Long-Term Follow-up Protocol

Recruiting

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

• Registration Number: NCT04917887
• Lead Sponsor: Krystal Biotech, Inc.

Brief 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.

Study Timeline

• Study Start: 2021-05-25
• Status Verified: 2021-06
• Study First Submitted: 2021-06-02
• Study First Submitted QC: 2021-06-07
• Last Update Submitted: 2021-06-07
• Study First Posted: 2021-06-08
• Last Update Posted: 2021-06-08
• Primary Completion: 2028-05-25
• Study Completion: 2028-05-25

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

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.

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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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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Serious Adverse Events (AE)	5 years	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

Trial Locations

Locations (3)

Mission Dermatology Center; 🇺🇸 Rancho Santa Margarita, California, United States

Pediatric Skin Research; 🇺🇸 Coral Gables, Florida, United States

Stanford University; 🇺🇸 Redwood City, California, United States