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hATTR amyloidosis treatment research continues
Learn more about participating in the HELIOS-A research study
HELIOS-A Research Study
HELIOS-A is a research study seeking individuals diagnosed with hATTR amyloidosis to determine if an investigational medicine can help improve neurologic symptoms.
Am I Eligible for the HELIOS-A Study?
You may be eligible to participate in HELIOS-A study if you are 18 to 85 years old and have been diagnosed with hATTR amyloidosis. Other eligibility criteria apply.
Use our site location finder to see the closest participating site location near you and take a questionnaire to determine if you may qualify to participate.
The main goal of the HELIOS-A research study is to evaluate the safety and effectiveness of the investigational medication, ALN-TTRSC02, for the treatment of neurological symptoms caused by hereditary ATTR amyloidosis (hATTR amyloidosis). The study will also evaluate any changes in quality of life experienced by study participants.
75% of participants will receive ALN-TTRSC02 for the total duration of the study. The other 25% of participants will receive the study medication, patisiran, for the first 18 months and then receive ALN-TTRSC02 for the next 18 months in the study treatment extension period.
All study-related procedures and study medications will be provided to HELIOS-A participants at no cost. Transportation to and from study visits, or reimbursement for travel-related costs may also be provided to those who qualify. Some participants may also be able to receive treatment at home after a certain point in the study limiting the number of in-person physician visits.
Who Can Participate in the HELIOS-A Study?
Approximately 160 people will participate in HELIOS-A. You may be eligible to participate in HELIOS-A study if you:
- Are 18 to 85 years old
- Have been diagnosed with hATTR amyloidosis and have a confirmed TTR mutation
- Have not had a liver transplant
- Do not require a wheelchair for everyday mobility
- Do not have heart failure symptoms that significantly limit your physical activity
- Have not received TTR-lowering treatment or participated in a gene therapy study for hATTR amyloidosis in the past
- Have not been diagnosed with type 2 diabetes 5 or more years ago
Additional criteria will be assessed by the study team to determine eligibility.
View study locations & find out if you qualify
FREQUENTLY ASKED QUESTIONS
Before a medicine can become available to the public, it must be tested in a series of clinical trials to evaluate safety and effectiveness. This is a requirement of government regulated health authorities. Health authorities use the information from clinical trials to decide whether investigational medicines should be approved for routine medical use. Every medication goes through the clinical trial process, therefore, participants like you play a very important role in advancing medicine for present and future generations.
The main goal of this clinical research study is to determine if the investigational medication, ALN-TTRSC02, is safe and effective at improving the neurological symptoms that people with hereditary ATTR amyloidosis (hATTR amyloidosis) experience. The study will also evaluate any changes in quality of life experienced by study participants.
The symptoms of hereditary ATTR amyloidosis (hATTR amyloidosis) are caused by a build-up of the misfolded TTR protein in certain parts of the body. It is thought that if the production of the TTR protein can be reduced, then this may reduce neurological symptoms or slow down the progression of hATTR amyloidosis.
ALN-TTRSC02 is an investigational medication that, in a previous clinical study of healthy volunteers, has been shown to reduce production of TTR protein. In the HELIOS-A study, ALN-TTRSC02 is being studied to determine if using this medication to reduce the production of TTR protein in adults with hATTR amyloidosis will result in improvement in neurological symptoms and quality of life.
- ALN-TTRSC02 is given once every 3 months (quarterly) as a subcutaneous injection in the abdomen, arm, or thigh. The injection goes into the fatty tissue just under the skin. It does not go into the muscle and does not require access to a vein. It is not approved by health authorities in the United States or any other country for the treatment of hATTR amyloidosis.
- Patisiran is administered intravenously (directly into a vein) once every 3 weeks. Patisiran is approved in the United States and the European Union for the treatment of polyneuropathy caused by hATTR amyloidosis in adults.
For the first 18 months of the trial, HELIOS-A participants will be randomly (by chance) assigned to 1 of 2 study treatment groups:
- ALN-TTRSC02: There is an 75% chance of being assigned to this group (120 people)
- Patisiran: There is a 25% chance of being assigned to this group (40 people)
HELIOS-A is an open-label study, which means each participant and his / her study team will know which study treatment group they are in. Because the group is selected randomly, neither the participant or the study team will be able to choose the treatment group as this is done by a computer.
At month 19, all participants will receive ALN-TTRSC02 for the remainder of their time in the treatment period of the study, which is expected to last for up to 18 months.
After receiving their last dose of ALN-TTRSC02, participants will enter the follow-up period for up to 1 year.
No. No one in this study will receive placebo. A placebo looks like the study medication but does not contain any active drug. All participants in this study will receive one of the active study drugs.
Approximately 160 people will participate in HELIOS-A globally. Each participant will be in the study for a approximately 4 years. There are 4 study periods:
- Screening Period: Up to 6 weeks
- Treatment Period: 1.5 years (18 months)
- 75% of participants (120 people) will receive ALN-TTRSC02 every 3 months via subcutaneous injection
- 25% of participants (40 people) will receive patisiran every 3 weeks via intravenous infusion
- Treatment Extension Period: 1.5 years (18 months)
- Follow-up Period: Up to 1 year after last dose of ALN-TTRSC02
Starting at study month 9, most visits may take place at home with a visiting nurse if preferred.
There are no guaranteed benefits to taking part in any clinical trial. Some reasons people participate in clinical trials include:
- Gaining access to investigational medications that are not yet available to the public
- Having their health monitored more closely
- Helping others with the same condition by participating in research on the disease
Risks involved in clinical trials may include:
- Side effects from the study medications
- The investigational medication not working
Before deciding to participate in this, or any clinical trial, the study team will explain the potential risks and benefits of participation.
Clinical study participation is completely voluntary. You do not need to take part in this study. If you do, you can end your participation at any time, for any reason. If you would like to stop participating in the study at any time, talk to the study doctor. Participants who leave the study early will be expected to complete a follow-up period for up to a year to ensure their health and safety.
All study-related procedures and study medications will be provided to HELIOS-A participants at no cost. Transportation to and from study visits, or reimbursement for travel-related costs may also be provided for eligible participants.
Thank you for your interest in the HELIOS-A study.
The study is not currently enrolling patients in the US, but to see global study locations please visit:
If you are interested in learning more about future study opportunities, please contact firstname.lastname@example.org.