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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03740724
Recruitment Status : Recruiting
First Posted : November 14, 2018
Last Update Posted : December 24, 2019
Information provided by (Responsible Party):
Fibrocell Technologies, Inc.

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

Condition or disease Intervention/treatment Phase
Morphea Scleroderma, Localized Scleroderma Genetic: FCX-013 Drug: veledimex Phase 1 Phase 2

Detailed Description:
Fibrocell 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : December 18, 2019
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : March 2021

Arm Intervention/treatment
Experimental: FCX-013 + veledimex
At Day 0 FCX-013 will be administered intradermally to sclerotic lesions with the possibility of an additional administration pending Week 12 results. Veledimex will be initiated on the day of injection an continue for 2 weeks after the injection of FCX-013.
Genetic: FCX-013
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.
Other Name: Genetically-Modified Autologous Human Dermal Fibroblasts

Drug: veledimex
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
Other Name: a small-molecule activator ligand for the RheoSwitch (RTS®) system

Primary Outcome Measures :
  1. Safety of intradermal FCX-013 and oral veledimex based on evaluation of adverse events. [ Time Frame: Study initiation through study completion: up to 20 months ]
    Evaluation of adverse events as assessed by CTCAE. Adverse events will be summarized based on the incidence, intensity and type of adverse event.

Secondary Outcome Measures :
  1. Skin thickness and fibrosis by histology compared to Day 0 [ Time Frame: Week 12 and Week 25 ]
    Evaluation of dermal and subdermal thickness and fibrosis of sentinel lesion(s) by Histology compared to Day 0

  2. Skin thickness and fibrosis by ultrasound/durometry compared to Day 0 [ Time Frame: Week 4 and Week 12, Week 17, Week 25 ]
    Evaluation of dermal and subdermal thickness and fibrosis of sentinel lesion(s) by ultrasound and/or durometry compared to Day 0

  3. Skin thickness and fibrosis by MRI (if available at site) compared to Day 0 [ Time Frame: Week 4 and Week 12, Week 17, Week 25 ]
    Evaluation of dermal and subdermal thickness and fibrosis of sentinel lesion(s) by MRI compared to Day 0

  4. Skin Thickness (ST) component of modified Localized Scleroderma Skin Severity Index (mLoSSI) compared to Day 0 [ Time Frame: Week 4, Week 8, Week 12, Week 16, Week 17, Week 21, Week 25 Week 29 ]

    Skin Thickness (ST) component of modified Localized Scleroderma Skin Severity Index (mLoSSI) of sentinel lesion where higher score represents worse outcome:

    0 = normal; 1 = mild increase in thickness; 2 = moderate increase in thickness, difficult to move skin; 3 = severe thickness, unable to move skin.

  5. Skin thickness by modified Rodnan Skin Score (mRSS) compared to Day 0 [ Time Frame: Week 4, Week 8, Week 12, Week 16, Week 17, Week 21, Week 25 Week 29 ]

    Improvement in the thickness of the skin as compared to Day 0. Scoring of sentinel lesion cutaneous area on a scale of 0 to 3 where higher value represents worse outcome.

    mRSS =0 is "normal skin" where the examiner appreciates fine wrinkles but no skin thickness is present.

    mRSS =1 is defined as definite but "mild" skin thickness where the examiner can easily make skin folds between 2 fingers; fine wrinkles are acceptable.

    mRSS =2 is defined as "moderate" skin thickness with difficulty in making skin folds and no wrinkles.

    mRSS =3 is defined as "severe" skin thickness with inability to make skin folds between 2 examining fingers.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Adults 18 years or older
  • Clinical diagnosis of any subtype of localized scleroderma with sclerotic lesions
  • Subject has not participated in previous clinical research study in the last six months
  • Subject must be on stable course of systemic immunosuppressive therapy without plan to change or is not on systemic immunosuppressive therapy
  • Subjects with good control of disease but have sclerotic lesions that have not responded to standard of care

Exclusion Criteria:

  • Patients with a non-morphea skin disorder in the region of interest
  • Localized scleroderma/morphea lesions on the face or over a joint, or lesions that can be successfully managed with topical medications or phototherapy
  • Current or <2 months prior to baseline use of UVA1 phototherapy
  • Presence of morphea profunda or eosinophilic fasciitis
  • Active infection with HIV, hepatitis B or hepatitis C
  • Contraindications to MRI
  • Any history of malignancy (except completely excised basal cell carcinoma or squamous cell carcinoma)
  • History of liver abnormalities or abnormal liver function tests in screening
  • Clinically significant cardiac abnormalities
  • Signs of infection of target lesion
  • Consistent course of physical therapy or plans to initiate physical therapy during the study
  • Current evidence of metastatic squamous cell carcinoma at the site to be injected.
  • Known allergy to any of the constituents of the product
  • Active drug or alcohol addiction.
  • Hypersensitivity to anesthesia used at medical site
  • Receipt of a chemical or biological study product for the specific treatment of localized scleroderma in the past six months
  • Pregnant or nursing
  • Any additional clinically significant laboratory abnormalities which may affect ability to safety participate in the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03740724

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Contact: Hirak Routh, MD 215-563-7330
Contact: Jennifer Parish, MD 215-563-7330

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United States, Pennsylvania
Paddintgon Testing Co., Inc. Recruiting
Philadelphia, Pennsylvania, United States, 19103
Contact: Hirak Routh, MD    215-563-7330   
Sponsors and Collaborators
Fibrocell Technologies, Inc.

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Responsible Party: Fibrocell Technologies, Inc. Identifier: NCT03740724    
Other Study ID Numbers: FI-SC-001
First Posted: November 14, 2018    Key Record Dates
Last Update Posted: December 24, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Fibrocell Technologies, Inc.:
Additional relevant MeSH terms:
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Scleroderma, Systemic
Scleroderma, Diffuse
Scleroderma, Localized
Connective Tissue Diseases
Skin Diseases