RecruitingNot Applicable

Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

United States145 participantsStarted 2023-06-01

Plain-language summary

This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.

Who can participate

Age range8 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. For Group 1 participants only (Undergone Gene Therapy):
✓. For Group 2 participants only (Offered, but did not Undergo Gene Therapy):
✓. For Group 3 participants only (Provider Interviews):
✓. For Group 4 participants only (Undergone Gene Therapy for Bone Marrow Failure Condition):
✓. For Group 5 participants only (Offered, but did not Undergo Gene Therapy for Bone Marrow Failure Condition ):
✓. For Group 6 participants only (Never offered gene therapy for Bone Marrow Failure Condition):
✓. For Group 7 participants only (Provider Interviews for Bone Marrow Failure Condition):

What they're measuring

Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.

Timeframe: 2 years

Trial details

NCT IDNCT05810181

SponsorSt. Jude Children's Research Hospital

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-12

Contact for this trial

Liza-Marie Johnson, MD, MPH, MSB

888-226-4343 referralinfo@stjude.org

View on ClinicalTrials.gov More Sickle Cell Disease trials