Trial Outcomes & Findings for A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (NCT NCT04213261)
NCT ID: NCT04213261
Last Updated: 2024-04-01
Results Overview
Complete wound closure of the first wound pair (treated vs. control)
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE3
Target enrollment
6 participants
Primary outcome timeframe
Week 24
Results posted on
2024-04-01
Participant Flow
Unit of analysis: wound
Participant milestones
| Measure |
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.
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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.
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Overall Study
STARTED
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6 13
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6 13
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Overall Study
COMPLETED
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6 13
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6 13
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Overall Study
NOT COMPLETED
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0 0
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0 0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
Baseline characteristics by cohort
| Measure |
FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
n=6 Participants
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.
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|---|---|
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Age, Categorical
<=18 years
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3 Participants
n=5 Participants
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Age, Categorical
Between 18 and 65 years
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3 Participants
n=5 Participants
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
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Age, Continuous
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28.7 years
STANDARD_DEVIATION 16.77 • n=5 Participants
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Sex: Female, Male
Female
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1 Participants
n=5 Participants
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Sex: Female, Male
Male
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5 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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2 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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4 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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1 Participants
n=5 Participants
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Race (NIH/OMB)
White
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4 Participants
n=5 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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1 Participants
n=5 Participants
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Region of Enrollment
United States
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6 participants
n=5 Participants
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PRIMARY outcome
Timeframe: Week 24Complete wound closure of the first wound pair (treated vs. control)
Outcome measures
| Measure |
FCX-007 Treated Success
n=6 wound
Primary wound treated with FCX-007 with complete wound closure at serial assessments at Week 22 and 24
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Control Success
n=6 Participants
Primary control wound with complete wound closure at serial assessments at Week 22 and 24
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|---|---|---|
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Complete Wound Closure of the First Wound Pair at Week 24
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1 wound
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0 wound
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SECONDARY outcome
Timeframe: Week 12Complete wound closure of first wound pair (treated vs. control)
Outcome measures
| Measure |
FCX-007 Treated Success
n=6 wound
Primary wound treated with FCX-007 with complete wound closure at serial assessments at Week 22 and 24
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Control Success
n=6 wound
Primary control wound with complete wound closure at serial assessments at Week 22 and 24
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|---|---|---|
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Complete Wound Closure of the First Wound Pair at Week 12
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1 wound
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1 wound
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SECONDARY outcome
Timeframe: Week 24Complete wound closure
Outcome measures
| Measure |
FCX-007 Treated Success
n=13 Wounds
Primary wound treated with FCX-007 with complete wound closure at serial assessments at Week 22 and 24
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Control Success
n=13 Wounds
Primary control wound with complete wound closure at serial assessments at Week 22 and 24
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|---|---|---|
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Complete Wound Closure of All Wound Pairs at Week 24
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1 wound
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2 wound
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SECONDARY outcome
Timeframe: Week 12Complete wound closure
Outcome measures
| Measure |
FCX-007 Treated Success
n=13 wound
Primary wound treated with FCX-007 with complete wound closure at serial assessments at Week 22 and 24
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Control Success
n=13 wound
Primary control wound with complete wound closure at serial assessments at Week 22 and 24
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|---|---|---|
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Complete Wound Closure of All Wound Pairs at Week 12
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1 wound
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3 wound
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Adverse Events
FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
n=6 participants at risk
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.
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Injury, poisoning and procedural complications
Lower limb fracture
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16.7%
1/6 • Number of events 1 • 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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Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
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16.7%
1/6 • Number of events 1 • 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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Respiratory, thoracic and mediastinal disorders
Pulmonary infarction
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16.7%
1/6 • Number of events 1 • 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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Other adverse events
| Measure |
FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
n=6 participants at risk
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.
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|---|---|
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Cardiac disorders
Tachycardia
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16.7%
1/6 • Number of events 1 • 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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General disorders
Injection site pain
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16.7%
1/6 • Number of events 2 • 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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Musculoskeletal and connective tissue disorders
Growth retardation
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16.7%
1/6 • Number of events 1 • 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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Musculoskeletal and connective tissue disorders
Osteoporosis
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16.7%
1/6 • Number of events 1 • 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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Skin and subcutaneous tissue disorders
Pruritus
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16.7%
1/6 • Number of events 1 • 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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Vascular disorders
Superficial vein thrombosis
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16.7%
1/6 • Number of events 1 • 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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General disorders
Injection site haemorrhage
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16.7%
1/6 • Number of events 1 • 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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General disorders
Injection site swelling
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16.7%
1/6 • Number of events 1 • 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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Infections and infestations
COVID-19
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16.7%
1/6 • Number of events 1 • 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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Infections and infestations
Skin infection
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16.7%
1/6 • Number of events 1 • 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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Infections and infestations
Wound infection
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16.7%
1/6 • Number of events 1 • 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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Additional Information
Associate VP of Clinical Trials
Castle Creek Biosciences
Phone: 16506901024
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60