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| ID | Type | Description | Link |
|---|---|---|---|
| R01-7289-01 | Other Grant/Funding Number | FDA OOPD |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| FCX-007 (dabocemagene autoficel; see below for FCX-007 description) | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Wound Closure of the First Wound Pair at Week 24 | Complete wound closure of the first wound pair (treated vs. control) | Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Wound Closure of the First Wound Pair at Week 12 | Complete wound closure of first wound pair (treated vs. control) | Week 12 |
| Complete Wound Closure of All Wound Pairs at Week 24 | Complete wound closure |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Stanford | California | 94305 | United States | ||
| Children's Hospital Colorado |
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| ID | Title | Description |
|---|---|---|
| FG000 | FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts | All subjects, 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): 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. |
| FG001 | Control (no Treatment) | All subjects, 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): 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Complete Wound Closure of the First Wound Pair at Week 24 | Complete wound closure of the first wound pair (treated vs. control) | Posted | Number | wound | Week 24 | wound | wound |
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Treatment-emergent adverse events (AEs), regardless of relationship to FCX-007, are those reported from start of treatment to end of treatment period (Week 48)
All treatment-emergent AEs, regardless of relationship to FCX-007, are reported. AEs at a treated wound were to be described and coded as injection site reactions, AEs at other wounds (including but not limited to control wounds) were described without specific reference to injection-site. Only a single interventional group is included, inclusive of all study participants who have received FCX-007 and had at least one control wound.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tachycardia | Cardiac disorders | MedDRA (25.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Associate VP of Clinical Trials | Castle Creek Biosciences | 16506901024 | rblumenthal@castlecreekbio.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 4, 2022 | Feb 27, 2024 | Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 29, 2022 | Feb 27, 2024 | SAP_003.pdf |
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| ID | Term |
|---|---|
| D016108 | Epidermolysis Bullosa Dystrophica |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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Intra-patient Randomized, Controlled, Open-label, Multi-center
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| Week 24 |
| Complete Wound Closure of All Wound Pairs at Week 12 | Complete wound closure | Week 12 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Solutions Through Advanced Research, Inc. | Jacksonville | Florida | 32256 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Dell Children's Medical Group | Austin | Texas | 78723 | United States |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
|
| Counts |
|---|
| Participants |
|
| wound |
|
|
| Secondary | Complete Wound Closure of the First Wound Pair at Week 12 | Complete wound closure of first wound pair (treated vs. control) | Posted | Number | wound | Week 12 | wound | wound |
|
|
|
| Secondary | Complete Wound Closure of All Wound Pairs at Week 24 | Complete wound closure | Posted | Number | wound | Week 24 | Wounds | Wounds |
|
|
|
| Secondary | Complete Wound Closure of All Wound Pairs at Week 12 | Complete wound closure | Posted | Number | wound | Week 12 | wound | wound |
|
|
|
| 0 |
| 6 |
| 2 |
| 6 |
| 2 |
| 6 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
|
| Pulmonary infarction | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA (25.0) | Systematic Assessment |
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| Growth retardation | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
|
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
|
| Superficial vein thrombosis | Vascular disorders | MedDRA (25.0) | Systematic Assessment |
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| Injection site haemorrhage | General disorders | MedDRA (25.0) | Systematic Assessment |
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| Injection site swelling | General disorders | MedDRA (25.0) | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
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| Skin infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
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| Wound infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
|
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| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D012872 | Skin Diseases, Vesiculobullous |