Long-Term Follow-up Protocol
Purpose
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
Eligibility
- Eligible Ages
- Over 6 Months
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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 Design
- Phase
- Study Type
- Observational
- Observational Model
- Other
- Time Perspective
- Prospective
Recruiting Locations
Rancho Santa Margarita 5386082, California 5332921 92688
Redwood City 5386834, California 5332921 94063
Coral Gables 4151871, Florida 4155751 33146
Edilmary Urdaneta
More Details
- Status
- Recruiting
- Sponsor
- Krystal Biotech, Inc.
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.