A Study of FCX-013 Plus Veledimex for the Treatment of Moderate to Severe Localized Scleroderma (Morphea)

• Status: Recruiting
• Trial end date: 2036-01-01
• Target enrollment: 0
• Participant gender: All

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 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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Castle Creek Biosciences, LLC.; Fibrocell Technologies, Inc.

Criteria

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