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
Overview
- Phase
- N/A
- Intervention
- Not specified
- Conditions
- Dystrophic Epidermolysis Bullosa
- Sponsor
- Krystal Biotech, Inc.
- Enrollment
- 50
- Locations
- 3
- Primary Endpoint
- Serious Adverse Events (AE)
- Status
- Recruiting
- Last Updated
- 4 years ago
Overview
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.
Investigators
Eligibility Criteria
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.
Outcomes
Primary Outcomes
Serious Adverse Events (AE)
Time Frame: 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