Trials Sponsors Conditions Drugs Pricing

Conditions

Epidermolysis Bullosa Dystrophica, Recessive RDEB Recessive Dystrophic Epidermolysis Bullosa

Keywords

Skin AbnormalitiesCongenital AbnormalitiesEpidermolysis BullosaSkin Diseases, GeneticGenetic Diseases, InbornCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin DiseasesSkin Diseases, VesiculobullousEpidermolysis Bullosa DystrophicaGenetic therapyAutologous Skin GraftSIN Retroviral VectorType VII CollagenCOL7A1

Outcome Measures

Primary Outcomes (3)

Adverse events
Record of Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs).
Up to 12 months
Pain score changes using a Visual Analogue Scale (VAS)
Pain score of the grafted wounds for the past 7 days. Horizontal line, typically 10 centimeters in length, anchored by two verbal descriptors: 0 = No pain; 10 = Worst pain possible.
Baseline, week 2, months 1, 3, 6 and 12
Itch score changes using a Visual Analogue Scale (VAS)
Itch score of the grafted wounds for the past 7 days (100-point visual analog scale). Horizontal line, typically 10 centimeters in length, anchored by two verbal descriptors: 0 = No itch, 100 = Worst itch Time Frame: Baseline, week 2, months 1, 3, 6 and 12

Study Arms (1)

GMEB-SASS

EXPERIMENTAL

All patients will receive GMEB-SASS

Drug: Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)

Interventions

Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)Drug

Wound debridement will be performed, followed by the application of temporary allogeneic skin grafts for 3-5 days. The allografts will then be removed, and the GMEB-SASS grafts will be applied.

GMEB-SASS

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age7 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Age
Learning phase:

Sponsors & Collaborators

CHU de Quebec-Universite Laval
Otherlead

Study Sites (1)

The Hospital for Sick Children (SickKids)

Toronto, Ontario, M5G 1E8, Canada

RECRUITING

Hanna FadzeyevaCONTACT416-813-7835 hanna.fadzeyeva@sickkids.ca

Pope Elena, MD, FRCPCCONTACT416-813-6883 Elena.Pope@sickkids.ca

Elena Pope, MD, FRCPCPRINCIPAL INVESTIGATOR

MeSH Terms

Conditions

Epidermolysis Bullosa DystrophicaSkin AbnormalitiesCongenital AbnormalitiesEpidermolysis BullosaSkin Diseases, GeneticGenetic Diseases, InbornCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin DiseasesSkin Diseases, Vesiculobullous

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

Lucie Germain, PhD
CHU de Québec-Université Laval
STUDY DIRECTOR
Elena Pope, MD, FRCPC
The Hospital for Sick Children
PRINCIPAL INVESTIGATOR

Central Study Contacts

Lucie Germain, PhD

CONTACT

418-525-4444 lucie.germain@fmed.ulaval.ca

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

September 16, 2025

First Posted

September 25, 2025

Study Start

January 7, 2026

Actual (reported)

Primary Completion

December 2030

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (3)

Study Arms (1)

GMEB-SASS

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

Secondary Outcomes (3)

Other Outcomes (9)

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (3)

Study Arms (1)

GMEB-SASS

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

Secondary Outcomes (3)

Other Outcomes (9)

You may not qualify if:

Data Sharing

Source record

Locations