Study to Assess Sarilumab in Halting Progression of Morphea

Study Purpose

An open-label single center trial studying the efficacy and safety of sarilumab on morphea patients.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Male or female subject 18 years of age or older. 2. Only circumscribed (plaque) morphea with at least one active morphea target lesion (0.5-10 cm2), with mLoSSI ≥ 5. 3. Body surface area affected by morphea lesions: ≤ 50% at start of treatment. 4. If subject has received any morphea treatment, subject must be on a stable regimen, which is defined as not starting a new drug or changing dosage within 8 weeks prior to Day 1. Subject must be willing to stay on a stable regimen during the duration of the study. 5. Willingness of subject to follow all study procedures. 6. Willingness to avoid excessive exposure of diseased areas to natural or artificial sunlight.

Exclusion Criteria:

1. Use any topical medication treating morphea within 14 days prior to Day 1. 2. Pregnancy or breast feeding. 3. Any condition (e.g. HIV, diabetes, ANC(absolute neutrophil count) < 2,000/mm3, platelets < 150,000/mm3 or AST(aspartate transaminase)/ALT(alanine aminotransferase) > 1.5 times normal limits) or therapy that in the investigator's opinion may pose a risk to the subject or that could interfere with any evaluation in the study. 4. Cancer within 5 years other than non-melanoma skin cancer or cervical cancer in situ that has been fully treated. 5. Known hypersensitivity to any of the constituents or excipients of the investigational product. 6. Use of any prescription or non-prescription medication that could interfere with efficacy evaluations in the study. 7. Participation in another clinical research study with an investigational drug within 4 weeks before this study

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03679845
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1/Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Massachusetts General Hospital
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other, Industry
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Morphea, Plaque Form
Additional Details

Morphea, like systemic sclerosis (SSc), presents with sclerotic hardening of the skin. However, unlike SSc, morphea has no systemic involvement. While this skin-limited, chronic sclerosis, has no impact on mortality, it can have significant morbidity, as lesions can affect joint mobility, and be disfiguring, especially when involving the face. While some patients respond well to use of steroids, whether topical or intralesional, antimalarials such as plaquenil, or traditional immuno-suppressants there is a significant proportion of patients who remain non-responsive to these treatments, or require high dosages of these, oral steroids, or experimental therapies to suppress the condition. For this group of patients there is a high clinical need to find alternate therapies. In addition, as the disease creates permanent scarring, and traditional immuno-suppressants take several months to take effect there is a need for medications that can act more quickly to halt the progression of scarring. Although the pathways of inflammation are poorly understood, one cytokine of potential interest is IL (interleukin)-6. In the bleomycin mouse model of skin sclerosis targeting IL-6 by both passive and active immunization strategies prevented the development of bleomycin-induced dermal fibrosis. Dysregulation of IL-6 has also been seen in the serum of patients suffering from SSc. As a result, preliminary clinical trials have been undertaken in SSc. While results did not reach statistical significance the skin thickening was better in the treatment group than in placebo and the lack of signal is likely due to the late stage of patients skin involvement, and the slow progression of thickening in SSc. In contrast morphea often progresses rapidly and as such is much more likely to give a clear clinical signal. Recently sarilumab, an anti-IL-6 monoclonal antibody, has been approved for use in severe rheumatoid arthritis (RA) after rapid and sustained results in clinical trials. Given its good safety profile, its impressive response in RA, and the immunohistochemical evidence that IL-6 may be important in creation of sclerosis in SSc, the investigators propose a pilot study to determine the safety and efficacy of sarilumab in morphea.

Arms & Interventions

Arms

Experimental: Sarilumab Arm

200 mg of sarilumab every two weeks

Interventions

Drug: - Sarilumab

Sarilumab is an IL-6 monoclonal antibody that has shown to be potentially important in the creation of sclerosis in SSc.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

CURTIS (Massachusetts General Hospital), Boston, Massachusetts

Status

Recruiting

Address

CURTIS (Massachusetts General Hospital)

Boston, Massachusetts, 02114

Site Contact

Maria Alora, MD

[email protected]

617-726-5066