HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
This research, called HELIOS-A, is a large global study looking into a new medicine known as Amvuttra. It's for people who have an inherited condition called hereditary transthyretin amyloidosis, or hATTR amyloidosis. This condition can affect nerves and other body parts. The study wants to find out if Amvuttra is an effective and safe treatment. Researchers will compare how much Amvuttra helps improve nerve symptoms and quality of life against previous findings from a different treatment called Onpattro. Participants will receive either Amvuttra or, for comparison, information from those who previously received a placebo (dummy medicine) or Onpattro.
At a glance
What is this study about?
This study, called HELIOS-A, is investigating a new medicine called Amvuttra. It’s for people who have a rare inherited genetic condition called hereditary transthyretin amyloidosis (hATTR amyloidosis). This condition happens when a specific protein, called transthyretin, doesn't fold correctly and builds up in different parts of the body, including nerves, the heart, and other organs. These protein deposits can cause various symptoms, such as numbness, tingling, and weakness, as well as problems with the heart or digestive system.
The main goal of this study is to see if Amvuttra can help improve the nerve-related symptoms of hATTR amyloidosis. Researchers will be looking at changes in a special score that measures nerve damage and how well people can do daily activities. They will compare the results for people taking Amvuttra with information gathered from a previous study that looked at another medicine (Onpattro) or a dummy treatment (placebo). This helps them understand how well Amvuttra works.
Beyond just nerve symptoms, the study will also look at other important things. This includes how the medicine affects a person's quality of life, their ability to walk a certain distance, how their body weight changes, and overall disability. By looking at all these different measures, the researchers hope to get a full picture of how Amvuttra might benefit people living with hATTR amyloidosis.
Key takeaways
- This study is testing a new medicine called Amvuttra for a rare inherited condition.
- It aims to see if Amvuttra is safe and helps improve symptoms like nerve problems.
- Participation involves regular clinic visits for assessments and receiving the study medicine.
- Both men and women, aged 18 and over, with hATTR amyloidosis can be considered.
- Comparing Amvuttra's effect on symptoms and quality of life is a key focus.
- You can stop participating in the study at any time.
Who may be eligible?
To join this study, you must be an adult, aged 18 years or older, with a diagnosis of hereditary transthyretin amyloidosis. Both men and women are welcome to participate.
The study team will carefully check your medical history and current health to make sure this study is right for you. There are specific health requirements that need to be met, and others that might mean you couldn't take part. This is to ensure your safety and that the study results are clear.
It's important to remember that the research team makes the final decision on who can join, based on a detailed review of your individual health circumstances. They will explain all the requirements to you clearly.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Have you been diagnosed with hereditary transthyretin amyloidosis (hATTR amyloidosis)?
- Are you able to attend regular clinic appointments?
- Are you willing to receive the study medication as an injection?
What does participation involve?
If you join this study, you will receive the study medicine, Amvuttra, which is given as an injection using a pre-filled syringe. The study will compare your progress with information from people who previously received another treatment (Onpattro) or a dummy medicine called a placebo. Over the course of the study, you'll have regular visits to the clinic. During these visits, the study team will carry out various assessments. These will include checks on your general health, measuring changes in your nerve symptoms using a specific scoring system, and tests to see how your quality of life, walking ability, and overall disability are affected. You'll also have blood tests. The total duration of your participation in the study, including all follow-up appointments, will be clearly explained by the study team.
Potential risks and benefits
Locations (7)
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Common questions
What is hereditary transthyretin amyloidosis (hATTR amyloidosis)?
It's an inherited condition where faulty proteins build up in your body, affecting nerves and other organs.
What is Amvuttra?
Amvuttra is the new medicine being tested in this study for hATTR amyloidosis. It's given as an injection.
Will I get the new medicine?
Yes, if you join this study, you will receive Amvuttra. Your progress will be compared to past results from other treatments or dummy medicines.
What will researchers be looking for?
They will measure changes in your nerve symptoms, quality of life, walking ability, and overall disability.
How long will the study last?
The study team will tell you the exact duration, including how many visits and follow-ups you will need.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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