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The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

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ClinicalTrials.gov Identifier: NCT02658175
Recruitment Status : Completed
First Posted : January 18, 2016
Last Update Posted : December 24, 2020
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE January 12, 2016
First Posted Date  ICMJE January 18, 2016
Last Update Posted Date December 24, 2020
Actual Study Start Date  ICMJE December 31, 2015
Actual Primary Completion Date January 15, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 4, 2017)
Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [ Time Frame: 117 Weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 15, 2016)
Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [ Time Frame: 52 Weeks ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
Official Title  ICMJE ISIS 304801-CS7 The APPROACH Open Label Study Volanesorsen (ISIS 304801) An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Brief Summary An Open-Label Study of Volanesorsen (IONIS 304801) Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
Detailed Description This is a multi-center, open-label study for Group 1: ISIS 304801-CS6 (index study) roll-over FCS patients, Group 2: ISIS 304801-CS16 (index study) roll-over FCS patients, and Group 3: FCS patients who did not participate in the ISIS 304801-CS6 or ISIS 304801-CS16 index studies. All patients will receive volanesorsen 300 mg once per week for 52 weeks. Patients will have the option of continuing dosing for an additional 52 weeks until an expanded access program is approved and available in their country.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Familial Chylomicronemia Syndrome
  • Lipoprotein Lipase Deficiency
  • Hyperlipoproteinemia Type 1
Intervention  ICMJE Drug: Volanesorsen
300 mg volanesorsen
Other Names:
  • IONIS-APOCIIIRx
  • ISIS 304801
Study Arms  ICMJE Experimental: Volanesorsen
Intervention: Drug: Volanesorsen
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: February 13, 2018)
69
Original Estimated Enrollment  ICMJE
 (submitted: January 15, 2016)
60
Actual Study Completion Date  ICMJE January 15, 2020
Actual Primary Completion Date January 15, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Must give written informed consent to participate in the study (signed and dated) and any authorization required by law
  • Able and willing to participate in a 65-week study

Group 1 and 2:

  • Satisfactory completion of ISIS 304801-CS6 or ISIS 304801-CS16 (index studies) with an acceptable safety profile, per Sponsor and Investigator judgment

Group 3:

  • Patients who did not participate in the CS6 or CS16 index studies and meet additional inclusion criteria of Familial Chylomicronemia Syndrome (FCS) may enroll in the study.
  • History of chylomicronemia
  • A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
  • Fasting triglycerides ≥ 750 mg/dL (8.4mmol/L) at Screening

Exclusion Criteria:

  • Unwilling to comply with lifestyle requirements for the duration of the study

Group 1 and 2:

  • Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study.

Group 3:

  • Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
  • Active pancreatitis within 4 weeks of screening
  • Acute Coronary Syndrome within 6 months of screening
  • Major surgery within 3 months of screening
  • Treatment with Glybera therapy within 2 years of screening
  • Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil,   Canada,   France,   Germany,   Israel,   Italy,   Netherlands,   South Africa,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02658175
Other Study ID Numbers  ICMJE ISIS 304801-CS7
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ionis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Ionis Pharmaceuticals, Inc.
Collaborators  ICMJE Akcea Therapeutics
Investigators  ICMJE Not Provided
PRS Account Ionis Pharmaceuticals, Inc.
Verification Date December 2020

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