Clinical trial NCT03747224: Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients
Official title: A Phase 1 Single and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-ANG3 in Adult Healthy Volunteers and in Dyslipidemic Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Dyslipidemias
Familial Hypercholesterolemia
Hypertriglyceridemia
Lead sponsor: Arrowhead Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-11-20
Enrollment: 93
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.


Eligibility criteria: Inclusion Criteria:

* Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
* Willing to provide written informed consent and to comply with study requirements
* On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
* Normal electrocardiogram (ECG) at Screening

Exclusion Criteria:

* Clinically significant health concerns
* Regular use of alcohol within one month prior to Screening
* Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study
* Recent use of illicit drugs
* Use of more than two tobacco/nicotine containing or cannabis products per month within 6 months prior to drug administration (applicable only to Normal Healthy Volunteers)

NOTE: additional inclusion/exclusion criteria may apply, per protocol

Primary outcomes:
measure: Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
timeFrame: Up to 113 (+/- 3 days) post-dose
Secondary outcomes:
measure: Pharmacokinetics (PK) of ARO-ANG3: Maximum Observed Plasma Concentration (Cmax)
timeFrame: Single dose phase: Up to 48 hours post-dose
measure: PK of ARO-ANG3: Time to Maximum Plasma Concentration (Tmax)
timeFrame: Single dose phase: Up to 48 hours post-dose
measure: PK of ARO-ANG3: Terminal Elimination Half-Life (t1/2)
timeFrame: Single dose phase: Up to 48 hours post-dose
measure: PK of ARO-ANG3: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)
timeFrame: Single dose phase: Up to 48 hours post-dose
measure: PK of ARO-ANG3: Area Under the Plasma Concentration Versus Time Curve From Zero to infinity (AUCinf)
timeFrame: Single dose phase: Up to 48 hours post-dose
measure: Reduction in Fasting Serum ANGPTL3 from Pre-Dose Baseline
timeFrame: Baseline, Up to Day 113 (+/- 3 days)
Other outcomes:
Locations:
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Nedlands
country: Australia
facility: Linear Clinical Research
city: Grafton
country: New Zealand
facility: Auckland Clinical Studies Limited
state: Auckland
city: Papatoetoe
country: New Zealand
facility: Middlemore Hospital
state: Aukland
city: Christchurch
country: New Zealand
facility: Lipid & Diabetes Research Group
Study officials:
References:
citation: Ying Q, Chan DC, Watts GF. Angiopoietin-like protein 3 inhibitors and contemporary unmet needs in lipid management. Curr Opin Lipidol. 2021 Jun 1;32(3):210-212. doi: 10.1097/MOL.0000000000000747. No abstract available.