Recessive Dystrophic Epidermolysis Bullosa
Conditions
Keywords
RDEB
Brief summary
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
Detailed description
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.
Interventions
BIOLOGICALFCX-007 (dabocemagene autoficel; see below for FCX-007 description)
FCX-007 is comprised of fibroblasts isolated from the subject's skin biopsies which are genetically corrected with the full length COL7A1 gene encoding for type VII collagen.
Sponsors
Castle Creek Biosciences, LLC.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Intra-patient Randomized, Controlled, Open-label, Multi-center
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Male or female ≥2 years of age at the Screening visit. * Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation. Key
Exclusion criteria
* Medical instability limiting ability to travel to the investigative site. * Active infection with human immunodeficiency virus, hepatitis B or hepatitis C. * The presence of COL7 antibodies. * Evidence of systemic infection. * Evidence or history of squamous cell carcinoma at the site to be injected. * Evidence of or history of metastatic squamous cell carcinoma. * Known allergy to any of the constituents of the product. * Female who is pregnant or breastfeeding. * Receipt of a chemical or biological intervention for the specific treatment of RDEB in the past three (3) months prior to screening or anticipated/planned during the screening and treatment period for this study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete Wound Closure of the First Wound Pair at Week 24 | Week 24 | Complete wound closure of the first wound pair (treated vs. control) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete Wound Closure of the First Wound Pair at Week 12 | Week 12 | Complete wound closure of first wound pair (treated vs. control) |
| Complete Wound Closure of All Wound Pairs at Week 24 | Week 24 | Complete wound closure |
| Complete Wound Closure of All Wound Pairs at Week 12 | Week 12 | Complete wound closure |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts Intra-subject randomized (paired wounds in each subject receive experimental treatment, FCX-007, or remain untreated). Up to three target wound pairs will be identified for each subject. Following pairing, target wounds will be randomly assigned as the treatment wound (FCX-007 is administered) or control wound. 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.
FCX-007 (dabocemagene autoficel; see below for FCX-007 description): FCX-007 is comprised of fibroblasts isolated from the subject's skin biopsies which are genetically corrected with the full length COL7A1 gene encoding for type VII collagen. | 6 |
| Total | 6 |
Baseline characteristics
| Characteristic | FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts |
|---|---|
| Age, Categorical <=18 years | 3 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants |
| Age, Continuous | 28.7 years STANDARD_DEVIATION 16.77 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 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 | 4 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 | 0 / 6 |
| other Total, other adverse events | 2 / 6 |
| serious Total, serious adverse events | 2 / 6 |
Outcome results
Primary
Complete Wound Closure of the First Wound Pair at Week 24
Complete wound closure of the first wound pair (treated vs. control)
Time frame: Week 24
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| FCX-007 Treated Success | Complete Wound Closure of the First Wound Pair at Week 24 | 1 wound |
| Control Success | Complete Wound Closure of the First Wound Pair at Week 24 | 0 wound |
Secondary
Complete Wound Closure of All Wound Pairs at Week 12
Complete wound closure
Time frame: Week 12
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| FCX-007 Treated Success | Complete Wound Closure of All Wound Pairs at Week 12 | 1 wound |
| Control Success | Complete Wound Closure of All Wound Pairs at Week 12 | 3 wound |
Secondary
Complete Wound Closure of All Wound Pairs at Week 24
Complete wound closure
Time frame: Week 24
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| FCX-007 Treated Success | Complete Wound Closure of All Wound Pairs at Week 24 | 1 wound |
| Control Success | Complete Wound Closure of All Wound Pairs at Week 24 | 2 wound |
Secondary
Complete Wound Closure of the First Wound Pair at Week 12
Complete wound closure of first wound pair (treated vs. control)
Time frame: Week 12
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| FCX-007 Treated Success | Complete Wound Closure of the First Wound Pair at Week 12 | 1 wound |
| Control Success | Complete Wound Closure of the First Wound Pair at Week 12 | 1 wound |