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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
13 Years and older
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Questionnaires, Interviews

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Parents of children with SCD and adolescent patients will be approached to complete a short survey during a routine clinic visit, in the medicine room or in-patient."
  • "Survey questions will be administered at the time of the informed consent conversation."
  • "Those who agree will be given a paper survey or an ipad to complete survey items which focus on genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD."
  • "Participants also have the option to have questions read to them or they can take the survey on paper."
  • "Completion of the survey is expected to take \< 30 minutes."
  • "Patient and parent can complete surveys simultaneously."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study uses interviews and surveys to learn what families affected by sickle cell disease know and think about DNA research and care. It will also create and test a website about treatment choices to help families talk with their care team. It is for parents of children with sickle cell disease, teens, and some adults with the disease.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Survey and interview group: Parents of children ages 1–18, or teens ages 13–18 with sickle cell disease, may join.
  • For the survey and interview group, the child or teen must have HbSS, HbS/β0 thalassemia, or HbSC. These are types of sickle cell disease.
  • For the survey and interview group, you must have been asked before about joining SCRIPP.
  • Focus group: Adults with the disease, parents of children ages 1–18 with the disease, or teens ages 16–18 with the disease may join.
  • Website testing group: Parents of children ages 1–18 with the disease, or patients ages 13 and older with the disease, may join.
  • For the focus group and website testing group, listed disease types are HbSS, HbS/β0 thalassemia, β+ thalassemia, or HbSC.
  • The disease can be mild or severe. Age limits for children are based on their age when the study starts.
  • A parent or legal guardian must give permission, and teens must agree to take part.

Who can't join

  • You cannot speak English fluently.
  • The study doctor thinks a health condition makes taking part unsafe or not possible. Examples include problems with thinking or a current sudden illness. You may be checked again later.
  • You or your legal guardian cannot or will not give written permission to take part.

Simplified for readability. The official criteria below are what the study team uses.

Official 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.

Study sites (1)

TN