Study Stopped
Study withdrawn due to lack of available patients meeting entry criteria
Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)
1 other identifier
interventional
N/A
1 country
1
Brief Summary
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
Started Apr 2018
Shorter than P25 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 22, 2018
CompletedFirst Posted
Study publicly available on registry
March 7, 2018
CompletedStudy Start
First participant enrolled
April 12, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
December 31, 2018
CompletedDecember 3, 2018
November 1, 2018
6 months
February 22, 2018
November 29, 2018
Conditions
Outcome Measures
Primary Outcomes (1)
Reduction of low density lipoprotein cholesterol (LDL-C).
Percent change in LDL-C from Baseline to week 14.
14 weeks
Secondary Outcomes (4)
Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).
7 and 14 Weeks
Effect of ISIS 703802 on lipid parameters.
7 and 14 Weeks
Evaluate plasma trough levels of ISIS 703802.
14 Weeks
The safety of ISIS 703802 by the incidence of treatment-emergent adverse events
14 Weeks
Study Arms (1)
AKCEA-ANGPTL3-LRX Dose 1
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- Body mass index (BMI) ≤ 35 kg/m2,
- Genetic confirmation of two mutant alleles at the LDLR, APOB, PCSK9, or LDLRAP1 gene locus OR an untreated LDL-C \> 500 mg/dL (13 mmol/L) or treated LDL-C ≥ 300 mg/dL (2.59 mmol/L) together with either cutaneous or tendon xanthoma before age 10 years OR familial medical history of genetically confirmed heterozygous FH in both parents OR untreated elevated LDL-C and TC \> 250 mg/dL consistent with the disease,
- Patients must be on stable LDL-C lowering agents or on regular apheresis
You may not qualify if:
- Myocardial infarction, percutaneous transluminal coronary intervention, or coronary artery bypass graft surgery within 12 weeks prior to Screening, or cerebrovascular accident within 24 weeks prior to Screening.
- Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Akcea Therapeuticslead
- Ionis Pharmaceuticals, Inc.collaborator
Study Sites (1)
Clinical Site
Québec, Quebec, G1V4W2, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 22, 2018
First Posted
March 7, 2018
Study Start
April 12, 2018
Primary Completion
September 30, 2018
Study Completion
December 31, 2018
Last Updated
December 3, 2018
Record last verified: 2018-11