Long-Term Follow-up Protocol


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Trial ID: NCT04917887


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).

Official Title

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

Stanford Investigator(s)

M. Peter Marinkovich, MD
M. Peter Marinkovich, MD

Associate Professor of Dermatology


Inclusion Criteria:

* 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 Criteria:

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


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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Sinem Bagci