Clinical Trials and Studies

What is a Clinical Trial?

A clinical trial is a study that involves a set number of people to test the effectiveness of a certain medication.

What is a Study?

This is normally done in  your clinic by various pharmaceuticals or researches gathering information where there is no medication involved. Usually based on survey's or blood draws to study the dynamics of  

Why are Clinical Trials important?

They play a very important role in the development of new medications to treat any number of diseases. Without trials, there would be no new treatments developed to help live a better life than what we are!

Why participate in a Clinical trial or a study?

Speaking from a Patient point of view living with Pulmonary Hypertension, a rare and debilitating disease, there aren't enough medications out there compared to other well known diseases like that of cancer, diabetes etc. So why not help find better medications and give your time to answer questions regarding your overall health?

To be a part of a clinical trial you have to take into consideration all of the pro's and cons. I remember when my mom was diagnosed in 1997 with PH, reminder, mine is hereditary.  She came home and said she only had the option of taking one type of drug, flolan. She was a full time employee (Manager) and a mother of 3 and grandmother of 2 small grandchildren with one on the way.   She came home from one of her appointments and said, we need to have a family meeting because I have a decision to make.  So we all sat down and she said, I can go on this drug which will hinder a lot of things I can currently do, or I was given an option to go on a blind clinical trial to test a drug for a new medication they are testing called Subcutaneous Remodulin.  I won't know until after a year if the medication is the real thing or a placebo. I actually still have all that paperwork 😊 Some of the family was against it, but the more her and I spoke the more she was leaning to going for the trial.  Finally she made her decision and went with the trial.  She had to go to the hospital for a few days to learn how to administer the medication, had a visiting nurse come to the house to show us how to help her in the event she couldn't do this for herself.  It was a long process going every few weeks to get monitored. But the thing with this was, we knew right off the bat she had the real thing, it stunk to high heaven! Her site became inflamed and she was gaining more energy. She also was  breathing easier.  She mentioned it to her doctor and he said he couldn't tell her that information, as he didn't know either, it was a blind study.  Anyway, without her partaking in this trial, she wouldn't have lived as long as she did, and it may have taken longer for it to be developed. 

Participating in a clinical trial can be a very satisfying and a worthwhile experience.

• Taking an active role in managing your care
• Gaining access to new treatments before they are more widely available
• Helping advance our knowledge of PH
• Receiving a treatment that works for you 

I have and still am in studies.  One was with medication that was already FDA approved. It was called the Symphony Program. You had to be willing to add Macitentan (Opsumit) to your current pathway, and they would monitor you for a total of 5 1/2 months with going back and forth every 6 weeks to the doctors office and answering questionnaires on a table and sending the information to the study company on how we were feeling while taking the medication.   Within a few weeks of me starting this, I was able to increase my 6 MWT by a total of 6 laps. You can find that trial and the results here: https://clinicaltrials.gov/ct2/show/NCT01841762

The other study I participated in was the Genetic code testing. Where they they took two bottles, not test tubes, of your blood and they study it for deficiencies and other things to see how they can possibly come up with more medication/cure for the disease.  The current study I am in is a sister study to the one I just mentioned. It is to do a case study on women, since we are most prone to getting this horrid disease.  It is basically an environmental case study that only involves questions. I will do these questionnaires every 6 months. 

There are also risks when considering a clinical trial:

• Side effects of the medications or treatments being studied
• Unwanted events during the trial that may or may not be related to the study drug
• Failure of a treatment to work

When doing a clinical trial, there has to be so many patients willing to partake in order for it to be a successful study, we'll get into that now. 

What is the protocol for a clinical trial?

How many patients will participate
Who is eligible
What tests patients will get and how often
What type of data will be collected
Detailed information about the treatment plan

Every clinical trial is different and lasts for a different amount of time.  If they do not get enough participants, the trial ends without any results.  There are a lot of factors to consider, the biggest is who is eligible. 

How can you get involved with a clinical trial or study?

Speak with your PH Team and see if they have any trials or studies taking place. If not, ask them to refer you to another clinic that has one that is open and registering patients. Remember though, they will discuss with you your condition and whether the trial would be a match for your condition.  Not all trials are able to be done by all patients. 

Where can you look up clinical trials and studies?

You can go here:  www.clinicaltrials.gov 

You will find currently recruiting, past studies/trials, completed, and terminated trials/studies.  I hope you found this information helpful, and thank you for reading. Stay tuned for my next blog:  Not being alone in  your PHight 😊💜