Genomic and gene therapy needs for families and patients with sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This prospective mixed-method project uses semi-structured interviews with parents and surveys of parents and adolescents to capture attitudes, concerns, and expectations about genomic sequencing and gene therapy in sickle cell disease. Surveys measure genetic/genomic knowledge, trust in health care providers, and literacy/numeracy, while interviews explore deeper beliefs and decision-making factors. The team will develop a web-based decision-support tool to foster patient-clinician communication and shared decision-making and will conduct usability and pilot testing with families. Participants are recruited at routine clinic or inpatient visits at St. Jude and include parents and adolescents with specified SCD genotypes, many of whom were previously approached for SCCRIP.

Why it matters

Who can join: the full rules

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.

Where it's running

• St. Jude Children's Research Hospital
  Memphis, Tennessee, United States Recruiting

Study contacts

• Principal investigator: Liza M. Johnson, MD, MPH, MSB — St. Jude Children's Research Hospital
• Study coordinator: Liza M. Johnson, MD, MPH, MSB
• Email: referralinfo@stjude.org
• Phone: 888-226-4343

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Virtual reality options to help with VOC pain in adults with SCD

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.