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Recruiting NCT04416178

NCT04416178 Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

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Clinical Trial Summary
NCT ID NCT04416178
Status Recruiting
Phase
Sponsor St. Jude Children's Research Hospital
Condition Sickle Cell Disease
Study Type OBSERVATIONAL
Enrollment 352 participants
Start Date 2020-12-17
Primary Completion 2024-03-26

Eligibility & Interventions

Sex All sexes
Min Age 13 Years
Max Age N/A
Study Type OBSERVATIONAL

Eligibility Fast-Check

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What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 352 participants in total. It began in 2020-12-17 with a primary completion date of 2024-03-26.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers and second, to use surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD. Secondary objectives are development of a web-based tool about treatment options for SCD that fosters patient-clinician communication and promotes shared decision-making. The web-based tool will undergo usability and pilot testing to ensure it is accessible to families and provide data about strategies for integrating into clinical conversations about treatment options. Investigators will use the data generated to reduce the risk of misunderstanding about DNA and genetic research and build strong relationships between SCD families and researchers in the future. The project will design educational information and study materials to help parents of children with SCD understand important details about genomic medicine in SCD care.

Eligibility Criteria

Inclusion Criteria Group 1 (Survey and Interview) Participants only: * Parent of child with HbSS, HbS/ β0thalassemia, or HbSC aged 12 months to 18 years at study initiation, irrespective of clinical severity or patient aged 13-18 with aforementioned SCD genotype. * Informed consent from parent or legal guardian and assent of adolescent participant. * Has been previously approached for SCRIPP. Group 2 (Focus Group) Participants only: * Adult Patient with or Parent of child with HbSS, HbS/ β0thalassemia, β+ thalassemia or HbSC aged 12 months to 18 years at study initiation, irrespective of clinical severity or patient aged 16-18 with aforementioned SCD genotype. * Informed consent from parent or legal guardian and assent of adolescent participant. Group 3 (Usability and Pilot Testing) Participants only: * Parent of child with HbSS, HbS/ β0thalassemia, β+ thalassemia or HbSC aged 12 months to 18 years at study initiation, irrespective of clinical severity or patient aged 13 and up with aforementioned SCD genotype. * Informed consent from parent or legal guardian and assent of adolescent participant. Exclusion Criteria (All groups): * Participants who are unable to converse fluently in English will be excluded. (Permanent) * Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation unsafe or untenable (i.e. cognitive impairment, concurrent acute morbidity). Participant may be re-evaluated. * Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Contact & Investigator

Central Contact

Liza M. Johnson, MD, MPH, MSB

✉ referralinfo@stjude.org

📞 888-226-4343

Principal Investigator

Liza M. Johnson, MD, MPH, MSB

PRINCIPAL INVESTIGATOR

St. Jude Children's Research Hospital

Frequently Asked Questions

Who can join the NCT04416178 clinical trial?

This trial is open to participants of all sexes, aged 13 Years or older, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT04416178 currently recruiting?

Yes, NCT04416178 is actively recruiting participants. Contact the research team at referralinfo@stjude.org for enrollment information.

Where is the NCT04416178 trial being conducted?

This trial is being conducted at Memphis, United States.

Who is sponsoring the NCT04416178 clinical trial?

NCT04416178 is sponsored by St. Jude Children's Research Hospital. The principal investigator is Liza M. Johnson, MD, MPH, MSB at St. Jude Children's Research Hospital. The trial plans to enroll 352 participants.

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ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: April 2026  ·  Data Methodology