Brief Title
Long-Term Follow-up Protocol
Official Title
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
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 Type
Observational
Primary Outcome
Serious Adverse Events (AE)
Condition
Dystrophic Epidermolysis Bullosa
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
50
Start Date
May 25, 2021
Completion Date
May 25, 2028
Primary Completion Date
May 25, 2028
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.
Gender
All
Ages
6 Months - N/A
Accepts Healthy Volunteers
No
Contacts
, 4125865830, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04917887
Organization ID
KRYS-LTFU-01
Responsible Party
Sponsor
Study Sponsor
Krystal Biotech, Inc.
Study Sponsor
, ,
Verification Date
June 2021