Long-term follow-up for gene therapy in skin conditions

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• KB803 eye drops versus placebo for people with dystrophic epidermolysis bullosa

  This trial tests whether KB803 eye drops, given three times a week, can reduce recurrent corneal abrasions in children and adults with dystrophic epidermolysis bullosa.

  Recruiting at 5 sites Phase 3 Krystal Biotech, Inc. · Rancho Santa Margarita, California and 4 other locations · NCT07016750

• Using IV immunoglobulin to lower collagen VII antibodies in dystrophic epidermolysis bullosa

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• Energy-F703EB Cream for Epidermolysis Bullosa: a skin cream test in adults

  This phase 2 study tests ENERGI-F703EB Cream versus a matching vehicle to see if it is safe and helps small EB skin lesions in adults.

  Recruiting Phase 2 Energenesis Biomedical Co., Ltd. · Tainan · NCT07858422

• Long-Term Safety Follow-Up After pz-cel for RDEB

  This study follows people with RDEB who were treated with pz-cel to see how safe it is over up to 15 years and whether their wounds stay healed.

  Recruiting at 4 sites Abeona Therapeutics, Inc · Palo Alto, California and 3 other locations · NCT07700966

• Ev.FV to heal wounds in dystrophic epidermolysis bullosa

  This trial will test whether Ev.FV, a mesenchymal stem cell-derived extracellular vesicle therapy, helps heal wounds in people with dystrophic epidermolysis bullosa.

  Recruiting Phase 1/2 Isfahan University of Medical Sciences · Isfahan · NCT07230223

• Gene-modified autologous skin graft (GMEB-SASS) for RDEB

  This trial will try a gene-modified skin graft grown from a patient's own cells to help wounds heal in people with recessive dystrophic epidermolysis bullosa (RDEB).

  Recruiting Phase 1/2 CHU de Quebec-Universite Laval · Toronto, Ontario · NCT07193134

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.