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STRIVE: Ethics in Rare Disease Research

About this study

Rare diseases are disorders that affect fewer than 200,000 people in the United States. Some of these diseases are ultra-rare and affect only a few thousand people, but collectively, 25 to 30 million Americans are living with rare diseases. Many of these diseases are:

  • Caused by a single gene change
  • Begin in childhood
  • Do not have approved treatments

Personalized treatments are being developed to help treat patients with rare diseases, based on what is causing their disease and their own physical challenges. These treatments may offer hope, but they also raise important questions about safety, fairness, and ethics. Families may face hard choices, especially when treatments are new or untested, and when it is not clear who should have access.

The STRIVE study will ask patients, families, health care providers, and researchers what they think about personalized treatments for ultra-rare diseases, including different brain and blood disorders. We will use surveys, interviews, and focus groups to learn how people feel about:

  • Understanding and agreeing to treatment
  • Who is responsible for decisions
  • Weighing risks and benefits
  • Choosing patients in a fair way

This study will not test any drugs or treatments. Its goal is to understand different ways that patients and families think about the treatment of rare diseases and help guide future research in an ethical way.

Participants will share their thoughts in interviews every 6 months or in a one-time session. Families will also complete surveys about quality of life, trust in health care providers, and coping skills. Doctors, researchers, and other experts will join focus groups to talk about best ways to do personalized treatments.

This study will help us understand what matters most to families and experts. We want to learn what concerns them and what they believe is fair. This information will help create clear and ethical guidance for future studies and treatments. It will help us to better inform patients and families about clinical trials so they can get access to treatments that are safe, ethical, and effective

Eligibility overview

  • Parent or primary caregiver of a child under 21 years old who has an ultra-rare genetic disorder
  • Patients up to age 25 with an ultra-rare genetic disorder and pediatric onset
  • Family members (siblings at least 13 years old, grandparents)
  • Non-family stakeholders involved in clinical care, research, or advocacy for pediatric rare genetic disorders

Request a referral

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full title:

STRIVE: Stakeholders of Rare diseases Informing Values in Ethics

Study goal:

To study the perspectives of patients, families, and professionals on ethical challenges in personalized treatments for ultra-rare diseases and create a best-practice ethical framework

Diagnosis:

Neurological diseases, blood disorders, other ultra-rare diseases

Age:

Parents/caregivers of children under 21 years; patients up to age 25; family members 13 years and older

Clinical trial categories:

Neurological Disorders Blood Disorders

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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