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).
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
50
Mission Dermatology Center
Rancho Santa Margarita, California, United States
RECRUITINGStanford University
Redwood City, California, United States
RECRUITINGPediatric Skin Research
Coral Gables, Florida, United States
RECRUITINGSerious 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
Time frame: 5 years
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