| NCT ID | NCT04917887 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Krystal Biotech, Inc. |
| Condition | Dystrophic Epidermolysis Bullosa |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2021-05-25 |
| Primary Completion | 2028-05-25 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 50 participants in total. It began in 2021-05-25 with a primary completion date of 2028-05-25.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT04917887 clinical trial?
This trial is open to participants of all sexes, aged 6 Months or older, studying Dystrophic Epidermolysis Bullosa. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04917887 currently recruiting?
Yes, NCT04917887 is actively recruiting participants. Contact the research team at bagostini@krystalbio.com for enrollment information.
Where is the NCT04917887 trial being conducted?
This trial is being conducted at Rancho Santa Margarita, United States, Redwood City, United States, Coral Gables, United States.
Who is sponsoring the NCT04917887 clinical trial?
NCT04917887 is sponsored by Krystal Biotech, Inc.. The trial plans to enroll 50 participants.