NCT04917887

Long-Term Follow-up Protocol

A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the Long-Term Safety of the Krystal Biotech, Inc. Gene Therapy Backbone Products Using HSV-1 Backbone

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

Summary

The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).

Detailed description

Due to the guidelines for gene therapy for vectors which may undergo latency/ reactivation, Krystal Biotech, Inc. has established a Long-Term Follow-up Protocol which will identify and mitigate the long-term risks to the participants receiving their gene therapy products and capture delayed adverse events in participants, annually, for 5-years following the last study visit. Additionally, it will allow the Sponsor to further understand the persistence of the gene therapy products used in their parent treatment protocols.

Conditions

• Dystrophic Epidermolysis Bullosa
• Recessive Dystrophic Epidermolysis Bullosa
• Dominant Dystrophic Epidermolysis Bullosa

Timeline

• Start date: 2021-05-25
• Primary completion: 2028-05-25
• Completion: 2028-05-25
• First posted: 2021-06-08
• Last updated: 2021-06-08

Locations

3 sites across 1 country: United States