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Trial record 1 of 4 for:    BROADEN | BROADEN
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The BROADEN Study: A Study of Volanesorsen (Formerly ISIS-APOCIIIRx) in Patients With Familial Partial Lipodystrophy

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02527343
Recruitment Status : Active, not recruiting
First Posted : August 19, 2015
Last Update Posted : January 10, 2020
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to evaluate the efficacy and safety of volanesorsen (IONIS-APOCIIIRx) given for 52 weeks in patients with Familial Partial Lipodystrophy. Patients will then be allowed to continue in a 2 year Open Label Extension of the study.

Condition or disease Intervention/treatment Phase
Familial Partial Lipodystrophy Drug: volanesorsen Drug: Placebo Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, With an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Partial Lipodystrophy
Actual Study Start Date : October 31, 2015
Actual Primary Completion Date : June 30, 2018
Estimated Study Completion Date : September 30, 2021


Arm Intervention/treatment
Active Comparator: volanesorsen 300mg
volanesorsen administered subcutaneously once weekly for 52 weeks
Drug: volanesorsen
Other Name: ISIS 304801, IONIS-APOCIIIRx

Placebo Comparator: Placebo
Placebo administered subcutaneously once weekly for 52 weeks.
Drug: Placebo
Active Comparator: Open Label Extension (OLE)
Volanesorsen administered subcutaneously once weekly for 104 weeks
Drug: volanesorsen
Other Name: ISIS 304801, IONIS-APOCIIIRx




Primary Outcome Measures :
  1. Efficacy of volanesorsen (IONIS-APOCIIIRx) measured as the percent change in fasting triglycerides from baseline. [ Time Frame: 13 weeks ]

Secondary Outcome Measures :
  1. Change from Baseline in hepatic steatosis (as assessed by hepatic fat fraction using magnetic resonance imaging (MRI). [ Time Frame: 52 weeks ]
  2. Change from Baseline in hemoglobin A1c (HbA1c) [ Time Frame: 52 weeks ]
  3. A composite endpoint at Month 6 for percent of patients who achieve a. ≥ 40% reduction in fasting TG, and b. ≥ 30% reduction of hepatic fat fraction percent [ Time Frame: 52 weeks ]
  4. Change in patient-reported outcomes (PRO) [ Time Frame: 52 weeks ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must give written informed consent to participate in the study (signed and dated) and any authorizations required by law
  • Clinical diagnosis of FPL plus diagnosis of type 2 diabetes mellitus, hypertriglyceridemia, and fatty liver

    • Diagnosis of FPL is based on deficiency of subcutaneous body fat in a partial fashion assessed by physical examination and low skinfold thickness in anterior thigh by caliper measurement: men (≤ 10 mm) and women (≤ 22 mm), and at least 1 of the following:

      1. Genetic diagnosis of FPL OR
      2. Family history of FPL or of similar abnormal fat distribution plus 1 Minor Criteria OR
      3. In the absence of FPL-associated genetic variant or family history, 2 Minor Criteria and BMI< 35 kg/m2
    • Diabetes not well controlled on antidiabetic therapy with HbA1c ≥ 7% to ≤ 12% at Screening
    • Hypertriglyceridemia with Fasting TG levels ≥ 500 mg/dL (≥ 5.7 mmol/L) at Screening and Qualification visit, or Fasting TG levels ≥ 200 (≥ 2.26 mmol/L) at both Screening and Qualification Visits for patients who meet the genetic or family history criteria
    • Presence of hepatosteatosis (fatty liver), as evidenced by a Screening MRI indicating a hepatic fat fraction (HFF) ≥ 6.4%.

Exclusion Criteria:

  • A diagnosis of generalized lipodystrophy
  • A diagnosis of acquired partial lipodystrophy
  • Acute pancreatitis within 4 weeks of Screening
  • History within 6 months of Screening of acute or unstable cardiac condition
  • LDL-C > 130 mg/dL on maximal tolerated statin therapy
  • Platelet count < lower limit of normal (LLN)
  • Treatment with metreleptin within the last 3 months prior to Screening

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): NCT02527343


Locations
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United States, Colorado
IONIS Investigative Site
Aurora, Colorado, United States, 80045
United States, Michigan
IONIS Investigative Site
Ann Arbor, Michigan, United States, 48105
United States, Minnesota
IONIS Investigative Site
Rochester, Minnesota, United States, 55905
United States, Missouri
IONIS Investigative Site
Saint Louis, Missouri, United States, 63110
United States, North Carolina
IONIS Investigative Site
Morehead City, North Carolina, United States
United States, Pennsylvania
IONIS Investigative Site
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
IONIS Investigative Site
Dallas, Texas, United States, 75390
Belgium
IONIS Investigative Site
Leuven, Belgium, 3000
Brazil
IONIS Investigative Site
Brasilia, Brazil, 71625009
IONIS Investigative Site
Rio de Janeiro, Brazil, 20211-340
IONIS Investigative Site
Sao Paulo, Brazil, 14049-900
Canada, Nova Scotia
IONIS Investigative Site
Halifax, Nova Scotia, Canada, B3H 1C2
Germany
IONIS Investigative Site
Berlin, Germany
IONIS Investigative Site
Koeln, Germany, 50937
IONIS Investigative Site
Muenster, Germany, 48149
Greece
IONIS Investigative Site
Athens, Greece, 12462
IONIS Investigative Site
Ioannina, Greece, 45500
Israel
IONIS Investigative Site
Haifa, Israel
Italy
IONIS Investigative Site
Bologna, Italy, 40138
IONIS Investigative Site
Catania, Italy, 95123
IONIS Investigative Site
Pisa, Italy, 56124
Netherlands
IONIS Investigative Site
Amsterdam-Zuidoost, Netherlands, 1105 AZ
Portugal
IONIS Investigative Site
Porto, Portugal, 4200-319
Russian Federation
IONIS Investigative Site
Moscow, Russian Federation, 117036
Spain
IONIS Investigative Site
Santiago de Compostela, Spain, 15782
IONIS Investigative Site
Sevilla, Spain, 41013
Turkey
IONIS Investigative Site
Izmir, Turkey, 35340
United Kingdom
IONIS Investigative Site
Cambridge, United Kingdom, CB2 0QQ
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02527343    
Other Study ID Numbers: ISIS 304801-CS17
First Posted: August 19, 2015    Key Record Dates
Last Update Posted: January 10, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Additional relevant MeSH terms:
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Lipodystrophy
Lipodystrophy, Familial Partial
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases