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

NCT03740724 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: FI-SC-001
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Morphea; Scleroderma, Localized; Scleroderma

Keywords

Morphea

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Evaluate the Antifibrotic Effects of FCX-013 Plus Veledimex

  Week 4

Study Arms (1)

FCX-013 + veledimex

EXPERIMENTAL

Following the injection of FCX-013, subjects will initiate a 14-day course of veledimex to be taken orally daily

Genetic: FCX-013; Drug: veledimex

Interventions

FCX-013 GENETIC

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.

Also known as: Genetically-Modified Autologous Human Dermal Fibroblasts

FCX-013 + veledimex

veledimex DRUG

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

Also known as: a small-molecule activator ligand for the RheoSwitch (RTS®) system

FCX-013 + veledimex

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Castle Creek Biosciences, LLC.: lead

Study Sites (1)

Paddington Testing Co., Inc.

Philadelphia, Pennsylvania, 19103, United States

Location

MeSH Terms

Conditions

Scleroderma, Localized; Scleroderma, Diffuse

Interventions

veledimex; Drug Delivery Systems

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Scleroderma, Systemic

Intervention Hierarchy (Ancestors)

Drug Therapy; Therapeutics

Results Point of Contact

• Title: Castle Creek Biosciences Clinical Trial Director
• Organization: Castle Creek Biosciences

clinicalresearch@castlecreekbio.com

484-713-6000

Study Officials

• Clinical Trial Director

  Castle Creek Biosciences, LLC.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2018
• First Posted: November 14, 2018
• Study Start: December 18, 2019
• Primary Completion: September 23, 2020
• Study Completion: April 21, 2022
• Last Updated: January 23, 2024
• Results First Posted: June 24, 2022

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)