Morphea, Plaque FormPhase 1/2NCT03679845
Study to Assess Sarilumab in Halting Progression of Morphea
A Phase 1/2 study testing Sarilumab in people with Morphea, Plaque Form, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
An open-label single center trial studying the efficacy and safety of sarilumab on morphea patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Morphea, Plaque Form
- What is being tested?
- Sarilumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2019to 30 Jun 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subject 18 years of age or older
- Only circumscribed (plaque) morphea with at least one active morphea target lesion (0.5-10 cm2), with mLoSSI ≥ 5.
- Body surface area affected by morphea lesions: ≤ 50% at start of treatment
- 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.
- Willingness of subject to follow all study procedures
- Willingness to avoid excessive exposure of diseased areas to natural or artificial sunlight
Who cannot join
- Use any topical medication treating morphea within 14 days prior to Day 1
- Pregnancy or breast feeding
- 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
- Cancer within 5 years other than non-melanoma skin cancer or cervical cancer in situ that has been fully treated.
- Known hypersensitivity to any of the constituents or excipients of the investigational product
- Use of any prescription or non-prescription medication that could interfere with efficacy evaluations in the study
- Participation in another clinical research study with an investigational drug within 4 weeks before this study
Trial locations and contacts
1 site in 1 country.
United States1 site
CURTIS (Massachusetts General Hospital)
Boston, Massachusetts, 02114
About this study
Official title: A Pilot Study to Assess the Safety and Efficacy of Sarilumab in Halting Progression of Morphea
An open-label single center trial studying the efficacy and safety of sarilumab on morphea patients.
Read the detailed description
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.
- Conditions
- Morphea, Plaque Form
- Being tested
- Sarilumab
- Medical subject headings
- Scleroderma, Localized
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SarilumabDrug
Also called Kevzara
Sarilumab is an IL-6 monoclonal antibody that has shown to be potentially important in the creation of sclerosis in SSc.
| Study group | Role | What they receive |
|---|---|---|
| Sarilumab Arm | Experimental | 200 mg of sarilumab every two weeks Receives: Drug: Sarilumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
efficacy of sarilumab in plaque type morphea
To determine the efficacy of sarilumab in plaque type morphea by clinical responder rate at week 24.
Measured: Week 24
Secondary outcomes (1)
Physician Global Assessment of Activity (PGA-A)
100-mm morphea activity scale anchored by "inactive" at 0 and "markedly active" at 100.
Measured: Baseline and Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2018
- Study start1 Sep 2019
- Primary completion30 Jun 2021
- Study completion30 Jun 2021
Why it stopped: Difficulty in recruiting patients
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Morphea, Plaque FormStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Regeneron Pharmaceuticals
- Responsible party
- Principal investigator, Gideon Piers Smith, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03679845
- Sponsor's protocol ID
- 2018P002128
- Status last verified
- Aug 2021
- First submitted
- 17 Sep 2018
- Last updated
- 8 Sep 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Assess the Safety and Efficacy of Sarilumab in Halting Progression of Morphea
- ClinicalTrials.gov ID
- NCT03679845
- Acronym / sponsor ID
- 2018P002128
- Conditions
- Morphea, Plaque Form
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Difficulty in recruiting patients
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Regeneron Pharmaceuticals
- Study start
- 1 Sep 2019 (actual)
- Primary completion
- 30 Jun 2021 (actual)
- Study completion
- 30 Jun 2021 (actual)
- First posted
- 20 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An open-label single center trial studying the efficacy and safety of sarilumab on morphea patients.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sarilumab Arm | Experimental | Drug: Sarilumab200 mg of sarilumab every two weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female subject 18 years of age or older
- Only circumscribed (plaque) morphea with at least one active morphea target lesion (0.5-10 cm2), with mLoSSI ≥ 5.
- Body surface area affected by morphea lesions: ≤ 50% at start of treatment
- 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.
- Willingness of subject to follow all study procedures
Key exclusion criteria (7 listed)
- Use any topical medication treating morphea within 14 days prior to Day 1
- Pregnancy or breast feeding
- 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
- Cancer within 5 years other than non-melanoma skin cancer or cervical cancer in situ that has been fully treated.
- Known hypersensitivity to any of the constituents or excipients of the investigational product
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SarilumabAlso: KevzaraSarilumab is an IL-6 monoclonal antibody that has shown to be potentially important in the creation of sclerosis in SSc. | Drug | Sarilumab Arm |
Primary outcome
efficacy of sarilumab in plaque type morphea
Time frame: Week 24
To determine the efficacy of sarilumab in plaque type morphea by clinical responder rate at week 24.
1 secondary outcome
Physician Global Assessment of Activity (PGA-A)
Time frame: Baseline and Week 24
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2021.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sarilumab in people with Morphea, Plaque Form, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 8 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
efficacy of sarilumab in plaque type morphea (time frame: Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2021; AzTrials last refreshed its copy on 7 Nov 2026.