RecruitingNCT04917887

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


Sponsor

Krystal Biotech, Inc.

Enrollment

50 participants

Start Date

May 25, 2021

Study Type

OBSERVATIONAL

Conditions

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).


Eligibility

Min Age: 6 Months

Inclusion Criteria3

  • All adult and pediatric participants who received at least one gene therapy treatment in a previous Krystal Biotech, Inc. sponsored study, and have discontinued or completed the parent treatment protocol (which do not have the LTFU embedded into the parent protocol), as applicable.
  • Consent/Assent must be obtained by the participants (and, parental/legal representative, when applicable) prior to any study-related data being collected.
  • Participant is willing and able to adhere to the protocol requirements.

Exclusion Criteria1

  • Participants who enroll into a non-Krystal Biotech, Inc. interventional gene therapy clinical trial will be excluded from the study.

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Locations(3)

Mission Dermatology Center

Rancho Santa Margarita, California, United States

Stanford University

Redwood City, California, United States

Pediatric Skin Research

Coral Gables, Florida, United States

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NCT04917887


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