Alnylam Pharmaceuticals, Inc is currently recruiting participants for the APOLLO study, an international clinical research study designed to evaluate the effectiveness of patisiran in the treatment of transthyretin-mediated amyloidosis.
The purpose of this clinical research study is to evaluate the efficacy and safety of the investigational drug patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis (also known as Familial Amyloidotic Polyneuropathy or FAP).
FAP is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for retinol binding protein. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.
What does the study involve?
This is a multicenter, multinational, randomized, double-blind study comparing patisiran (ALN-TTR02) to placebo in ATTR patients with symptomatic Familial Amyloidotic Polyneuropathy (FAP).
Consented eligible patients will be randomized to receive either patisiran or placebo in a 2:1 ratio (patisiran to placebo) every 21 days. Patients who complete the 18-month efficacy assessments can elect to participate in an extension study in which patients would receive open-label administration of patisiran.
Ages Eligible for Study: 18 years to 85 years
Genders Eligible for Study: Both
Accepts Healthy Volunteers: No
– Male or female of 18 to 85 years of age (inclusive);
– Have a diagnosis of FAP;
– Meet Neuropathy Impairment Score requirements and 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)
– 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.
You can find more information about the study and where Alnylam is currently recruiting patients published on the US website www.clinicaltrials.gov.
Clinical research studies are generally considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol. Participating in a clinical research study allows you to be proactive in the management of your health, have access to new research treatments before they become available to the general public, and help others by contributing to medical research. To learn more, visit www.clinicaltrials.gov, type in “neuropathy” and click “search.” Be sure to also talk with your doctor.