Long-term follow-up for gene therapy in skin conditions
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
This study is checking the long-term safety of a gene therapy for people with a skin condition called dystrophic epidermolysis bullosa who have already received the treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | 6 Months and up |
| Sex | All |
| Sponsor | Krystal Biotech, Inc. Industry-sponsored |
| Locations | 3 sites (Rancho Santa Margarita, California and 2 other locations) |
| Trial ID | NCT04917887 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the long-term safety of gene therapy products developed by Krystal Biotech, Inc. for patients with dystrophic epidermolysis bullosa. Participants who have previously received at least one dose of the investigational gene therapy will be monitored annually for five years to identify any delayed adverse events and understand the persistence of the therapy. The study will help ensure participant safety and provide valuable data on the long-term effects of the treatment.
Who should consider this trial
Good fit: Ideal candidates include adults and pediatric patients who have previously received gene therapy from Krystal Biotech, Inc. and have completed or discontinued their parent treatment protocol.
Not a fit: Patients currently enrolled in non-Krystal Biotech, Inc. interventional gene therapy trials will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance the understanding of the long-term safety and efficacy of gene therapies for patients with dystrophic epidermolysis bullosa.
How similar studies have performed: While long-term follow-up studies for gene therapies are common, the specific approach and products being evaluated in this study are novel.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Rancho Santa Margarita, California and 2 other locations
- Mission Dermatology Center — Rancho Santa Margarita, California, United States (Recruiting)
- Stanford University — Redwood City, California, United States (Recruiting)
- Pediatric Skin Research — Coral Gables, Florida, United States (Recruiting)
Study contacts
- Study coordinator: Brittani Agostini
- Email: bagostini@krystalbio.com
- Phone: 4125865830
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.