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New treatment option for RDEB
Official title GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
ClinicalTrials.gov ID: NCT07193134
What this study is testing
What is Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)?
Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS) is an investigational medicine, given as an once-daily, being studied as a potential treatment for rdeb.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people with RDEB (Recessive Dystrophic Epidermolysis Bullosa). The GMEB-SASS graft contains two types of living skin cells: keratinocytes and fibroblasts.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 and older
You may be able to join if
- Age Learning phase:
- people 1 to 3: Eighteen (18) years old or older.
- people 4 to 6: Twelve (12) years old or older.
- people 7 to 9: Seven (7) years old or older. Other
- Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene.
You likely can't join if
- Medical instability limiting the ability to travel to the investigative center.
- Any medical condition or illness that may impact study participation or compromise the safety of the participants, as per the investigator's judgment.
- Evidence of systemic infection.
- Current evidence or a history of non-metastatic or metastatic squamous cell carcinoma at the site to be grafted.
- Any clinically significant abnormal laboratory values or abnormal findings identified during physical examination or through medical history that...
- History of or known allergy to bovine proteins.
See the full eligibility criteria
- Age Learning phase:
- people 1 to 3: Eighteen (18) years old or older.
- people 4 to 6: Twelve (12) years old or older.
- people 7 to 9: Seven (7) years old or older. Other
- Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene.
- Candidates - or their parents/caregivers if the candidates have limited comprehension, who are able to understand the study and to comply with the study procedures.
- On the day of grafting, one or more blistered and/or erosive skin areas on the trunk and/or extremities large enough to graft at least three 25 to 50 cm2 GMEB-SASS grafts.
- Ability to undergo anesthesia.
- Medical instability limiting the ability to travel to the investigative center.
- Any medical condition or illness that may impact study participation or compromise the safety of the participants, as per the investigator's judgment.
- Evidence of systemic infection.
- Current evidence or a history of non-metastatic or metastatic squamous cell carcinoma at the site to be grafted.
- Any clinically significant abnormal laboratory values or abnormal findings identified during physical examination or through medical history that could compromise participant safety, as per the investigator's judgment.
- History of or known allergy to bovine proteins.
- Active drug or alcohol addiction.
- Female candidate who are pregnant or breast-feeding.
- Candidate who has received immunotherapy, including oral corticosteroids (Prednisolone \> 1 mg/kg), for more than one week, within 2 weeks prior to the study intervention (initial biopsy and GMEB-SASS grafting)...
- Candidate who has received chemotherapy within 60 days prior to the study intervention (initial biopsy and GMEB-SASS grafting).
- Candidate who has received, in the last 6 months prior to the study intervention (initial biopsy and GMEB-SASS grafting), any gene therapy, chemical or biological product modifying collagen 7 expression.
The study team makes the final eligibility decision.
Where it's taking place
- Toronto, Ontario, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 7 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Toronto, Ontario, Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.