Weighing the pros and cons of participating in a clinical trial

Earlier this month, I read something in the Amyloidosis News Today newsletter that rekindled my curiosity about the investigational gene-editing therapy nexiguran ziclumeran (nex-z). Its developers shared an encouraging update about an analysis they’d done of the Phase 3 clinical trial known as MAGNITUDE. My strong interest in the development of nex-z is rooted deeply in my hopes for a better treatment for my mom, and potentially myself.

My mom was diagnosed with hereditary cardiac amyloidosis in Taiwan last year. Later, I was confirmed to be a carrier of the TTR gene mutation that causes the condition. Around that time, I came across an Amyloidosis Research Consortium video discussing ATTR amyloidosis clinical trials, including MAGNITUDE.

I was particularly curious about nex-z because it could potentially offer a one-time treatment that stops the body from producing the TTR protein that misfolds to form amyloid. I researched further and learned that the study was recruiting patients in Taiwan as well. My hope quietly and gingerly rose to the surface when I saw the possibility for my mom to participate.

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Emotional dilemmas

The thought of having my mom participate in a clinical trial is a complicated one that provokes numerous internal debates. If efficacy is proven, it offers a chance to access the newest treatment years ahead of commercial availability in the local market. However, if a negative reaction occurs, the result may be irreversible — or, worse, it may lead to death.

My newly sprouted curiosity came to an abrupt halt last year after the U.S. Food and Drug Administration (FDA) placed a clinical hold on the study in October 2025, after a participant suffered severe liver toxicity and died. I decided not to discuss the trial with my mom or the rest of the family. That news sounded exactly like what patients and family members fear most.

On the other side of the debate, accessing the newest treatments outside the U.S. is often difficult. On top of the already long development and regulatory approval timelines in the U.S., international patients often wait an additional two or three years or longer for the therapy’s application filing, regulatory review, and pricing negotiations. For a condition like transthyretin amyloidosis with cardiomyopathy, or ATTR-CM, which doesn’t yet have a cure, access to newer, more advanced treatments is critical.

After reading the second quarter updates from nex-z developer Intellia Therapeutics and listening to its webcast, I came to better understand the profiles of patients who are more likely to have an adverse reaction, and how each event in a study offers insights that help optimize treatments for other patients later. This time, I brought up the topic with my parents to gauge their initial reactions. Like me, they were curious about the potential benefits and cautious about the risks.

The reality is, even if we decide to participate after discussing it with her doctor, we’re not certain that my mom is eligible for the study or even if there’s still room for enrollment. And once enrolled, there’s a one-in-three chance of being given a placebo.

Somehow, all this uncertainty seems to ease the anxiety. We agreed to start the conversation with my mom’s cardiologist in a few weeks and go from there. I’m telling myself not to have high expectations but to make a lot of preparations for a new adventure as a family living with a rare disease.

Off we go!

Note: Amyloidosis News Today is strictly a 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 other 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 Amyloidosis News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.