Starting a new clinical trial for a higher dose of hypoparathyroidism treatment
A columnist says she is eager to start a higher-dosage Yorvipath trial
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“It’s like déjà vu all over again.” — Yogi Berra
Getting on a plane recently brought back a rush of familiar feelings. I remembered 2019, when I took this same flight from Florida to New York City for my first clinical trial appointment.
I felt like I was reliving the same experience, except this trip was a screening appointment for Ascendis Pharma’s PaTHway60 Phase 3 clinical trial to investigate the safety and efficacy of palopegteriparatide (known by the brand name Yorvipath) at doses greater than 30 mcg/day, in adults like me with hypoparathyroidism who need larger doses.
Yorvipath, which helps to restore normal calcium and phosphate levels in the blood, is so far the only treatment approved by the U.S. Food and Drug Administration (FDA) to treat the underlying cause of hypoparathyroidism in adults. It was approved in 2024.
Seven years ago, I walked down the same Jetway to see Mishaela Rubin, MD, my hypoparathyroidism specialist, to be screened for the Phase 2 PaTH Forward clinical trial for the precursor to Yorvipath, known then as TransCon PTH. At the time, I felt excited, fearless, and hopeful.
Joining a clinical trial for an investigational medication was a big decision, but it gave me hope for managing my condition. I was so fortunate to be the first patient selected in the Phase 2 trial at Columbia University.
A new me, and then the old me again
After I stopped taking the prescription parathyroid hormone Natpara in 2017, I couldn’t seem to recover. I couldn’t stabilize my calcium levels and was quite unwell. I was ready to try something new, even if it meant feeling somewhat like a guinea pig. Participating in the TransCon PTH trial provided great hope for a better future, despite the unknown.
After my first dose of TransCon PTH, I felt alive again. In those first few years after starting treatment, I Rollerbladed five miles every day, worked, spent time with friends, and truly enjoyed life. It was incredible to feel normal again, something I hadn’t experienced in years.
Over time, my body responded so well to the medication that I needed higher doses. My dosage eventually was increased to the maximum of 60 mcg per day by the time the TransCon PTH trial ended for me in early 2025. But then I had to reduce the dosage to the FDA-approved dosage of 30 mcg per day, which my body did not like.
As a result, this past year has been difficult. The hopelessness I felt in 2017 after stopping Natpara returned. I felt alone, and no one knew how to help me. Being one of the first clinical trial participants who needed the highest dose, and then having to cut it in half, was really hard. There was no advice or solution to offer, since so few people were in my situation. I was the data.
That’s what being in a clinical trial is like. You don’t know what the outcome will be. I loved how it began, but the ending proved difficult.
I have many feelings about starting this over-30 trial, because I don’t want to repeat what happened last time by having to decrease my dosage if the FDA doesn’t approve the data that are collected.
It took five hospital stays and a lot of effort to get to where I am now. I don’t want to relive that. I do want to have the amazing life I was gifted while on my trial dose of 60 mcg per day.
In many ways, going through this process feels very familiar: It involves the same steps, the same hopes, and the same nerves. But things are a little different this time, and it feels like repeating the past with a new twist.
When I sat with Rubin at my recent screening, we agreed that we had started this journey together as doctor and patient number one, and we should finish it together. She reminded me that what I’m doing for science will help many people. My doctor and I are a team and will work together to help me live my best hypopara life.
Acknowledging the signs
Columnist Bari Vapnek rides the Central Park Carousel in New York City, after an encouraging screening with her doctor for a clinical trial. (Courtesy of Bari Vapnek)
After leaving Columbia, I had a lot to think about. I walked through New York’s Central Park and found the carousel. Naturally, I rode it like I used to do as a kid. I remembered that 2026 is the Year of the Horse, and I need to saddle up and see this through.
When I returned to my hotel room and turned on the TV, I saw a Yorvipath commercial. I took these two signs as proof that I need to finish what I started with Rubin.
As I boarded the plane for my return flight to Florida, I felt excited and hopeful, just like I did in 2019. There is nothing better than having hope and knowing you are helping future generations.
This brave butterfly looks forward to continuing to support scientific research, to foster hope in the community, and to raise awareness for everyone with hypopara, one Yorvipath injection at a time.
Note: Hypoparathyroidism News is a strictly news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hypoparathyroidism News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hypoparathyroidism.
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