Scleroderma Clinical Trial
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)
Verified date | January 2024 |
Source | Castle Creek Biosciences, LLC. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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
Status | Terminated |
Enrollment | 1 |
Est. completion date | April 21, 2022 |
Est. primary completion date | September 23, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - 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 Exclusion Criteria: - 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. - 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 |
Country | Name | City | State |
---|---|---|---|
United States | Paddington Testing Co., Inc. | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Castle Creek Biosciences, LLC. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | Study initiation through study completion | |
Secondary | Evaluate the Antifibrotic Effects of FCX-013 Plus Veledimex | Evaluate the antifibrotic effects of FCX-013 plus veledimex | Week 4 |
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