If you are living with scleroderma, joining this study might be a suitable option for you.

Scleroderma Australia has joined the global effort to find better choices for treatment in the future for people with scleroderma.

This Clinical Study is looking at an investigational product as a potential treatment for people who have diffuse cutaneous systemic sclerosis (dcSSc – a form of scleroderma).

Why?
There is no cure yet for scleroderma, and current treatment for the disease centres around treating individual symptoms as they occur. There are currently no therapies to address the underlying cause of the disease. In this study we are looking at the safety and effectiveness of an investigational product (the study drug). This study drug is a chemical that enhances your body’s natural mechanism for relaxing blood vessels. We think that this might help slow down the damage scleroderma causes.

What?
People who join the study will need to make at least 14, and up to approximately 20 visits to our study clinic. The main part of the study lasts around a year, but participants have the option to continue receiving study treatment for up to sixteen months after that. Not everyone in the study will be given the study drug, some will be given a placebo that looks the same as study drug but contains no active ingredients. 

Who?

You may be able to participate if you:

• Are 18 years of age or older
• Have Diffuse Scleroderma (Systemic Sclerosis) of less than 5 years duration
• To be classified as Diffuse Scleroderma you must have thick and tight skin in at least one of the following places – the upper arms, the thighs, or the abdomen.

In addition to these major criteria to be eligible you will also need to have:

• Some progression of your skin thickening over the last 6 months or have very early Scleroderma that started less than 2 years ago
• Raynaud’s and digital ulcers in the past
• Raised inflammation markers [blood tests Erythrocyte sedimentation rate (ESR)
  or C-reactive protein (CRP)]

You will not be able to enter the study if you:

• Have Limited Scleroderma with skin thickening only on hands , arms, face and chest
• Have had Diffuse Scleroderma for more than 5 years
• Have problems with your kidneys and liver as assessed by the study doctor
• Have had Severe lung disease or severe Pulmonary hypertension
• Have received Rituximab in the last 6 months

YOU can however be currently taking Mycophenolate, methotrexate or azathioprine, but the dose must be stable for 4 months before you can start the trial.

Please note, there will be other requirements you need to be met to take part in the study.

Why should you take part in this study?

Taking part in this study is completely voluntary.

By taking part in the study, participants will help the research team learn more about whether the investigational product has the potential to become a treatment for scleroderma in the future.
Participants receive the study medicine, study-related medical tests, and follow-up care at no cost. Reimbursement for study-related expenses (such as meals and travel expenses) may also be available.
It is possible that participants could experience side effects while being in the study. There may be side effects that are not yet known. There are other potential risks from tests and procedures that will be done as part of this study. Everyone who joins the study will be closely monitored by the study team throughout their participation.

Potential Benefits?

We cannot promise you will directly benefit from joining the study, but your participation could help patients in the future. Taking part in the study is free and you may receive reimbursement for study-related expenses (for example, travel and meals).

Risks?

It is possible that you could experience side effects while being in the study. There may be side effects that are not yet known. There are other potential risks from tests and procedures that will be done as part of this study. You will be closely monitored by the study team throughout your participation in the study.

What should you do if you are interested?

Contact the Investigators in Australia to find out more

PI: Dr Wendy Stevens
Site: St Vincent’s Hospital Melbourne
Study Coordinator: Elizabeth Reidy
Phone Number: +61 3 9231 3983
Email Address: Elizabeth.Reidy@svha.org.au

PI: Prof Susanna Proudman
Site: Royal Adelaide Hospital
Study Coordinator: Cindy Hall
Phone Number: +61 8 7074 4068
Email Address: Cindy.Hall@sa.gov.au

PI: A/Prof Tamera Corte
Site: Royal Prince Alfred Hospital
Study Coordinator: Tina Li
Phone Number: +61 2 9515 4188
Email Address: Qi.Lin@health.nsw.gov.au

PI: Dr Ken Cai
Site: Westmead Hospital
Study Coordinator: Annemarie Saliba
Phone Number: +61 2 8890 4605
Email Address: Anne.Saliba@health.nsw.gov.au

PI: Dr Michael Oliffe
Site: Liverpool Hospital
Study Coordinator: Fiona Briggs
Phone Number: +61 2 8738 4088 |
Email Address: Fiona.Briggs@health.nsw.gov.au