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HELIOS-A: A Phase 3 Global Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis
EU CTIS ID: 2023-508365-33-00
What this study is testing
To determine the efficacy of ALN-TTRSC02 in patients with hATTR amyloidosis by evaluating the effect on neurologic impairment
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female age 18 (or age of legal consent, whichever is older) to 85 years of age
- Have a diagnosis of hATTR amyloidosis with documented TTR mutation
- Have a Neuropathy Impairment Score of 5 to 130 (inclusive; this criterion must be met at the Screening Visit 2)
- Have a Polyneuropathy Disability score of ≤3b (this criterion must be met at the Screening Visit 2)
- Have a Karnofsky Performance Status (KPS) of ≥60%
- Patient is willing and able to comply with the study requirements and to provide written informed consent
You likely can't join if
- Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the 18-month Treatment Period of the study
- Is currently taking diflunisal; if previously on this agent, must have at least a 3-day washout prior to dosing (Day 1)
- Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis
- Has a New York Heart Association heart failure classification >2
- Has any of the following laboratory parameter assessments: a. ALT and/or AST >1.5× upper limit of normal reference range (ULN); b. Total bilirubin >ULN (>1.5 ULN in patients with Gilbert's Syndrome) c. INR >1.2 (patients on anticoagulant therapy with an INR of ≤3.5 will be allowed)
- Estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m2
See the full eligibility criteria
- Male or female age 18 (or age of legal consent, whichever is older) to 85 years of age
- Have a diagnosis of hATTR amyloidosis with documented TTR mutation
- Have a Neuropathy Impairment Score of 5 to 130 (inclusive; this criterion must be met at the Screening Visit 2)
- Have a Polyneuropathy Disability score of ≤3b (this criterion must be met at the Screening Visit 2)
- Have a Karnofsky Performance Status (KPS) of ≥60%
- Patient is willing and able to comply with the study requirements and to provide written informed consent
- Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the 18-month Treatment Period of the study
- Is currently taking diflunisal; if previously on this agent, must have at least a 3-day washout prior to dosing (Day 1)
- Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis
- Has a New York Heart Association heart failure classification >2
- Has any of the following laboratory parameter assessments: a. ALT and/or AST >1.5× upper limit of normal reference range (ULN); b. Total bilirubin >ULN (>1.5 ULN in patients with Gilbert's Syndrome) c. INR >1.2 (patients on anticoagulant therapy with an INR of ≤3.5 will be allowed)
- Estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m2
- Has known human immunodeficiency virus (HIV) infection; or evidence of acute or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection
- Has other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease, monoclonal gammopathy) that the treating physician believes to be contributing to the neuropathy
- Current or future participation in another investigational device or drug study
- Is currently taking tafamidis, doxycycline, or tauroursodeoxycholic acid; acid; if previously on any of these agents, must have completed a 14-day wash-out prior to dosing (Day 1)
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Japan
- Mexico
- Canada
- Australia
- Argentina
- Taiwan
- Turkey
- Malaysia
- Korea, Democratic People's Republic of
- Brazil
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Japan; Mexico; Canada; Australia; Argentina and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.