Page 4 - Scleroderm NZ Inc Newsletter November 16

Scleroderma Research Clinical Trials

Recently I had an enquiry asking for information on clinical trials in New Zealand
and Australia. Gordon sent me to a clinical trial registry called the Australian New
Zealand Registry (ANZCTR) on line: www.anzctr.org.au/BasicSearch.aspx

There are no trials in New Zealand on this
register but there are a number in Australia,
some of which they are currently recruiting
for.

There are drug trials to test the efficiency and safety of
Ambrisentan for pulmonary arterial hypertension (PAH). The
trial will also evaluate the effects of Ambrisentan on other
clinical measures of PAH, long-term treatment success, and
survival.

They are trialling another PAH drug: apixaban, an
anticoagulant, hoping that it will lessen the possibility of
blot clots in the lungs. The 3-year study will be double-blind,
placebo-controlled. It includes quality-of-life measures and
health resource use.

Another one for PAH is nuclear perfusion tomography for the I found details of a study into the effect of gloves in
Assessment of the Pulmonary Circulation. combination with traditional Thai massage, heat, and
stretching to improve hand mobility in scleroderma patients.
There are currently no widely available imaging assessments Recruiting is complete on this study.
for blood flow to capillaries in the lung, which is decreased in
PAH. Single photon emission computed tomography (SPECT) For Raynaud's there is Oral N-acetylcysteine in the Treatment
uses nuclear medicine to assess flow three-dimensionaly. They of Raynaud's Phenomenon Secondary to Systemic Sclerosis: a
wish to evaluate SPECT's usefulness to assess and detect PAH. randomised, double-blind, placebo-controlled clinical trial,
Recruiting hasn't started.
For severe cases of systemic sclerosis there is a study of the
There is also a study to investigate the effect of bosentan safety and efficacy of stem cell transplantation (HSCT) for
on pulmonary artery remodelling in PAH. It will investigate severe auto-immune diseases. Many of auto-immune diseases,
whether bosentan (Tracleer®) affects the wall thickness of the including scleroderma, resist available therapies. The study will
pulmonary arteries in patients with PAH of unknown cause try to find whether HSCT may work for them. Patients assessed
and PAH related to systemic sclerosis. Recruitment has not for suitability undergo procedures that include chemo and
started. immuno-suppressive therapy, followed by stem cell re-infusion.

For patients with limited scleroderma there's a study to find There is also a review of past HSCT on patients with severe
responses to genetic disorders. It compares the effects of systemic sclerosis and rheumatoid arthritis. The trial aims to
steroids that the body produces with introduced steroids assess its safety and efficacy. HSCT is reserved for patients with
and tranilast, an anti allergic drug, which inhibits collagen severe disease who have failed multiple previous therapies.
synthesis.
Evidence suggests HSCT may help suppress the inflammatory
The trial compares the safety and efficacy of tranilast in conditions and perhaps re-educate the immune system,
localised scleroderma. The drug has a good safety record especially, the thymus. As well as assessing HSCT's success, the
and has been available in Japan and Korea for 20-30 years. study will assess whether the thymus has a role to play in those
It has approval for the management of skin scarring in those who respond. They are currently recruiting.
countries.
All in all it is good to see that there are a few trials happening
The trial involves testing a corticosteroid cream on one close to home and it will be interesting to see the outcome.
affected part of the body and a second cream containing both
corticosteroid and the trial agent on another for 3 months. Dianne

www.scleroderma.org.nz