CompletedPhase 3

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

United States, Argentina, Australia225 participantsStarted 2013-11

Plain-language summary

The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis. An open-label, single-arm, long-term follow-up extension study NCT02510261 (ALN-TTR02-006) was initiated to provide participants who completed this study with continued patisiran-LNP (lipid nanoparticle) treatment.

Who can participate

Age range

18 Years – 85 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female of 18 to 85 years of age (inclusive); * Have a diagnosis of FAP * Neuropathy Impairment Score requirement of 5-130 * Meet Karnofsky performance status requirements * Have adequate complete blood counts and liver function tests * Have adequate cardiac function * Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV) Exclusion Criteria: * Had a prior liver transplant or is planned to undergo liver transplant during the study period; * Has untreated hypo- or hyperthyroidism; * Has known human immunodeficiency virus (HIV) infection; * Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated; * Recently received an investigational agent or device * Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Modified Neuropathy Impairment Score +7 (mNIS+7)

Timeframe: 18mo

Trial details

NCT IDNCT01960348

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-08

Results submitted2018-08-07

View on ClinicalTrials.gov More TTR-mediated Amyloidosis trials