A phase 3 study to evaluate the efficacy and safety of ARO-APOC3 in adults with familial chylomicronemia syndrome (FCS)

Arrowhead Pharmaceuticals, Inc.0 sites76 target enrollmentApril 6, 2022

ConditionsFamilial Chylomicronemia Syndrome Genetic Diseases

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Familial Chylomicronemia Syndrome
Sponsor: Arrowhead Pharmaceuticals, Inc.
Enrollment: 76
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

April 6, 2022

End Date

May 3, 2026

Last Updated

last year

Study Type

Interventional

Sex

All

Investigators

Sponsor

Arrowhead Pharmaceuticals, Inc.

Eligibility Criteria

Inclusion Criteria

•1\. Males or nonpregnant (who do not plan to become pregnant), nonlactating females \=18 years of age
•2\. Able and willing to provide written informed consent prior to the performance of any study specific procedures
•3\. Fasting TG \=10 mmol/L (\~880 mg/dL) at screening, that are refractory to standard lipid lowering therapy (sample drawn after at least the minimum time on stable lipid lowering regimen described in the protocol). Two repeat tests are allowed to qualify.
•4\. A diagnosis of FCS based on a documented history of fasting TG levels in excess of 1000 mg/dL on repeated testing (for at least 3 occasions), and at least one of the following:
•4\.1\. A supportive genetic test (from a source\-verifiable medical record or based on screening genotype). Supportive genetic testing includes but is not limited to homozygous, compound heterozygous, or double heterozygote for loss of function or otherwise inactivating mutations in genes affecting lipoprotein lipase activity including LPL, APOC2, APOA5, GPIHBP1, GPD1, or LMF1; or evidence of low LPL activity (\<20% of normal) based on source verifiable documentation; or
•4\.2\. Documented history of recurrent episodes of acute pancreatitis, not caused by alcohol or cholelithiasis; or
•4\.3\. Documented history of recurrent hospitalizations for severe abdominal pain without other explainable cause; or
•4\.4\. Documented history of childhood pancreatitis; or
•4\.5\. Family history of hypertriglyceridemia\-induced pancreatitis
•5\. Willing to follow dietary counseling as per PI judgment based on local standard of care, consistent with an intake of \= 20 g of fat per day during the study

Exclusion Criteria

•1\. Current use or use within the last 365 days from Day 1 of any hepatocyte\-targeted siRNA or antisense oligonucleotide molecule
•2\. Diabetes mellitus with any of the following:
•a. Newly diagnosed within 12 weeks of screening
•b. HbA1c \=9\.0% at screening
•3\. Active pancreatitis within 12 weeks before Day 1
•4\. History of acute coronary syndrome event within 24 weeks of Day 1
•5\. History of major surgery within 12 weeks of Day 1
•6\. Any of the following laboratory values at screening:
•6\.1\. ALT or AST \=3×ULN at screening
•6\.2\. Total bilirubin \=1\.5 ULN (if the participant has a prior diagnosis and documentation of Gilbert’s syndrome, then total bilirubin must be \=3 mg/dL at screening)