MorpheaPhase 1/2NCT03740724
A Study of FCX-013 Plus Veledimex for the Treatment of Moderate to Severe Localized Scleroderma (Morphea)
A Phase 1/2 study testing FCX-013 and veledimex in people with Morphea and Scleroderma, Localized, run by Castle Creek Biosciences, LLC. at 1 site in United States.
Trial summary
A two-component therapeutic consisting of FCX-013 and veledimex for the treatment of localized scleroderma (or morphea). The first component, FCX-013, is autologous human fibroblasts genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Morphea, Scleroderma, Localized, Scleroderma
- What is being tested?
- FCX-013 and veledimex
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2019to 21 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject is an adult, ≥ 18 years of age with moderate to severe localized scleroderma/morphea with sclerotic lesions which have been unresponsive to standard of care therapy.
- Subject has stable control of localized disease (clinically inactive) over the 3 months prior to Screening and through Baseline
- Subject has not participated in previous clinical research study in the 3 months prior to Screening and through Baseline
- Subject has provided informed written consent
- Female subjects of childbearing potential and male subjects engaging in sexual activity that could lead to pregnancy agree to use adequate birth control regimen
- Subject is able to understand the study, cooperate with the study procedures and willing to return to the clinic for the required follow-up visits
Who cannot join
- Subject has a clinically significant skin disorder other than localized scleroderma/morphea in the anatomical area of interest
- Subject has localized scleroderma/morphea only located on the face or over a joint, or lesions that can be successfully managed with topical medications or phototherapy
- Subject has symptoms consistent with systemic scleroderma that have not been stable, or that require treatment that has not been stable for 3 months prior to Screening and through Baseline
- Subject has been treated with UVA1 phototherapy within 2 months prior to Baseline
- Subject requires treatment with a non-stable regimen of systemic immunosuppressive therapy, for any medical condition, or plans to initiate such treatment during the study period
- Subject requires treatment with a non-stable regimen of physical therapy, for localized scleroderma/morphea, or plans to initiate such treatment during the study period.
Show all 17Show fewer
- Subject has any medical instability limiting ability to travel to the investigative center.
- Subject has clinical signs of infection at (or in close proximity to) the target lesion.
- Subject has a history of, or current, malignancy at/near site of injection (except basal cell carcinoma or squamous cell carcinoma that have been treated)
- Subject has a history of, or current, clinically significant liver abnormalities.
- Subject has a history of, or current, clinically significant cardiac abnormalities, or a significant abnormality on ECG
- Subject has clinically significant laboratory abnormalities
- Subject has active infection with human immunodeficiency virus (HIV), or hepatitis B/C
- Subject has an active drug or alcohol addiction
- Subject has any known allergy to any of the constituents of the product
- Subject has received an interventional chemical or biological investigational study product for the specific treatment of localized scleroderma in the 3 months prior to Screening and through Baseline
- Subject is pregnant or nursing or plans to become pregnant or nurse during the study period
Trial locations and contacts
1 site in 1 country.
United States1 site
Paddington Testing Co., Inc.
Philadelphia, Pennsylvania, 19103
About this study
Official title: A Phase 1/2 Study of a Combination of FCX-013 (Genetically-Modified Autologous Human Dermal Fibroblasts) Plus Veledimex for the Treatment of Moderate to Severe Localized Scleroderma (Morphea)
A two-component therapeutic consisting of FCX-013 and veledimex for the treatment of localized scleroderma (or morphea). The first component, FCX-013, is autologous human fibroblasts genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With the FCX-013 therapy, the patient will take an oral compound (Veledimex) to induce MMP-1 protein expression from the injected cells. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further MMP-1 production from the injected cells. FCX-013 plus veledimex is being developed in anticipation of improving skin function in patients by resolving fibrotic lesions and normalizing dermal collagen production
Read the detailed description
Castle Creek is developing a two-component therapeutic product for the treatment of moderate to severe localized scleroderma (morphea). The first component is FCX-013, an autologous genetically modified human dermal fibroblast (GM-HDFs) cell product that is genetically modified with a lentiviral vector (LV) to express human matrix metalloproteinase 1 (MMP1). MMP1 is also known as interstitial collagenase or fibroblast collagenase and is an enzyme that under normal physiological conditions breaks down the extracellular matrix. Specifically, MMP1 breaks down interstitial collagens, types I, II and III. In addition, the FCX-013 cells are also transduced with genetic constructs that encode the RheoSwitch Therapeutic System® (RTS®) an inducible promotor system that in the presence of a small molecule activator ligand controls expression of the MMP1 gene. The RTS® is activated to express MMP1 by the oral administration of the small molecule component. The purpose of this open-label single arm Phase 1/2 study is to investigate the safety and effectiveness of FCX-013 plus veledimex.
- Conditions
- Morphea, Scleroderma, Localized, Scleroderma
- Being tested
- FCX-013, veledimex
- Keywords
- Morphea
- Medical subject headings
- Scleroderma, Localized, Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FCX-013Genetic
Also called Genetically-Modified Autologous Human Dermal Fibroblasts
FCX-013 is a genetically modified cell product obtained from the subject's own skin cells (autologous fibroblasts). The cells are expanded and genetically modified to express metalloproteinase-1 (MMP-1) under the control of a RheoSwitch (RTS®) system. FCX-013 cell suspension is injected intradermally.
veledimexDrug
Also called a small-molecule activator ligand for the RheoSwitch (RTS®) system
Veledimex, is a small molecule which activates the RTS to induce expression of MMP-1 and is and provided as a liquid filled gelatin capsule for oral administration
| Study group | Role | What they receive |
|---|---|---|
| FCX-013 + veledimex | Experimental | Following the injection of FCX-013, subjects will initiate a 14-day course of veledimex to be taken orally daily Receives: Genetic: FCX-013; Drug: veledimex |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the Safety of FCX-013 Plus Veledimex
Safety evaluations include assessment of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs); change in clinical laboratory values; change in vital signs; change in electrocardiograms (ECGs); and incidence of replication-competent lentivirus (RCL) antibodies.
Measured: Study initiation through study completion
Secondary outcomes (1)
Evaluate the Antifibrotic Effects of FCX-013 Plus Veledimex
Evaluate the antifibrotic effects of FCX-013 plus veledimex
Measured: Week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2018
- Study start18 Dec 2019
- Primary completion23 Sep 2020
- Study completion21 Apr 2022
Why it stopped: Sponsor decision
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 MorpheaStudies 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Castle Creek Biosciences, LLC.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Director, Study director, Castle Creek Biosciences, LLC.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03740724
- Sponsor's protocol ID
- FI-SC-001
- Status last verified
- Jan 2024
- First submitted
- 6 Nov 2018
- Last updated
- 23 Jan 2024
- 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 23 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study of a Combination of FCX-013 (Genetically-Modified Autologous Human Dermal Fibroblasts) Plus Veledimex for the Treatment of Moderate to Severe Localized Scleroderma (Morphea)
- ClinicalTrials.gov ID
- NCT03740724
- Acronym / sponsor ID
- FI-SC-001
- Conditions
- Morphea, Scleroderma, Localized, Scleroderma
- Keywords
- Morphea
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Castle Creek Biosciences, LLC.Industry (pharma, biotech, devices)
- Study start
- 18 Dec 2019 (actual)
- Primary completion
- 23 Sep 2020 (actual)
- Study completion
- 21 Apr 2022 (actual)
- First posted
- 14 Nov 2018 (actual)
- Results
- Posted 24 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A two-component therapeutic consisting of FCX-013 and veledimex for the treatment of localized scleroderma (or morphea). The first component, FCX-013, is autologous human fibroblasts genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With the FCX-013 therapy, the patient will take an oral compound (Veledimex) to induce MMP-1 protein expression from the injected cells. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further MMP-1 production from the injected cells. FCX-013 plus veledimex is being developed in anticipation of improving skin function in patients by resolving fibrotic lesions and normalizing dermal collagen production
Detailed description
Castle Creek is developing a two-component therapeutic product for the treatment of moderate to severe localized scleroderma (morphea). The first component is FCX-013, an autologous genetically modified human dermal fibroblast (GM-HDFs) cell product that is genetically modified with a lentiviral vector (LV) to express human matrix metalloproteinase 1 (MMP1). MMP1 is also known as interstitial collagenase or fibroblast collagenase and is an enzyme that under normal physiological conditions breaks down the extracellular matrix. Specifically, MMP1 breaks down interstitial collagens, types I, II and III. In addition, the FCX-013 cells are also transduced with genetic constructs that encode the RheoSwitch Therapeutic System® (RTS®) an inducible promotor system that in the presence of a small molecule activator ligand controls expression of the MMP1 gene. The RTS® is activated to express MMP1 by the oral administration of the small molecule component. The purpose of this open-label single arm Phase 1/2 study is to investigate the safety and effectiveness of FCX-013 plus veledimex.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FCX-013 + veledimex | Experimental | Genetic: FCX-013, Drug: veledimexFollowing the injection of FCX-013, subjects will initiate a 14-day course of veledimex to be taken orally daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Subject is an adult, ≥ 18 years of age with moderate to severe localized scleroderma/morphea with sclerotic lesions which have been unresponsive to standard of care therapy.
