Long-Term Follow-up Protocol

Description

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).

Conditions

Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa

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Study Overview

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Study Details

Study overview

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).

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

Long-Term Follow-up Protocol

Condition
Dystrophic Epidermolysis Bullosa
Intervention / Treatment

-

Contacts and Locations

Rancho Santa Margarita

Mission Dermatology Center, Rancho Santa Margarita, California, United States, 92688

Redwood City

Stanford University, Redwood City, California, United States, 94063

Coral Gables

Pediatric Skin Research, Coral Gables, Florida, United States, 33146

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

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

Ages Eligible for Study

6 Months to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Krystal Biotech, Inc.,

Study Record Dates

2028-05-25