Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03514420
• Date of registration: 21/04/2018
• Prospective Registration: Yes
• Primary sponsor: Akcea Therapeutics
• Public title: Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)
• Scientific title: An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy
• Date of first enrolment: May 30, 2018
• Target sample size: 4
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03514420
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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
HbA1c = 7% to = 12% at Screening and on anti-diabetic therapy as defined in study
protocol

- Hypertriglyceridemia as defined by fasting TG levels = 500 mg/dL at both Screening and
Qualification visits. Patients with the clinical diagnosis of FPL and with fasting TG
levels = 200 (= 2.26 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 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 patient participating in or completing the study



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Familial Partial Lipodystrophy

Intervention(s)

Drug: AKCEA-ANGPTL3-LRX

Primary Outcome(s)

Reduction of fasting triglycerides (TG). [Time Frame: 27 weeks]

Secondary Outcome(s)

Effect of ISIS 703802 on lipid parameters. [Time Frame: 27 weeks]

The safety of ISIS 703802 by the incidence of treatment-emergent adverse events. [Time Frame: 27 weeks]

Effect of ISIS 703802 on fat distribution in various areas of the body. [Time Frame: 27 weeks]

Effect of ISIS 703802 on mixed meal test (MMT). [Time Frame: 27 weeks]

Effect of ISIS 703802 on hepatic fat fraction. [Time Frame: 27 weeks]

Effect of ISIS 703802 on glycosylated hemoglobin (HbA1c). [Time Frame: 27 weeks]

Secondary ID(s)

ISIS 703802-CS5

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ionis Pharmaceuticals, Inc.