- Subject has stable control of localized disease (clinically inactive) over the 3 months prior to Screening and through Baseline
- Subject has not participated in previous clinical research study in the 3 months prior to Screening and through Baseline
- Subject has provided informed written consent
- Female subjects of childbearing potential and male subjects engaging in sexual activity that could lead to pregnancy agree to use adequate birth control regimen
Key exclusion criteria (17 listed)
- Subject has a clinically significant skin disorder other than localized scleroderma/morphea in the anatomical area of interest
- Subject has localized scleroderma/morphea only located on the face or over a joint, or lesions that can be successfully managed with topical medications or phototherapy
- Subject has symptoms consistent with systemic scleroderma that have not been stable, or that require treatment that has not been stable for 3 months prior to Screening and through Baseline
- Subject has been treated with UVA1 phototherapy within 2 months prior to Baseline
- Subject requires treatment with a non-stable regimen of systemic immunosuppressive therapy, for any medical condition, or plans to initiate such treatment during the study period
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-013Also: Genetically-Modified Autologous Human Dermal FibroblastsFCX-013 is a genetically modified cell product obtained from the subject's own skin cells (autologous fibroblasts). The cells are expanded and genetically modified to express metalloproteinase-1 (MMP-1) under the control of a RheoSwitch (RTS®) system. FCX-013 cell suspension is injected intradermally. | Genetic | FCX-013 + veledimex |
| veledimexAlso: a small-molecule activator ligand for the RheoSwitch (RTS®) systemVeledimex, is a small molecule which activates the RTS to induce expression of MMP-1 and is and provided as a liquid filled gelatin capsule for oral administration | Drug | FCX-013 + veledimex |
Primary outcome
Evaluate the Safety of FCX-013 Plus Veledimex
Time frame: Study initiation through study completion
Safety evaluations include assessment of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs); change in clinical laboratory values; change in vital signs; change in electrocardiograms (ECGs); and incidence of replication-competent lentivirus (RCL) antibodies.
1 secondary outcome
Evaluate the Antifibrotic Effects of FCX-013 Plus Veledimex
Time frame: Week 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Morphea
Active, not recruitingObservational study
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Patient Reported Outcomes With UVA-1 Therapy for Treatment of Sclerosing Skin Diseases
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Morphea in Adults and Children (MAC) Cohort Study: A Morphea Registry and DNA Repository
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Enrolling by invitationObservational study
Elastography Ultrasound in Localized Scleroderma (Morphea) Study
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Investigating the Effectiveness of the Biopsychosocial Model-Based Exercise Approach in Children and Adults Diagnosed With Scleroderma
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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.
Clinical Trial Director study director
Castle Creek Biosciences, LLC.
1 site 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-013 and veledimex in people with Morphea and Scleroderma, Localized, run by Castle Creek Biosciences, LLC. at 1 site 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 6 inclusion and 17 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 23 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 24 Jun 2022). 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?
Evaluate the Safety of FCX-013 Plus Veledimex (time frame: Study initiation through study completion). 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 23 Jan 2024.