Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT04917874 · ClinicalTrials.gov registry record · Phase 3

A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

A Phase 3 study, sponsored by Krystal Biotech.

Completed
Registry status
Phase 3
Development phase
47
Enrollment target

NCT04917874: Completed Phase 3 study, sponsored by Krystal Biotech.

NCT04917874 is a Phase 3 study that has completed, run by Krystal Biotech. The registered enrollment target is 47 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (94% lower). According to ClinicalTrials.gov, the official US trial registry.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT04917874, a Phase 3 study, has completed, sponsored by Krystal Biotech.

COMPLETED
Registry status
Phase 3
Development phase
47 participants
Enrollment target

Study Summary

This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.

Primary Outcome

Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.

Interventions

  • BIOLOGICAL Open Label Topical Beremagene Geperpavec (B-VEC)

Trial Details

FieldValue
Enrollment Target 47 participants
Start Date 2021-05-25
Est. Completion 2023-07-31
Phase Phase 3
Krystal Biotech

13 total trials

What the finished NCT04917874 record still lists

NCT04917874 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 47 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (94% lower).

The record links to 0 conditions, and to 1 intervention - of which Open Label Topical Beremagene Geperpavec (B-VEC) is the first listed.

NCT04917874 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT04917874 about?

NCT04917874 is a clinical study titled "A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa". This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoi...

What is the current status of trial NCT04917874?

This trial is currently completed. It is a Phase 3 study. The enrollment target is 47 participants. The study started on 2021-05-25. Estimated completion is 2023-07-31.

What interventions are being tested in trial NCT04917874?

The interventions under investigation include: Open Label Topical Beremagene Geperpavec (B-VEC) (BIOLOGICAL).

Who is sponsoring clinical trial NCT04917874?

This trial is sponsored by Krystal Biotech, which has 13 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT04917874's enrollment target sits among peer trials

47 1867th of 2000 higher than 133 of 2,000 other Phase 3 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

Nationwide trials with similar profiles

Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.

Similar enrollment target

  • NCT03313011 · 47 participants

    The Neurobiology of Two Distinct Types of Progressive Apraxia of Speech

  • NCT03467516 · 47 participants · Phase 2

    Adoptive Transfer of Tumor Infiltrating Lymphocytes for Metastatic Uveal Melanoma

  • NCT03886662 · 47 participants · Phase 1

    A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

  • NCT04013802 · 47 participants · Phase 1

    TETRAVI Multivirus CTL for Treatment of EBV, CMV, Adenovirus, and BK Infections Post Allogeneic SCT.

Similar registry start date

  • NCT05962346 · started 2026-12 · NA

    Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia

  • NCT07125183 · started 2026-12 · Phase 2

    Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma

  • NCT07292298 · started 2026-11 · Phase 2

    Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates

  • NCT04263285 · started 2026-10 · NA

    Treatment of Depression Post-SCI

Source: ClinicalTrials.gov NCT04917874, the US trial registry maintained by the National Library of Medicine. NCT04917874 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.