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

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ClinicalTrials.gov Identifier: NCT03783377
Recruitment Status : Not yet recruiting
First Posted : December 21, 2018
Last Update Posted : December 21, 2018
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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 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
Estimated Study Start Date : February 15, 2019
Estimated Primary Completion Date : March 1, 2020
Estimated Study Completion Date : March 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) ]


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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 fasting triglycerides at screening >/= 500 mg/dL 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

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
Contact: Medical Monitor +1-626-304-3400 clinicaltrials@arrowheadpharma.com

Sponsors and Collaborators
Arrowhead Pharmaceuticals

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: December 21, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
Hypertriglyceridemia
Hyperlipoproteinemia Type I
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias