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

Primary Purpose

Morphea, Scleroderma, Localized, Scleroderma

Status

Terminated

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

FCX-013

veledimex

About this trial

This is an interventional treatment trial for Morphea focused on measuring Morphea

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Paddington Testing Co., Inc.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

FCX-013 + veledimex

Arm Description

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Evaluate the Antifibrotic Effects of FCX-013 Plus Veledimex

Evaluate the antifibrotic effects of FCX-013 plus veledimex

Full Information

NCT ID

NCT03740724

First Posted

November 6, 2018

Last Updated

June 16, 2022

Sponsor

Castle Creek Biosciences, LLC.

1. Study Identification

Unique Protocol Identification Number

NCT03740724

Brief Title

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

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)

Study Type

Interventional

2. Study Status

Record Verification Date

June 2022

Overall Recruitment Status

Terminated

Why Stopped

Sponsor decision

Study Start Date

December 18, 2019 (Actual)

Primary Completion Date

September 23, 2020 (Actual)

Study Completion Date

April 21, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Castle Creek Biosciences, LLC.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Morphea, Scleroderma, Localized, Scleroderma

Keywords

Morphea

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

1 (Actual)

8. Arms, Groups, and Interventions

Arm Title

FCX-013 + veledimex

Arm Type

Experimental

Arm Description

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

Intervention Type

Genetic

Intervention Name(s)

FCX-013

Other Intervention Name(s)

Genetically-Modified Autologous Human Dermal Fibroblasts

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

veledimex

Other Intervention Name(s)

a small-molecule activator ligand for the RheoSwitch (RTS®) system

Intervention Description

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

Primary Outcome Measure Information:

Title

Evaluate the Safety of FCX-013 Plus Veledimex

Description

Time Frame

Study initiation through study completion

Secondary Outcome Measure Information:

Title

Evaluate the Antifibrotic Effects of FCX-013 Plus Veledimex

Description

Evaluate the antifibrotic effects of FCX-013 plus veledimex

Time Frame

Week 4

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Mary C Spellman, MD

Organizational Affiliation

Castle Creek Biosciences, LLC.

Official's Role

Study Director

Facility Information:

Facility Name

Paddington Testing Co., Inc.

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19103

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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