Recessive Dystrophic Epidermolysis BullosaPhase 3DEFI-RDEBNCT04213261
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
A Phase 3 study testing FCX-007 (dabocemagene autoficel; see below for FCX-007 description) in people with Recessive Dystrophic Epidermolysis Bullosa, run by Castle Creek Biosciences, LLC. at 5 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 5 sitesUnited States
- Study dates
- 9 Jun 2020to Jul 2037
- Phase
- Phase 3
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female ≥2 years of age at the Screening visit.
- Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Dell Children's Medical Group
Austin, Texas, 78723
Solutions Through Advanced Research, Inc.
Jacksonville, Florida, 32256
Mayo Clinic
Rochester, Minnesota, 55905
Stanford University
Stanford, California, 94305
About this study
Official title: A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
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
Read the 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.
- Being tested
- FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
- 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-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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Wound Closure of the First Wound Pair at Week 24
Complete wound closure of the first wound pair (treated vs. control)
Measured: Week 24
Secondary outcomes (3)
Complete Wound Closure of the First Wound Pair at Week 12
Complete wound closure of first wound pair (treated vs. control)
Measured: Week 12
Complete Wound Closure of All Wound Pairs at Week 24
Complete wound closure
Measured: Week 24
Complete Wound Closure of All Wound Pairs at Week 12
Complete wound closure
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2019
- Study start9 Jun 2020
- Primary completion17 Jan 2023
- Study completionJul 2037 (estimated)
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 Recessive Dystrophic Epidermolysis BullosaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Intra-patient Randomized, Controlled, Open-label, Multi-center
- 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
- NCT04213261
- Sponsor's protocol ID
- FI-EB-002
- Other IDs
- R01-7289-01
- Acronym
- DEFI-RDEB
- Status last verified
- Mar 2026
- First submitted
- 2 Dec 2019
- Last updated
- 18 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT04213261
- Acronym / sponsor ID
- DEFI-RDEB / FI-EB-002
- Keywords
- RDEB
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Castle Creek Biosciences, LLC.Industry (pharma, biotech, devices)
- Study start
- 9 Jun 2020 (actual)
- Primary completion
- 17 Jan 2023 (actual)
- Study completion
- Jul 2037 (estimated)
- First posted
- 30 Dec 2019 (actual)
- Results
- Posted 1 Apr 2024
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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Intra-patient Randomized, Controlled, Open-label, Multi-center
| Arm | Type | Receives |
|---|---|---|
| FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts | Experimental | Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)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. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Male or female ≥2 years of age at the Screening visit.
- Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
Key exclusion criteria (9 listed)
- 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.
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-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. | Biological | FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts |
Primary outcome
Complete Wound Closure of the First Wound Pair at Week 24
Time frame: Week 24
Complete wound closure of the first wound pair (treated vs. control)
3 secondary outcomes
Complete Wound Closure of the First Wound Pair at Week 12
Time frame: Week 12
Complete Wound Closure of All Wound Pairs at Week 24
Time frame: Week 24
Complete Wound Closure of All Wound Pairs at Week 12
Time frame: Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2026.
- 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.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing FCX-007 (dabocemagene autoficel; see below for FCX-007 description) in people with Recessive Dystrophic Epidermolysis Bullosa, run by Castle Creek Biosciences, LLC. at 5 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 2 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 18 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 1 Apr 2024). 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?
Complete Wound Closure of the First Wound Pair at Week 24 (time frame: Week 24). 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 18 Mar 2026.