Date: 2016-09-19
Type of information: update on patient enrollment
phase: 1-2
Announcement: update on patient enrollment
Company: Fibrocell Technologies (USA - PA) Intrexon (USA - MD)
Product: FCX-007
Action
mechanism: gene therapy. Recessive dystrophic epidermolysis bullosa (RDEB) is the most severe form of dystrophic epidermolysis bullosa (DEB). The disease is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen. FCX-007 is a genetically-modified autologous fibroblast that encodes COL7 and is being developed in collaboration with Intrexon. By genetically modifying autologous fibroblasts, ex vivo, to produce COL7, culturing them and then treating blisters and wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas, thereby avoiding systemic treatment.
Disease: recessive dystrophic epidermolysis bullosa
Therapeutic area: Rare diseases - Genetic diseases - Dermatological diseases
Country: USA
Trial
details: The purpose of this study is to evaluate the safety of FCX-007, evaluate C7 expression and the presence of anchoring fibrils resulting from FCX-007 and to analyze wound healing as a result of FCX-007 administration in subjects with RDEB. (NCT02810951)
Latest
news: * On September 19, 2016, Fibrocell Science announced that two additional adult subjects were enrolled in the Phase I/II clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), satisfying enrollment in one of two cohorts in the Phase I portion of the trial. * On July 27, 2016, Fibrocell Science announced that the first two adult subjects were enrolled in the Phase I/II clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). Fibrocell now expects to dose the first subject in this trial at the end of 2016. Additional adult subjects will be dosed after a required 90-day waiting period is complete to ensure there are no safety concerns for the first dose of the new gene-therapy product. Three-month post-treatment follow-up data for safety, mechanism of action and efficacy for the adult subjects in the Phase I portion of this trial are expected in the second half of 2017, as well as six-month data for a cohort of this group. * On April 18, 2016, Fibrocell Science and Intrexon announced that Fibrocell received allowance from the FDA to initiate a Phase I/II clinical trial for FCX-007 in adults. FCX-007 is Fibrocell’s lead orphan gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Six adult subjects are targeted for enrollment in the Phase I portion of the trial. The subjects are divided into two cohorts in order to evaluate the safety of FCX-007 in each population type. One cohort is comprised of those who have positive expression of the non-collagenous portion of the type VII collagen (COL7) protein (NC1+). The second cohort includes those who do not express the non-collagenous portion of the protein (NC1-).
Enrollment of the latest subjects fulfills the NC1+ cohort and also provides the first subject for the NC1- cohort. Two more subjects are required for the NC1- cohort to complete enrollment in the Phase I portion of the trial.
Fibrocell continues to expect to dose the first subject in this trial at the end of 2016. Additional adult subjects will be dosed after a required 90-day waiting period is complete to ensure there are no safety concerns for the first dose of the new gene-therapy product. Three-month post-treatment follow-up data for safety, mechanism of action and efficacy in the Phase I portion of the trial are expected in the second half of 2017, as well as six-month data for multiple subjects.
Fibrocell expects to initiate the open label, Phase I/II clinical trial in the second quarter of 2016. The primary objective of this trial will be to evaluate the safety of FCX-007 in RDEB subjects. Additionally, the trial will evaluate type VII collagen expression and the presence of anchoring fibrils resulting from FCX-007, as well evidence of wound healing. Six adult subjects are expected to be treated with FCX-007 in the Phase I portion of the trial and six pediatric subjects in the Phase II portion of the trial. Prior to conducting studies on pediatric subjects, Fibrocell is required to obtain allowance from the FDA and submit evidence of FCX-007 activity in adult subjects and final data from its ongoing toxicology study.
