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Long-Term Follow-up Protocol

Sponsor
Krystal Biotech, Inc. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04917887
Collaborator
(none)
50
Enrollment
3
Locations
84
Anticipated Duration (Months)
16.7
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease Intervention/Treatment Phase

    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 Design

    Study Type:
    Observational
    Anticipated Enrollment :
    50 participants
    Observational Model:
    Other
    Time Perspective:
    Prospective
    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
    Actual Study Start Date :
    May 25, 2021
    Anticipated Primary Completion Date :
    May 25, 2028
    Anticipated Study Completion Date :
    May 25, 2028

    Outcome Measures

    Primary Outcome Measures

    1. 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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Months and Older
    Sexes Eligible for Study:
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Mission Dermatology Center Rancho Santa Margarita California United States 92688
    2 Stanford University Redwood City California United States 94063
    3 Pediatric Skin Research Coral Gables Florida United States 33146

    Sponsors and Collaborators

    • Krystal Biotech, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Krystal Biotech, Inc.
    ClinicalTrials.gov Identifier:
    NCT04917887
    Other Study ID Numbers:
    • KRYS-LTFU-01
    First Posted:
    Jun 8, 2021
    Last Update Posted:
    Jun 8, 2021
    Last Verified:
    Jun 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Product Manufactured in and Exported from the U.S.:
    No
    Additional relevant MeSH terms:
    Epidermolysis Bullosa
    Epidermolysis Bullosa Dystrophica

    Study Results

    No Results Posted as of Jun 8, 2021