Recruiting

Observational Study

Sponsor:

Krystal Biotech, Inc.

Code:

NCT04917887

Conditions

Dystrophic Epidermolysis Bullosa

Recessive Dystrophic Epidermolysis Bullosa

Dominant Dystrophic Epidermolysis Bullosa

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

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).

Conditions

Dystrophic Epidermolysis Bullosa

Recessive Dystrophic Epidermolysis Bullosa

Dominant Dystrophic Epidermolysis Bullosa

Study ID

NCT04917887

Start date

May 25, 2021

Status verified date

Jun, 2021

Completion date

May 25, 2028

Anticipated

Primary completion date

May 25, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Serious Adverse Events (AE) [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Brittani Agostini

4125865830bagostini@krystalbio.com

Locations

Mission Dermatology Center

Recruiting

Rancho Santa Margarita, California, United States, 92688

Contacts

Guida Pinto

949-858-3376gpinto.missionderm@gmail.com

Principal Investigator:

Shireen Guide, MD

Stanford University

Recruiting

Redwood City, California, United States, 94063

Contacts

Sinem Bagci

650-484-6878isbagci@stanford.edu

Principal Investigator:

Peter Marinkovich, MD

Pediatric Skin Research

Recruiting

Coral Gables, Florida, United States, 33146

Principal Investigator:

Mercedes Gonzalez, MD

More Information

Sponsor

Krystal Biotech, Inc.

Last update posted

Jun 8, 2021

Last verified

Jun, 2021

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Krystal Biotech, Inc. on 2021-06-08.