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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03783377




Registration number
NCT03783377
Ethics application status
Date submitted
19/12/2018
Date registered
21/12/2018
Date last updated
1/08/2019

Titles & IDs
Public title
Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)
Scientific title
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome
Secondary ID [1] 0 0
AROAPOC31001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypertriglyceridemia 0 0
Familial Chylomicronemia 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ARO-APOC3
Treatment: Drugs - sterile normal saline (0.9% NaCl)

Experimental: ARO-APOC3 -

Placebo Comparator: Placebo -


Treatment: Drugs: ARO-APOC3
single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections

Treatment: Drugs: sterile normal saline (0.9% NaCl)
calculated volume to match active treatment

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
Timepoint [1] 0 0
Up to Day 113 (+/- 3 days)
Secondary outcome [1] 0 0
Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax)
Timepoint [1] 0 0
Single dose phase: Up to 48 hours post-dose
Secondary outcome [2] 0 0
PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax)
Timepoint [2] 0 0
Single dose phase: Up to 48 hours post-dose
Secondary outcome [3] 0 0
PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2)
Timepoint [3] 0 0
Single dose phase: Up to 48 hours post-dose
Secondary outcome [4] 0 0
PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)
Timepoint [4] 0 0
Single dose phase: Up to 48 hours post-dose
Secondary outcome [5] 0 0
PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf)
Timepoint [5] 0 0
Single dose phase: Up to 48 hours post-dose
Secondary outcome [6] 0 0
Reduction in Fasting Serum APOC3 from Pre-Dose Baseline
Timepoint [6] 0 0
Up to Day 113 (+/- 3 days)

Eligibility
Key inclusion criteria
- Women of childbearing 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

- Normal electrocardiogram (ECG) at screening

- Hypertriglyceridemic patients must have fasting triglycerides at screening >/= 500
mg/dL or verifiable diagnosis of FCS
Minimum age
18 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key 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

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
QPharm - Herston
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
4006 - Herston
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland
Country [2] 0 0
New Zealand
State/province [2] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Arrowhead Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and
pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and
in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
Trial website
https://clinicaltrials.gov/show/NCT03783377
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Monitor
Address 0 0
Country 0 0
Phone 0 0
+1-626-304-3400
Fax 0 0
Email 0 0
clinicaltrials@arrowheadpharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03783377