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Clinical Trials in the USA / NCT04917887
Recruiting Observational

Long-Term Follow-up Protocol

NCT04917887 · tracked via the Priya Life Science USA tracker
Sponsor
Krystal Biotech, Inc
Phase
Observational
Started
2021-05-25
Last updated
2021-06-08

Condition(s) studied

Dystrophic Epidermolysis BullosaRecessive Dystrophic Epidermolysis BullosaDominant Dystrophic Epidermolysis Bullosa

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).

Eligibility

Sex
ALL
Min age
6 Months
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Serious Adverse Events (AE) — 5 years
    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 congenital abnormality/birth defect

Trial sites (3)

FacilityCityRegionStatus
Mission Dermatology Center Rancho Santa Margarita California Recruiting
Stanford University Redwood City California Recruiting
Pediatric Skin Research Coral Gables Florida Recruiting

More Krystal Biotech, Inc. trials in the USA

A Study Assessing KB407 for the Treatment of Cystic Fibrosis Recruiting
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency Recruiting
A Study Assessing KB707 for the Treatment of Advanced Solid Tumor Malignancies Affecting the Lungs Recruiting
A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease Recruiting
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB) Recruiting

Other trials for the same condition

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients Active, not recruiting
A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study Recruiting
MSC EVs in Dystrophic Epidermolysis Bullosa Recruiting
Evaluation of D-Fi for the Treatment of Wounds Due to DEB Active, not recruiting
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04917887 on ClinicalTrials.gov ↗ ← All trials in the USA