Trial Outcomes & Findings for A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) (NCT NCT02810951)

NCT ID: NCT02810951

Last Updated: 2023-02-08

Results Overview

Number of subjects with adverse events.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 6 participants
• Primary outcome timeframe: 52 weeks post treatment
• Results posted on: 2023-02-08

Participant Flow

Participant milestones

Measure	All Study Participants All subjects will receive FCX-007 intradermal injections into a treated wound in a target pair and will have no FCX-007 injections into the other control wound in the target pair.
Overall Study STARTED	6
Overall Study FCX-007 Injection	6
Overall Study No FCX-007 Injection	6
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	All Study Participants All subjects will receive FCX-007 intradermal injections into a treated wound in a target pair and will have no FCX-007 injections into the other control wound in the target pair.
Overall Study Adverse Event	1

Baseline Characteristics

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Baseline characteristics by cohort

Measure	FCX-007 n=6 Participants 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.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	28.4 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants
Open target wounds	10 number of wounds n=5 Participants

PRIMARY outcome

Timeframe: 52 weeks post treatment

Number of subjects with adverse events.

Outcome measures

Measure	Subjects Who Received FCX-007 Injections n=6 Participants All subjects who received FCX-007 injections in at least one target wound.	Untreated Wounds No FCX-007 injected
Adverse Events	6 Participants	—

SECONDARY outcome

Timeframe: Through Week 52

Population: Number analyzed represents observed wounds/participants only

Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits

Outcome measures

Measure	Subjects Who Received FCX-007 Injections n=10 target wounds All subjects who received FCX-007 injections in at least one target wound.	Untreated Wounds n=10 target wounds No FCX-007 injected
Complete Wound Closure Complete closure at Week 4	40.0 percentage of target wounds	0 percentage of target wounds
Complete Wound Closure Complete closure at Week 12	80.0 percentage of target wounds	0 percentage of target wounds
Complete Wound Closure Complete closure at Week 25	75.0 percentage of target wounds	0 percentage of target wounds
Complete Wound Closure Complete closure at Week 52	50.0 percentage of target wounds	16.7 percentage of target wounds

Adverse Events

FCX-007

Serious events: 3 serious events

Other events: 3 other events

Deaths: 1 deaths

Serious adverse events

Measure	FCX-007 n=6 participants at risk 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.
Infections and infestations Gastroenteritis viral	16.7% 1/6 • Number of events 1 • 52 weeks Adverse events were reported on the whole participant level and not segmented to treated or control wounds
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasm progression	16.7% 1/6 • Number of events 1 • 52 weeks Adverse events were reported on the whole participant level and not segmented to treated or control wounds
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous cell carcinoma of skin	16.7% 1/6 • Number of events 1 • 52 weeks Adverse events were reported on the whole participant level and not segmented to treated or control wounds

Other adverse events

Measure	FCX-007 n=6 participants at risk 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.
Skin and subcutaneous tissue disorders Blister	16.7% 1/6 • Number of events 1 • 52 weeks Adverse events were reported on the whole participant level and not segmented to treated or control wounds
General disorders Injection site discolouration	16.7% 1/6 • Number of events 1 • 52 weeks Adverse events were reported on the whole participant level and not segmented to treated or control wounds
General disorders Injection site erythema	16.7% 1/6 • Number of events 1 • 52 weeks Adverse events were reported on the whole participant level and not segmented to treated or control wounds

Additional Information

Chief Medical Officer

Castle Creek Biosciences

Phone: (484) 713-6000

Email: medinfo@castlecreekbio.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: GT60