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Summary

for people ages 18–65 (full criteria)
at San Francisco, California and other locations
study started

Description

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.

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

Keywords

Familial Partial Lipodystrophy Lipodystrophy Lipodystrophy, Familial Partial

Eligibility

You can join if…

Open to people ages 18–65

  • Clinical diagnosis of FPL plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
  • Diagnosis of FLP 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:
  • Genetic diagnosis of familial PL (e.g., mutations in LMNA, PPAR-γ, AKT2, CIDEC, PLIN1 genes)

OR

-Family history of FPL or family history of abnormal and similar fat distribution plus 1 Minor Criteria

OR

-2 Minor Criteria (In the absence of FPL-associated genetic variant or family history) and BMI< 35 kg/m2

Minor Criteria

  1. Requirement for high doses of insulin, e.g., requiring ≥ 200 U/day, ≥ 2 U/kg/day, or currently taking U-500 insulin
  2. Presence of acanthosis nigricans on physical examination
  3. Evidence/history of polycystic ovary syndrome (PCOS) or PCOS-like symptoms (hirsutism,oligomenorrhea, and/or polycystic ovaries)
  4. History of pancreatitis associated with hypertriglyceridemia
  5. Evidence of non-alcoholic fatty liver disease
  6. Hepatomegaly and/or elevated transaminases in the absence of a known cause of liver disease or radiographic evidence of hepatic steatosis (e.g., on ultrasound or CT)
  7. Diagnosis of Type 2 diabetes mellitus as defined by the American Diabetes Association Standards of Medical Care in Diabetes 2015) made at least 12 weeks prior to the Screening visit, and 2 of the following:
  8. on standard-of-care anti-diabetic therapy, and
  9. HbA1c ≥ 7% to ≤ 10% at Screening.
  10. Hypertriglyceridemia is defined as Fasting TG levels ≥ 500 mg/dL (≥ 5.7 mmol/L)at Screening and Qualification visit.
  11. If the fasting TG value at Screening and/or Qualification visit is < 500 mg/dL (< 5.7 mmol/L) but ≥ 350 mg/dL (≥ 4.0 mmol/L) up to 2 additional tests may be performed in order to qualify

You CAN'T join if...

  • 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 ischemia(myocardial infarction, acute coronary syndrome, new onset angina), stroke,transient ischemic attack, or unstable congestive heart failure requiring a change in medication
  • Major surgery within 3 months of Screening
  • Treatment with Metreleptin within the last 3 months prior to Screening
  • Previous treatment with ISIS-APOCIIIRx

Locations

  • IONIS Investigative Site accepting new patients
    San Francisco, California, 94143, United States
  • IONIS Investigative Site accepting new patients
    Aurora, Colorado, 80045, United States
  • IONIS Investigative Site accepting new patients
    Dallas, Texas, 75390, United States
  • IONIS Investigative Site accepting new patients
    Rochester, Minnesota, 55905, United States
  • IONIS Investigative Site accepting new patients
    Saint Louis, Missouri, 63110, United States
  • IONIS Investigative Site accepting new patients
    Ann Arbor, Michigan, 48105, United States
  • IONIS Investigative Site withdrawn
    London, Ontario, N6A 5B7, Canada
  • IONIS Investigative Site accepting new patients
    Morehead City, North Carolina, United States
  • IONIS Investigative Site accepting new patients
    Albany, New York, 12208, United States
  • IONIS Investigative Site withdrawn
    Chicoutimi, Quebec, G7H 7K9, Canada
  • IONIS Investigative Site accepting new patients
    Halifax, Nova Scotia, B3H 1C2, Canada
  • IONIS Investigative Site accepting new patients
    Moscow, 117036, Russian Federation
  • IONIS Investigative Site accepting new patients
    Cambridge, CB2 0QQ, United Kingdom
  • IONIS Investigative Site not yet accepting patients
    Somerset, BA21 4AT, United Kingdom
  • IONIS Investigative Site not yet accepting patients
    Brasilia, 71625009, Brazil
  • IONIS Investigative Site accepting new patients
    Rio de Janeiro, 20211-340, Brazil
  • IONIS Investigative Site not yet accepting patients
    Sao Paulo, 14049-900, Brazil
  • IONIS Investigative Site accepting new patients
    Amsterdam-Zuidoost, 1105 AZ, Netherlands
  • IONIS Investigative Site accepting new patients
    GA Nijmegen, 6525 HB, Netherlands
  • IONIS Investigative Site withdrawn
    Leiden, 2333 ZA, Netherlands
  • IONIS Investigative Site accepting new patients
    Leuven, 3000, Belgium
  • IONIS Investigative Site accepting new patients
    Porto, 4200-319, Portugal
  • IONIS Investigative Site accepting new patients
    Berlin, Germany
  • IONIS Investigative Site accepting new patients
    Koeln, 50937, Germany
  • IONIS Investigative Site accepting new patients
    Muenster, 48149, Germany
  • IONIS Investigative Site accepting new patients
    Santiago de Compostela, 15782, Spain
  • IONIS Investigative Site accepting new patients
    Sevilla, 41013, Spain
  • IONIS Investigative Site accepting new patients
    Bologna, 40138, Italy
  • IONIS Investigative Site accepting new patients
    Catania, 95123, Italy
  • IONIS Investigative Site accepting new patients
    Pisa, 56124, Italy
  • IONIS Investigative Site accepting new patients
    Athens, 12462, Greece
  • IONIS Investigative Site accepting new patients
    Ioannina, 45500, Greece
  • IONIS Investigative Site accepting new patients
    Izmir, 35340, Turkey
  • IONIS Investigative Site accepting new patients
    Haifa, Israel

Details

Status
accepting new patients
Start Date
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT02527343
Phase
Phase 2/3
Study Type
Interventional
Last Updated
July 10, 2017
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