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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 : Completed
First Posted : December 21, 2018
Last Update Posted : March 3, 2021
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE December 19, 2018
First Posted Date  ICMJE December 21, 2018
Last Update Posted Date March 3, 2021
Actual Study Start Date  ICMJE March 8, 2019
Actual Primary Completion Date February 11, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 19, 2018)
Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment [ Time Frame: Up to Day 113 (+/- 3 days) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 19, 2018)
  • 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 ]
  • PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  • PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
  • 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 ]
  • 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 ]
  • Reduction in Fasting Serum APOC3 from Pre-Dose Baseline [ Time Frame: Up to Day 113 (+/- 3 days) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Hypertriglyceridemia
  • Familial Chylomicronemia
Intervention  ICMJE
  • Drug: ARO-APOC3
    single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections
  • Drug: sterile normal saline (0.9% NaCl)
    calculated volume to match active treatment
Study Arms  ICMJE
  • Experimental: ARO-APOC3
    Intervention: Drug: ARO-APOC3
  • Placebo Comparator: Placebo
    Intervention: Drug: sterile normal saline (0.9% NaCl)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 2, 2021)
112
Original Estimated Enrollment  ICMJE
 (submitted: December 19, 2018)
63
Actual Study Completion Date  ICMJE February 11, 2021
Actual Primary Completion Date February 11, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 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

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   New Zealand
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03783377
Other Study ID Numbers  ICMJE AROAPOC31001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Arrowhead Pharmaceuticals
Study Sponsor  ICMJE Arrowhead Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Arrowhead Pharmaceuticals
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP