How to decide on enrolling your child with special needs in a clinical trial

Parenting a special needs child comes with many trials, but one trial that I was not planning to face is the clinical trial.

Marfan syndrome

Every person’s experience with Marfan syndrome is slightly different. Some features of Marfan syndrome are easier to see than others. These include:

  • Long arms, legs and fingers
  • Tall and thin body type
  • Curved spine
  • Chest sinks in or sticks out
  • Flexible joints
  • Flat feet
  • Crowded teeth
  • Stretch marks on the skin that are not related to weight gain or loss
  • Harder-to-detect signs of Marfan syndrome include heart problems, especially those related to the aorta. Other signs can include sudden lung collapse and eye problems, including severe nearsightedness, dislocated lens, detached retina, early glaucoma and early cataracts.

If your life is like mine, then you can easily imagine you’re at Ann and Robert H. Lurie Children’s Hospital for a typical day-long visit with multiple doctors and tests, when suddenly you are blindsided with the mention of enrolling your child in a clinical trial.

Your initial reaction might be an emphatic “no” or an enthusiastic “yes,” but most likely it is “I have no idea what to do!” You think, I want to protect my child, but what if my child could benefit from the research gained from the trial?

You, of course, want a miracle, which seldom happens in a clinical trial.

Parents react differently to their child’s diagnosis. However, one thing most special needs parents have in common is our quest for knowledge and information. Learning as much as humanly possible about our child’s issues can arm us with the power to feel more in control of the situation.

Upon my daughter Gabriella’s diagnosis of Marfan syndrome at age 5, I spent every free moment delving into documents, articles and papers I could find on Google Scholar. I also was lucky to find a great resource in The Marfan Foundation. I immediately joined their community of parents online.

I quickly discovered Marfan syndrome is a life-threatening genetic disorder that affects the body’s connective tissue. An early, accurate diagnosis is essential.

I was suddenly wishing I had been more successful in the genetics unit of biology class. But it didn’t take long before I was being mistaken for a doctor when visiting the emergency department with my daughter. When a resident said, “It’s nice you are a doctor,” I smiled and said, “Oh, I’m not a doctor! Just an informed parent.”

The clinical trial decision must be treated in much the same way as the diagnosis, by becoming informed.

Recently, I spoke to Dr. Luciana Young, the cardiologist at Lurie Children’s Hospital, for tips other parents could use when contemplating a clinical trial. Young cares for my daughter and was the site coordinator for the “Atenolol (beta-blocker) vs. Losartan (Angiotensin Receptor Blocker) in Marfan Syndrome” clinical trial that my daughter Gabriella participated in for three years.

Young’s advice: Be well informed.

“In order to consent, you want to know the positive outcomes and possibilities as well as any potential negative effects, especially if your child will be starting a new medication,” she says.

Ask questions. Has the medication been tested before? Is the dosing approved? Is it FDA-approved?

“It’s very important that children enroll in these trials because the research must test what the doctors have learned,” she says.

Consider your child’s opinion. Young believes the child should be part of the decision about enrolling in the trial or not. Never underestimate what your child can handle.

In my family this was a big ordeal. Upon diagnosis, we faced the clinical trial question, then found she just missed qualifying.

However, we still had to make an immediate decision about medication. Should we start her on the standard treatment, Atenolol, now or wait for another opportunity to enroll in the trial?

Ultimately we chose to start treatment immediately, and by the time the possibility of enrolling in the trial came to be, we knew we wanted to do it if Gabriella qualified.

Some of the specific questions we wanted answered were about potential side effects of the two medications and what exactly would be required of Gabriella along the three-year journey. Gabriella was especially interested to know how many blood-draws she would need and how many heart monitors she would have to wear.

She also was very interested to know what the trial really meant. We discussed what the doctors were trying to learn and how they needed at least 500 children in the trial to give them valid research results.

In the end she said she wanted to help the doctors learn more so they could help kids like her.

Now that she is 10, and I’m looking back on all of this, I am not surprised by my daughter’s maturity.

I attended the press conference for the trial results at the American Heart Association’s Scientific Sessions held in Chicago late last year.

Dr. Ronald Lacro presented the results. The trial was very important on its own merit, but will be even more powerful combined with similar trials, he said.

The results didn’t provide a miracle, but the trial did shed light on valuable information that will help my daughter and other kids like her.

Both doctors agreed that dosing was an unexpected factor learned from the trial. The children were able to tolerate higher dosages without adverse effects, meaning doctors shouldn’t be afraid of higher doses to get desired results.

It was unexpectedly proven that early treatment may be even more beneficial than waiting until the aorta has reached a certain size.

Doctors also now know that both Atenolol and Losartan are effective in treating cardiac issues related to Marfan syndrome.

And Gabriella was a part of that valuable research.

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