Published on September 13, 2022 | At the age of 5, Ian Jones (name changed for privacy) was diagnosed with ARPKD. During a routine trip to his pediatrician, his mother mentioned that Ian was having a lot of trouble staying dry at night. Tests revealed that he had cysts on his kidneys. There was no family history of the disease, and Ian’s parents had never heard of PKD.
As research into PKD ramps up, there are increasing opportunities to enroll children in clinical studies, says Ashima Gulati, M.D., Ph.D., a pediatric nephrologist at Children’s National Hospital in Washington, D.C., where Ian follows up in the Inherited and Polycystic Kidney Diseases Program. “Choosing to participate is understandably a difficult decision for many families, and parents should feel free to ask as many questions as they want to make an informed decision,” Dr. Gulati says.
Now 9 years old, Ian’s parents enrolled him in an ARPKD clinical study. In order to learn about what went into their family’s decision, We talked to Ian’s mother, Alixandria.
What made you decide to have your son participate in a clinical study?
Alixandria: Ian’s diagnosis was devastating for me. I struggled with the fact that ARPKD is degenerative and has no treatment or cure. The only way I knew how to move forward was to get involved. I joined the PKD Foundation and connected with other parents on Facebook.
Through these contacts, I found out about the first clinical study we would participate in. I feel it’s important to do what we can to advance research toward finding treatments and eventually a cure.
Did you involve Ian in the decision?
Alixandria: We want Ian to feel empowered to take ownership of his body and health. When the opportunity to participate in a study came up, I explained to him that the doctors are trying to find new medicines for him and kids like him with ARPKD. If he participated, we would be helping the doctors.
What were your main concerns about enrolling him in a clinical study?
Alixandria: Prior to discovering Ian’s ARPKD, I didn’t personally know anyone who had participated in a clinical study. I was afraid of what I didn’t know, so I started asking questions. The study team did such a great job of addressing my concerns and answering Ian’s questions that we were really excited for him to participate.
What advice do you have for parents thinking about enrolling their children in a clinical study?
Alixandria: If there is a clinical study that you may be interested in, contact the research team and ask questions. The answers may help you make your decision and alleviate some common fears. I would also suggest talking to your medical team. They are on this journey with you, and it’s important to have an open dialogue with them. Lastly, trust yourself. You know what is right for you and your family.
There’s a lot to consider before joining a clinical study. To learn more about clinical studies, this helpful list of FAQs can get you started. When you’re ready, you can sign up for our Accelerating Clinical Trials (ACT) Alert program. Through ACT Alerts, you’ll receive emails about studies in your area. Additionally, alerts are separated into ARPKD and ADPKD studies. Together, we can further PKD research and help #endPKD.
Want to see more articles like this? Read the full issue of our magazine, PKD Life, and subscribe to future issues here. If you’re interested in ARPKD, you’ll also find more ARPKD-related articles in our summer issue.
This blog post is sponsored by Reata America Pharmaceuticals, Inc., a proud sponsor of the 2022 Walk for PKD.