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NCT Number: NCT04917887

Long-Term Follow-up Protocol

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).

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Key information

Conditions

Dystrophic Epidermolysis Bullosa Collagen Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Dominant Dystrophic Epidermolysis Bullosa Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Genetic Diseases, Inborn Recessive Dystrophic Epidermolysis Bullosa Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases

Age range

6 month and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Mission Dermatology Center, Rancho Santa Margarita, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  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

Study contacts

Contact information is provided by the study sponsor or research team.

Brittani Agostini

CONTACT

[email protected]

4125865830

Sponsors and collaborators

Lead sponsor

Krystal Biotech, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2021
Primary completion
2028
Study completion
2028
First posted
Jun 8, 2021
Registry last updated
Jun 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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