Volanesorsen Early Access Program for Patients With Familial

Clinical Trial Description

The Program is intended to provide expanded access to volanesorsen for eligible patients with FCS who have limited or no available treatment options. ;

Study Design

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Completed	`NCT03783377` - Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)	Phase 1
Active, not recruiting	`NCT05089084` - Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)	Phase 3

See also

Status

Clinical Trial

Phase

Recruiting

NCT05687474 - Baby Detect : Genomic Newborn Screening

Completed

NCT03783377 - Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)

Phase 1

Active, not recruiting

NCT05089084 - Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

Phase 3

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT03544060
Study type	Expanded Access
Source	Akcea Therapeutics
Contact
Status	No longer available
Phase

Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms