Epidermolysis Bullosa Dystrophica, RecessivePhase 1/2NCT02810951
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
A Phase 1/2 study testing FCX-007 in people with Epidermolysis Bullosa Dystrophica, Recessive, run by Castle Creek Biosciences, LLC. at 2 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Epidermolysis Bullosa Dystrophica, Recessive
- What is being tested?
- FCX-007
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Jul 2016to 18 Apr 2022
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age
- Phase I: Eighteen (18) years or older.
- Phase II: Seven (7) years or older.
- Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
Who cannot join
- Medical instability limiting ability to travel to the investigative center.
- Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection
- Current evidence of metastatic squamous cell carcinoma at the site to be injected
- Clinically significant abnormal laboratory result or other significant clinical abnormalities
- Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Stanford University
Stanford, California, 94305
About this study
Official title: A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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
Read the 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.
- Being tested
- FCX-007
- Keywords
- RDEB
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FCX-007Genetic
Also called Genetically-Modified Autologous Human Dermal Fibroblasts
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.
| Study group | Role | What they receive |
|---|---|---|
| FCX-007 | Experimental | 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 years 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 one or more paired target wounds as well as to intact skin 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. Receives: Genetic: FCX-007 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of subjects with adverse events.
Measured: 52 weeks post treatment
Secondary outcomes (1)
Complete Wound Closure
Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits
Measured: Through Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2016 (estimated)
- Study start1 Jul 2016
- Primary completion29 Sep 2020
- Study completion18 Apr 2022
Why it stopped: Subjects were all in Long-Term Follow Up and were transferred to the ongoing EB-002 study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Epidermolysis Bullosa Dystrophica, RecessiveStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Castle Creek Biosciences, LLC.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02810951
- Sponsor's protocol ID
- FI-EB-001
- Other IDs
- FD-R-6113-01
- Status last verified
- Feb 2023
- First submitted
- 13 Jun 2016
- Last updated
- 8 Feb 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- ClinicalTrials.gov ID
- NCT02810951
- Acronym / sponsor ID
- FI-EB-001
- Keywords
- RDEB
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Subjects were all in Long-Term Follow Up and were transferred to the ongoing EB-002 study
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Castle Creek Biosciences, LLC.Industry (pharma, biotech, devices)
- Study start
- 1 Jul 2016 (actual)
- Primary completion
- 29 Sep 2020 (actual)
- Study completion
- 18 Apr 2022 (actual)
- First posted
- 23 Jun 2016 (estimated)
- Results
- Posted 16 Sep 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FCX-007 | Experimental | Genetic: FCX-007In 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 years 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 one or more paired target wounds as well as to intact skin 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. |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Age
- Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
Key exclusion criteria (5 listed)
- Medical instability limiting ability to travel to the investigative center.
- Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection
- Current evidence of metastatic squamous cell carcinoma at the site to be injected
- Clinically significant abnormal laboratory result or other significant clinical abnormalities
- Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FCX-007Also: Genetically-Modified Autologous Human Dermal FibroblastsFCX-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. | Genetic | FCX-007 |
Primary outcome
Adverse Events
Time frame: 52 weeks post treatment
Number of subjects with adverse events.
1 secondary outcome
Complete Wound Closure
Time frame: Through Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FCX-007 in people with Epidermolysis Bullosa Dystrophica, Recessive, run by Castle Creek Biosciences, LLC. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Sep 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: 52 weeks post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Feb 2023.