Long-Term Follow-up Protocol

Launched by KRYSTAL BIOTECH, INC. · Jun 7, 2021

Keywords

ClinConnect Summary

This clinical trial is focused on understanding the long-term safety of a type of gene therapy for patients with Dystrophic Epidermolysis Bullosa (DEB), a condition that affects the skin and can lead to severe blistering. If you or a family member have previously received at least one dose of gene therapy from Krystal Biotech, Inc. and have completed or stopped the main treatment study, you may be eligible to participate in this follow-up study. This includes both adults and children. Consent will be needed from participants, or from a parent or legal representative for younger participants.

Participants in this study can expect to provide information about their health and any side effects they may experience over time. This research aims to gather important data that could help improve the understanding of gene therapy for DEB. It’s important to note that if someone is currently enrolled in a different gene therapy trial not related to Krystal Biotech, they will not be able to join this study. Your involvement could contribute to important findings that benefit future treatments for this condition.

Gender

ALL

Eligibility 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.