Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03783377
Recruitment Status : Recruiting
First Posted : December 21, 2018
Last Update Posted : September 13, 2019
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

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).

Condition or disease Intervention/treatment Phase
Hypertriglyceridemia Familial Chylomicronemia Drug: ARO-APOC3 Drug: sterile normal saline (0.9% NaCl) Phase 1

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 8, 2019
Estimated Primary Completion Date : June 1, 2020
Estimated Study Completion Date : June 1, 2020


Arm Intervention/treatment
Experimental: ARO-APOC3 Drug: ARO-APOC3
single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections

Placebo Comparator: Placebo Drug: sterile normal saline (0.9% NaCl)
calculated volume to match active treatment




Primary Outcome Measures :
  1. Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment [ Time Frame: Up to Day 113 (+/- 3 days) ]

Secondary Outcome Measures :
  1. Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  2. PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  3. PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  4. PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  5. PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  6. Reduction in Fasting Serum APOC3 from Pre-Dose Baseline [ Time Frame: Up to Day 113 (+/- 3 days) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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 a history of fasting triglycerides >/= 500 mg/dL (>/= 5.65 mmol/L) and fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03783377


Contacts
Layout table for location contacts
Contact: Medical Monitor +1-626-304-3400 clinicaltrials@arrowheadpharma.com

Locations
Layout table for location information
Australia, New South Wales
Royal Prince Alfred Hospital Recruiting
Camperdown, New South Wales, Australia, 2050
Contact: Kerry Kearins    +61 2 95157236    Kerry.Kearins@health.nsw.gov.au   
Principal Investigator: David Sullivan, MD         
Australia, Queensland
QPharm Withdrawn
Herston, Queensland, Australia, 4006
Australia, South Australia
Royal Adelaide Hospital Not yet recruiting
Adelaide, South Australia, Australia, 5000
Contact: Denise Healy    +61 409877807    Deanise.Healy@sa.gov.au   
Principal Investigator: Peter Clifton, MD         
New Zealand
Auckland Clinical Studies Limited Recruiting
Grafton, Auckland, New Zealand, 1010
Contact: Olivia Thame    + 64 9 373 3474    Olivia.Thame@clinicalstudies.co.nz   
Principal Investigator: Christian Schwabe, MD         
Middlemore Hospital Recruiting
Papatoetoe, Auckland, New Zealand, 2025
Contact: Alice Cassidy    +64 9 276 0044    Alice.Cassidy@mmclintrials.nz   
Principal Investigator: John Baker, MD         
Lipid & Diabetes Research Group Recruiting
Christchurch, New Zealand, 8011
Contact: Patrice McGregor    +64 3 364 1376    Patrice.McGregor@cdhb.health.nz   
Principal Investigator: Russell Scott, MD         
Sponsors and Collaborators
Arrowhead Pharmaceuticals

Layout table for additonal information
Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03783377     History of Changes
Other Study ID Numbers: AROAPOC31001
First Posted: December 21, 2018    Key Record Dates
Last Update Posted: September 13, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
Layout table for MeSH terms
Hyperlipoproteinemia Type I
Hypertriglyceridemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias