ILD Support Group Recap #2

What is learned in clinical trials? To advance medical and scientific knowledge. Results may not help an individual participating in a clinical trial but adds to overall knowledge for the larger group. And maybe our children and grandchildren.

At the April ILD Support Group last Tuesday, Archer Eller, the Clinical Trial Manager gave a brief interesting example.

Reading this blog, you may remember that my ILD Hypersensitivity Pneumonitis is the only lung disease that does not develop in smokers. In a Phase 2 study, the HP patients and a control group inhaled carbon dioxide and it was there that it was discovered that smoking killed the antigens before they were allowed to develop into HP. Interesting.

The next clinical trial we discussed was the BIBF 1120 #1199.34 study first introduced in this blog: http://livingwellwithabaddiagnosis.blogspot.com/2011/07/latest-drug-and-treatment-guidelines.html

This study is a worldwide study involving an anti-fibrotic drug. Fibrosis does not just happen in the lungs. It can also develop in the liver and kidneys. This drug inhibits the pathways to stop fibrosis from developing.

In Phase 2, 100 people were randomly given a placebo, 100 on 50 mgs, 100 on 100 mgs and 100 on 150 mgs. They found the difference in the dose effect was 60%. A big deal.

In Phase 3, 500-600 people will be tracked. The number of qualified patients exceeded the goal and the study is now closed waiting to begin by the end of the year. It will be a 52-week trial.

We next discussed the PANTHER study first discussed in this blog here: http://livingwellwithabaddiagnosis.blogspot.com/2011/08/research-regarding-pulmonary-fibrosis.html

When problems appeared, I reported back in this blog:  http://livingwellwithabaddiagnosis.blogspot.com/2011/10/scary-news-about-n-acetylcysteine-nac.html

Here is latest on this study:

The study began in Europe with 100 IPF patients on a placebo and 100 on N-acetycysteine (NAC) which is an over the counter supplement. They were divided up into three groups:

Placebo

NAC

Prednisone, Imuran and NAC

Archer reported that of the three people from this university hospital in the third group of all three drugs, two dropped out due to side effects. They were primarily suffering from hallucinations. While this study was happening, the Data Safety Management Board discovered very serious problems in the third group of iPF patients taking prednisone, Imuran and NAC. They pulled that part of the study.

The other two groups will continue with a trial for a 52-week study, which is still being enrolled soon. After that year, the data will be reviewed and the results of this study should be published in two years.

He also briefly mentioned a new Phase 2 study of a twice a week injection in IPF patients to treat fibrosis.

The next Clinical Trial discussed was for connective tissue diseases like Scleroderma. The National Institute of Health is running the 52-week study and will follow it for two years afterwards. Scleroderma is an autoimmune disease but when it develops in the lungs, it is the most serious aspect of the disease. The only treatment now is a lung transplant.

Scleroderma Lung Study I (SLS I) involved a doctor using Cellcept as an immunosuppressant in 2 or 3 people with Scleroderma noticed that it was helping. He approached the drug company Roche then did another small study with 7 patients. They all did better.

SLS II had a group of people placed on a placebo for an entire year. After the year, half were given Cytoxan. The study was to compare Cellcept to Cytoxan for an additional year. There was no placebo-only group. They are half way in their enrollment and the results will be published approximately two years after the study. If you have Scleroderma, please ask your doctor to include you in this study.

Tomorrow’s blog will discuss observational studies and bench work as well as a story about a new person to our group.