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.|
|ClinicalTrials.gov Identifier: NCT03740724|
Recruitment Status : Recruiting
First Posted : November 14, 2018
Last Update Posted : December 5, 2018
|Condition or disease||Intervention/treatment||Phase|
|Morphea Scleroderma, Localized Scleroderma||Genetic: FCX-013 Drug: veledimex||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Estimated Study Start Date :||December 2018|
|Estimated Primary Completion Date :||December 2020|
|Estimated Study Completion Date :||March 2021|
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.
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
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
- 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.
- 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
- 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
- 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
- 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.
- 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.
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 ClinicalTrials.gov identifier (NCT number): NCT03740724
|Contact: Jennifer L Parish, MDfirstname.lastname@example.org|
|United States, Pennsylvania|
|Paddintgon Testing Co., Inc.||Recruiting|
|Philadelphia, Pennsylvania, United States, 19103|
|Contact: Hirak Routh, MD 215-563-7330 email@example.com|