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

NCT04917887 · ClinicalTrials.gov registry record

Long-Term Follow-up Protocol

A clinical trial of Dystrophic Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, sponsored by Krystal Biotech.

Recruiting
Registry status
50
Enrollment target
3
Study locations

NCT04917887: Recruiting study of Dystrophic Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, sponsored by Krystal Biotech.

NCT04917887 is a study of Dystrophic Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa that is actively recruiting participants, run by Krystal Biotech. The registered enrollment target is 50 participants, above the 13-participant average among 7 other Dystrophic Epidermolysis Bullosa trials with a reported enrollment target (285% higher). The trial reports 3 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT04917887, a study of Dystrophic Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, is actively recruiting participants, sponsored by Krystal Biotech.

RECRUITING
Registry status
50 participants
Enrollment target
3
Study locations

Study 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).

Primary Outcome

Incidence of delayed Adverse Events suspected to be related to a Krystal Biotech HSV-1 vector backbone investigational product. A Serious Adverse Event (SAE) is any untoward medical occurrence (whether considered to be related to gene therapy or not) that: * Is fatal * Is life-threatening (places the participant at immediate risk of death) * Requires inpatient hospitalization or prolongation of existing hospitalization * Is a persistent or significant disability/incapacity, or * Is a congenita

Study Locations (3)

California

  • Mission Dermatology Center - Rancho Santa Margarita
  • Stanford University - Redwood City

Florida

  • Pediatric Skin Research - Coral Gables

Trial Details

FieldValue
Enrollment Target 50 participants
Start Date 2021-05-25
Est. Completion 2028-05-25
Krystal Biotech

13 total trials

What NCT04917887 shows while recruiting

NCT04917887 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target, above the 13-participant average among 7 other Dystrophic Epidermolysis Bullosa trials with a reported enrollment target (285% higher).

The record links to 3 conditions, with Dystrophic Epidermolysis Bullosa appearing as the primary indexed condition, and to 0 interventions.

NCT04917887 reports a single indexed study location in California, Florida.

Frequently Asked Questions

What is clinical trial NCT04917887 about?

NCT04917887 is a clinical study titled "Long-Term Follow-up Protocol". 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...

What is the current status of trial NCT04917887?

This trial is currently recruiting. The enrollment target is 50 participants. The study started on 2021-05-25. Estimated completion is 2028-05-25.

What conditions does trial NCT04917887 study?

This clinical trial studies the following conditions: Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa.

Who is sponsoring clinical trial NCT04917887?

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

Where is trial NCT04917887 being conducted?

This trial has 3 study locations across California, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for Dystrophic Epidermolysis Bullosa

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

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

  • NCT00200889 · 50 participants · Early Phase 1

    Mapping Auricular Vagus Nerve Circuitry

  • NCT00409435 · 50 participants · Phase 2

    A Study of Pyridostigmine in Postural Tachycardia Syndrome

  • NCT01202305 · 50 participants

    The Use of Lymph Node Biopsies to Support HIV Pathogenesis Studies

  • NCT01225913 · 50 participants · Phase 4

    Mechanism(s) of Airflow Limitation During Exacerbation of Asthma

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 NCT04917887, the US trial registry maintained by the National Library of Medicine. NCT04917887 (small enrollment · single site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.