Case Study

Site Selection, Manufacturing & Long-Term Follow-Up: Looming Challenges For A Rescue Phase 1/2 Gene Therapy Trial

By Naomi Kautz

DNA Gene Strand

 

In the following article we introduce a case study as a framework for exploring critical study design considerations of gene therapy trials and offering strategies for addressing those hurdles.

 

This case study is based on Premier Research’s work operationalizing a rescue Phase 1/2 gene therapy trial involving localized administration of gene therapy using specialized equipment. The study comprised two parts, the second of which includes a sham procedure (see Figure 1):

  • Part A: A dose-escalation study consisting of three cohorts and dose levels, with safety reviews in between each dose
  • Part B: Once an optimal dose is found, randomize study participants to either gene therapy or sham procedure with subsequent follow-up
VIEW THE CASE STUDY!
Signing up provides unlimited access to:
Signing up provides unlimited access to:
  • Trend and Leadership Articles
  • Case Studies
  • Extensive Product Database
  • Premium Content
HELLO. PLEASE LOG IN. X

Not yet a member of Clinical Leader? Register today.

ACCOUNT SIGN UP X
Please fill in your account details
Login Information
ACCOUNT SIGN UP

Subscriptions

Sign up for the newsletter that brings you the industry's latest news, technologies, trends and products.

You might also want to: