Long-Term Follow-up Protocol

Recessive Dystrophic Epidermolysis Bullosa Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04917887
• Other study ID #: KRYS-LTFU-01
• Status: Recruiting
• Start date: May 25, 2021
• Est. completion date: May 25, 2028

Study information

• Verified date: June 2021
• Source: Krystal Biotech, Inc.
• Contact: Brittani Agostini
• Phone: 4125865830
• Email: bagostini[@]krystalbio.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: May 25, 2028
• Est. primary completion date: May 25, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months and older

Eligibility

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.

Related Conditions & MeSH terms

• Dominant Dystrophic Epidermolysis Bullosa
• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Recessive Dystrophic Epidermolysis Bullosa

Locations

Country	Name	City	State
United States	Pediatric Skin Research	Coral Gables	Florida
United States	Mission Dermatology Center	Rancho Santa Margarita	California
United States	Stanford University	Redwood City	California

Sponsors (1)

Lead Sponsor	Collaborator
Krystal Biotech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Serious 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	5 years