A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: EB-101.
- Who it may be relevant to
- Registry conditions: RDEB. Basic parameters: from 6 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Overview
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Detailed description
This is an open-label, long-term, follow-up study in participants from prior interventional trials involving surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites. Up to 22 participants will be enrolled in this study, with follow-up through at least 5 years post treatment. Patient-reported outcomes and safety will be assessed throughout the study, and where applicable, compared to Baseline of the preceding interventional EB-101 trial. Upon completion, participants will transfer to an annual monitoring program for additional 10 years of phone visits.
Interventions
- Biological EB-101
This is non-interventional and follows patients treated with EB-101
Primary outcome measures
- Wound Closure [Time frame: 5 years]
- Pain Severity Scale (Wong-Baker FACES) [Time frame: 5 years]
- Itch Severity Scale [Time frame: 5 years]
- Zarit Burden Interview Scale [Time frame: 5 years]
- Quality of Life in Epidermolysis Bullosa [Time frame: 5 years]
- Caregiver Global Impression of Change for Pain Scale [Time frame: 5 years]
- Wound Infection and Adverse Events [Time frame: 5 years]
- The number of treatment-emergent adverse events. [Time frame: 5 years]
- Serious Adverse Events [Time frame: 5 years]
- Incidence of squamous cell carcinoma. [Time frame: 5 years]
Eligibility criteria
Inclusion criteria
- Willing and able to give consent/assent;
- If under the age of 18, guardian(s) is/are willing and able to give consent;
- Prior study treatment with EB-101.
Exclusion criteria
- Inability to properly follow protocol as determined by the Principal Investigator
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 2 centers
- Stanford University — Redwood City
- University of Massachusetts Medical School — Worcester
Identifiers
NCT: NCT05708677 · EB-101-LT-001