.
- Derived from this study: PubMed 34407676Champlin G, Hwang SN, Heitzer A, Ding J, Jacola L, Estepp JH, Wang W, Ataga KI, Owens CL, Newman J, King AA, Davis R, Kang G, Hankins JS. Progression of central nervous system disease from pediatric to young adulthood in sickle cell anemia. Exp Biol Med (Maywood). 2021 Dec;246(23):2473-2479. doi: 10.1177/15353702211035778. Epub 2021 Aug 18.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- SCCRIP: Sickle Cell Research and Intervention ProgramLink provided by the sponsor.
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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.
Deepa Manwani, MD principal investigator
St. Jude Children's Research Hospital
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Deepa Manwani, MD (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02098863, which also lists 5 linked publications.
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 4 Jun 2026.