Recessive Dystrophic Epidermolysis Bullosa Clinical Trial
Official title:
A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa. Funding Source - FDA OOPD
DEFI-RDEB is a multi-center, intra-patient randomized, controlled, open-label, Phase 3 study of FCX-007 for the treatment of persistent non-healing and recurrent RDEB wounds in approximately 24 subjects. Each subject will serve as his/her own control. Each subject's target wounds will be paired then randomized to receive FCX-007 (treatment wound) or remain untreated (control wound). Up to three target wound pairs will be identified for each subject. Subjects will receive intradermal injections of FCX-007 in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1 and the second at Week 12/Month 3. Additional treatment sessions may occur at Week 24/Month 6 and Week 36/Month 9 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated. Safety and efficacy assessments will occur at scheduled intervals through Week 48/Month 12, when the treatment period is completed, and a long-term safety follow-up period (through 15 years) commences for subjects who have received one or more FCX-007 injections. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02493816 -
Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa
|
Phase 1 | |
Completed |
NCT04599881 -
A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
|
Phase 2 | |
Completed |
NCT04520022 -
Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT03632265 -
Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa
|
Phase 3 | |
Recruiting |
NCT05944250 -
A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients
|
N/A | |
Recruiting |
NCT04917887 -
Long-Term Follow-up Protocol
|
||
Completed |
NCT04917874 -
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
|
Phase 3 | |
Not yet recruiting |
NCT04285294 -
Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
|
||
Active, not recruiting |
NCT02323789 -
Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
|
Phase 1/Phase 2 | |
Recruiting |
NCT01874769 -
Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
|
N/A | |
Recruiting |
NCT03392909 -
Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
|
Phase 1/Phase 2 | |
Completed |
NCT03752905 -
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
|
Phase 1/Phase 2 | |
Terminated |
NCT02984085 -
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.
|
Phase 1/Phase 2 | |
Completed |
NCT04227106 -
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
|
Phase 3 | |
Completed |
NCT02698735 -
Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
|
Phase 1/Phase 2 | |
Completed |
NCT03012191 -
Gentamicin for RDEB
|
Phase 1/Phase 2 | |
Completed |
NCT03529877 -
Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
|
Phase 1/Phase 2 | |
Completed |
NCT05143190 -
Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)
|
Phase 2 | |
Recruiting |
NCT04177498 -
Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC
|
Early Phase 1 | |
Completed |
NCT04491604 -
Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
|
Phase 3 |