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NCT04917887

Long-Term Follow-up Protocol

Recruiting now Last updated 8 June 2021
What this trial tests

trial in Dystrophic Epidermolysis Bullosa in 50 participants. Currently enrolling.

Timeline
25 May 2021
Primary endpoint
25 May 2028
25 May 2028

Quick facts

Lead sponsorKrystal Biotech, Inc.
StatusRecruiting now
Study typeOBSERVATIONAL
Enrollment50
Start date25 May 2021
Primary completion25 May 2028
Estimated completion25 May 2028
Sites3 locations across United States

Conditions studied

Sponsor

Krystal Biotech, Inc. — full company profile →

Who can join

6 Months and older, any sex, with Dystrophic Epidermolysis Bullosa or Recessive Dystrophic Epidermolysis Bullosa. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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).

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.
    Bischof J, Hierl M, Koller U. · · 2024 · cited 20× · PMID 38396920 · DOI 10.3390/ijms25042243
  2. CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa.
    Wang X, Wang X, Li Y, A S, et al · · 2023 · cited 9× · PMID 38027067 · DOI 10.1016/j.omtm.2023.101134
  3. Treatment of Epidermolysis Bullosa and Future Directions: A Review.
    Danescu S, Negrutiu M, Has C. · · 2024 · cited 7× · PMID 39090514 · DOI 10.1007/s13555-024-01227-8

Verify or expand the search:

Other recruiting trials for Dystrophic Epidermolysis Bullosa

Currently open trials in the same condition.

Other Krystal Biotech, Inc. trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04917887.

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