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Long-Term Follow-up Protocol

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04917887
Recruitment Status : Recruiting
First Posted : June 8, 2021
Last Update Posted : June 8, 2021
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Study Description
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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
Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa

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
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Study Type : Observational
Estimated 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
Estimated Primary Completion Date : May 25, 2028
Estimated Study Completion Date : May 25, 2028

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. 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


Eligibility Criteria
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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants who received at least one treatment of gene therapy from a Krystal Biotech, Inc. sponsored study, will be asked to roll-over to this LTFU protocol upon either premature discontinuation from, or completion of a prior parent treatment protocol. However, if a participant enrolls in a different Sponsor gene therapy trial during the LTFU phase this participant will be dropped out from the study.
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04917887


Contacts
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Contact: Brittani Agostini 4125865830 bagostini@krystalbio.com

Locations
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United States, California
Mission Dermatology Center Recruiting
Rancho Santa Margarita, California, United States, 92688
Contact: Guida Pinto    949-858-3376 ext 108    gpinto.missionderm@gmail.com   
Principal Investigator: Shireen Guide, MD         
Stanford University Recruiting
Redwood City, California, United States, 94063
Contact: Sinem Bagci    650-484-6878    isbagci@stanford.edu   
Principal Investigator: Peter Marinkovich, MD         
United States, Florida
Pediatric Skin Research Recruiting
Coral Gables, Florida, United States, 33146
Contact: Edilmary Urdaneta         
Principal Investigator: Mercedes Gonzalez, MD         
Sponsors and Collaborators
Krystal Biotech, Inc.
More Information
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Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT04917887    
Other Study ID Numbers: KRYS-LTFU-01
First Posted: June 8, 2021    Key Record Dates
Last Update Posted: June 8, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases