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Understanding Sickle Cell Disease and Gene Therapy Needs

This study aims to understand what parents of children with sickle cell disease (SCD) and adolescents with SCD think about research involving genomic sequencing (looking at a person's complete set of DNA). Researchers want to learn about their concerns, expectations, and how much they trust healthcare providers and researchers. They will also measure participants' knowledge about genetics. This information is important because genomic sequencing is becoming more common in SCD research, especially for new treatments like gene therapy. The study will involve parents of children with SCD (ages 12 months to 18 years) and adolescents with SCD (ages 13-18). The goal is to help develop better ways to talk about treatment options and make shared decisions between patients and doctors.

Study design
This is an observational study involving 352 participants. It uses both interviews and surveys to gather information.
What's involved
You would complete a short survey during a routine clinic visit. Some parents may also participate in semi-structured interviews.
Compensation
Not stated in the trial record.
Follow-up
Surveys are measured at Day 1. Interviews may occur at Day 1 or a future visit, up to approximately 1 year.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04416178

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

Recruiting
Not specifiedAges 13+Observational
St. Jude Children's Research Hospital
~352 participants
Updated 2026-05-01 on ClinicalTrials.gov

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Use of semi-structured interviews in parents of SCD patients to qualitatively describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers
Measured over Day 1, or at a future visit (up to approximately 1 year)
+1 more outcome measured

Conditions

Sickle Cell Disease

Explore related trials

Beta ThalassemiaHematologic NeoplasmHemoglobin E beta zero thalassemiaSickle Cell-Hemoglobin C DiseaseSickle Cell-SS Disease

Where it's being run

1 sites across 1 states
Tennessee1

Study leadership

  • Liza M. Johnson, MD, MPH, MSB · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

Who to contact

Liza M. Johnson, MD, MPH, MSB
[email protected]
888-226-4343
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.
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.
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

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.

What this trial measures

  • Use of semi-structured interviews in parents of SCD patients to qualitatively describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchersDay 1, or at a future visit (up to approximately 1 year)

    Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes

  • Use of surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider/researchers, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD.Day 1

    Patients and parents' demographic characteristics will be collected from the electronic medical record (EMR). Participants will complete various survey instruments designed to measure knowledge and attitudes around genetic testing and biobanks, self-reported literacy and numeracy, and trust in providers. Data will be analyzed quantitatively using descriptive statistics, generalized linear regression models and generalized estimation equations.