NCT03740724

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

Terminated Phase 1, PHASE2 Results posted Last updated 23 January 2024

What this trial tests

Phase 1, PHASE2 trial testing FCX-013 in Morphea in 1 participant. Terminated before completion.

Timeline

18 December 2019

Primary endpoint
23 September 2020

21 April 2022

Quick facts

Lead sponsor	Castle Creek Biosciences, LLC.
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	18 December 2019
Primary completion	23 September 2020
Estimated completion	21 April 2022
Sites	1 location across United States

Drugs / interventions tested

• FCX-013
• veledimex — full drug profile →

Conditions studied

• Morphea — all drugs for Morphea →
• Scleroderma, Localized — all drugs for Scleroderma, Localized →
• Scleroderma — all drugs for Scleroderma →

Sponsor

Castle Creek Biosciences, LLC. — full company profile →

Who can join

18 and older, any sex, with Morphea or Scleroderma, Localized. Patients with the condition only — healthy volunteers not accepted.

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were collected from the time the first subject signed the informed consent form until the final visit/contact with the subject and through study completion/termination (approximately 28 months).. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

FCX-013 + Veledimex

Serious: 0/1 (0%)

Deaths: 0/1

Other adverse events (1 terms — click to expand)

Reaction	System	FCX-013 + Veledimex
Injection site pain	Skin and subcutaneous tissue disorders	—

Data from ClinicalTrials.gov NCT03740724 adverse events section.

Sponsor's own description

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

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Morphea: The 2023 update.
   Papara C, De Luca DA, Bieber K, Vorobyev A, et al · · 2023 · cited 55× · PMID 36860340 · DOI 10.3389/fmed.2023.1108623
2. Immunomodulatory roles of metalloproteinases in rheumatoid arthritis.
   Bian Y, Xiang Z, Wang Y, Ren Q, et al · · 2023 · cited 45× · PMID 38035026 · DOI 10.3389/fphar.2023.1285455
3. Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease.
   Marquez Loza LI, Yuen EC, McCray PB. · · 2019 · cited 43× · PMID 30875857 · DOI 10.3390/genes10030218
4. Distinct Metalloproteinase Expression and Functions in Systemic Sclerosis and Fibrosis: What We Know and the Potential for Intervention.
   Leong E, Bezuhly M, Marshall JS. · · 2021 · cited 22× · PMID 34512395 · DOI 10.3389/fphys.2021.727451
5. Upcoming treatments for morphea.
   Wenzel D, Haddadi NS, Afshari K, Richmond JM, et al · · 2021 · cited 20× · PMID 34272836 · DOI 10.1002/iid3.475
6. Lentiviral vectors for inducible, transactivator-free advanced therapy medicinal products: Application to CAR-T cells.
   Tristán-Manzano M, Maldonado-Pérez N, Justicia-Lirio P, Cortijo-Gutierréz M, et al · · 2023 · cited 7× · PMID 37125150 · DOI 10.1016/j.omtn.2023.03.018