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

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).

Study Overview

• Status: Recruiting
• Conditions: Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa

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.

• Study Type: Observational
• Enrollment (Anticipated): 50

Contacts and Locations

• Study Contact: Name: Brittani Agostini; Phone Number: 4125865830; Email: bagostini@krystalbio.com

Study Locations

• United States
  • California
    • Rancho Santa Margarita, California, United States, 92688
      • Recruiting
      • Mission Dermatology Center
      • Contact: Guida Pinto; Phone Number: 108 949-858-3376; Email: gpinto.missionderm@gmail.com
      • Principal Investigator: Shireen Guide, MD
    • Redwood City, California, United States, 94063
      • Recruiting
      • Stanford University
      • Contact: Sinem Bagci; Phone Number: 650-484-6878; Email: isbagci@stanford.edu
      • Principal Investigator: Peter Marinkovich, MD
  • Florida
    • Coral Gables, Florida, United States, 33146
      • Recruiting
      • Pediatric Skin Research
      • Contact: Edilmary Urdaneta
      • Principal Investigator: Mercedes Gonzalez, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants who received at least one treatment of gene therapy from a Krystal Biotech, Inc. sponsored study, will be asked to roll-over to this LTFU protocol upon either premature discontinuation from, or completion of a prior parent treatment protocol. However, if a participant enrolls in a different Sponsor gene therapy trial during the LTFU phase this participant will be dropped out from the study.

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Serious Adverse Events (AE)	Incidence of delayed Adverse Events suspected to be related to a Krystal Biotech HSV-1 vector backbone investigational product. A Serious Adverse Event (SAE) is any untoward medical occurrence (whether considered to be related to gene therapy or not) that: Is fatal; Is life-threatening (places the participant at immediate risk of death); Requires inpatient hospitalization or prolongation of existing hospitalization; Is a persistent or significant disability/incapacity, or; Is a congenital abnormality/birth defect	5 years

Collaborators and Investigators

• Sponsor: Krystal Biotech, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 25, 2021
• Primary Completion (Anticipated): May 25, 2028
• Study Completion (Anticipated): May 25, 2028

Study Registration Dates

• First Submitted: June 2, 2021
• First Submitted That Met QC Criteria: June 7, 2021
• First Posted (Actual): June 8, 2021

Study Record Updates

• Last Update Posted (Actual): June 8, 2021
• Last Update Submitted That Met QC Criteria: June 7, 2021
• Last Verified: June 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Collagen Diseases; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica
• Other Study ID Numbers: KRYS-LTFU-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No