Epidermolysis Bullosa Dystrophica, Recessive
Conditions
Keywords
RDEB
Brief summary
The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects with recessive dystrophic epidermolysis bullosa (RDEB). Funding Source- FDA OOPD
Detailed description
RDEB is a rare skin and connective tissue disease characterized clinically by skin fragility with easy blistering, erosion and scarring of skin and mucous membranes, and caused by the deficiency of the protein type VII collagen (COL7). The objective of this study is evaluate the safety of FCX-007 intradermal injections in RDEB subjects. Additionally, the trial will evaluate COL7 expression, the presence of anchoring fibrils, as well evidence of wound healing. Approximately twelve subjects are expected to enroll in the Phase I/II trial. Phase I will enroll approximately six adult subjects. Phase II will enroll approximately six subjects both adults and pediatric (aged seven (7) years or older). All subjects will receive FCX-007 to one or more paired target RDEB wounds. Proof of mechanism will be monitored through digital photography of target wounds and assays conducted on biopsies taken from intact skin sites where FCX-007 is administered.
Interventions
GENETICFCX-007
FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional COL7. FCX-007 cell suspension is injected intradermally.
Sponsors
Castle Creek Biosciences, LLC.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Age 1. Phase I: Eighteen (18) years or older. 2. Phase II: Seven (7) years or older. 2. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) Key
Exclusion criteria
1. Medical instability limiting ability to travel to the investigative center. 2. Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection 3. Current evidence of metastatic squamous cell carcinoma at the site to be injected 4. Clinically significant abnormal laboratory result or other significant clinical abnormalities 5. Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | 52 weeks post treatment | Number of subjects with adverse events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete Wound Closure | Through Week 52 | Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| FCX-007 In Phase I, a target of three adult subjects will be enrolled into Group A and a target of three adult subjects will be enrolled into Group B.
In Phase II the study will target enrolling subjects (aged seven (7) or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal approximately 6 total subjects.
All subjects will receive FCX-007 into intact skin as well as to one or more paired target wounds at least one time during the study with a possible second administration pending laboratory results.
One wound in each target wound pair will be used as control for efficacy and safety evaluations. FCX-007 administered wounds will be compared within paired target wounds to untreated wounds.
FCX-007: FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional collagen VII. FCX-007 cell suspension is injected intradermally. | 6 |
| Total | 6 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
Baseline characteristics
| Characteristic | FCX-007 |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| Age, Continuous | 28.4 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Open target wounds | 10 number of wounds |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 5 Participants |
| Region of Enrollment United States | 6 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 6 |
| other Total, other adverse events | 3 / 6 |
| serious Total, serious adverse events | 3 / 6 |
Outcome results
Primary
Adverse Events
Number of subjects with adverse events.
Time frame: 52 weeks post treatment
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Subjects Who Received FCX-007 Injections | Adverse Events | 6 Participants |
Secondary
Complete Wound Closure
Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits
Time frame: Through Week 52
Population: Number analyzed represents observed wounds/participants only
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Subjects Who Received FCX-007 Injections | Complete Wound Closure | Complete closure at Week 4 | 40.0 percentage of target wounds |
| Subjects Who Received FCX-007 Injections | Complete Wound Closure | Complete closure at Week 12 | 80.0 percentage of target wounds |
| Subjects Who Received FCX-007 Injections | Complete Wound Closure | Complete closure at Week 25 | 75.0 percentage of target wounds |
| Subjects Who Received FCX-007 Injections | Complete Wound Closure | Complete closure at Week 52 | 50.0 percentage of target wounds |
| Untreated Wounds | Complete Wound Closure | Complete closure at Week 52 | 16.7 percentage of target wounds |
| Untreated Wounds | Complete Wound Closure | Complete closure at Week 4 | 0 percentage of target wounds |
| Untreated Wounds | Complete Wound Closure | Complete closure at Week 25 | 0 percentage of target wounds |
| Untreated Wounds | Complete Wound Closure | Complete closure at Week 12 | 0 percentage of target wounds |