The BROADEN Study: A Study of Volanesorsen (Formerly ISIS-APOCIIIRx) in Patients With Familial Partial Lipodystrophy

Overview

  • Study type

    Interventional
  • Study phase

    II/III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 15-004679
    NCT ID: NCT02527343
    Sponsor Protocol Number: ISIS 304801-CS17

About this study

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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility 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

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Vinaya Simha, M.D., M.B.B.S.

Closed for enrollment

Contact information:

Tamera Roberson

(507)255-8621

Roberson.Tamera@mayo.edu