Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-15

Study Completion Date

2019-08-21

Brief Summary

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This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of fasting triglycerides in participants with familial partial lipodystrophy.

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Detailed Description

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Conditions

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Familial Partial Lipodystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AKCEA-ANGPTL3-LRx 20 mg

Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection.

Group Type EXPERIMENTAL

AKCEA-ANGPTL3-LRx

Intervention Type DRUG

AKCEA-ANGPTL3-LRx solution for SC injection.

Interventions

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AKCEA-ANGPTL3-LRx

AKCEA-ANGPTL3-LRx solution for SC injection.

Intervention Type DRUG

Other Intervention Names

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ISIS 703802

Eligibility Criteria

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Inclusion Criteria

* Must give written informed consent to participate in the study.
* Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
* Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with hemoglobin A1c (HbA1c) ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol.
* Hypertriglyceridemia as defined by fasting triglycerides (TG) levels ≥ 500 milligrams per deciliter (mg/dL) at both Screening and Qualification visits. Participants with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 millimoles per liter \[mmol/L\]) to \< 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or family history criteria for study inclusion may be further screened and enrolled in the study.
* Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.

Exclusion Criteria

* Diagnosis of generalized lipodystrophy.
* Diagnosis of acquired partial lipodystrophy (APL).
* Acute pancreatitis within 4 weeks of Screening.
* Acute coronary syndrome within 6 months of Screening.
* Major surgery within 3 months of Screening.
* Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No