NCT03514420

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2018

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 21, 2018

Completed
11 days until next milestone

First Posted

Study publicly available on registry

May 2, 2018

Completed
1 month until next milestone

Study Start

First participant enrolled

June 15, 2018

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 30, 2019

Completed
22 days until next milestone

Study Completion

Last participant's last visit for all outcomes

August 21, 2019

Completed
1.5 years until next milestone

Results Posted

Study results publicly available

February 16, 2021

Completed
Last Updated

February 16, 2021

Status Verified

January 1, 2021

Enrollment Period

1.1 years

First QC Date

April 21, 2018

Results QC Date

January 6, 2021

Last Update Submit

January 27, 2021

Conditions

Familial Partial Lipodystrophy

Keywords

LipodystrophyLipodystrophy, Familial PartialLipid Metabolism DisordersDyslipidemiasKobberling-Dunnigan syndrome (type 1 and 2)Lipoatrophic Diabetes

Outcome Measures

Primary Outcomes (1)

  • Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)

    The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.

    Baseline and End of the Treatment (Week 27)

Secondary Outcomes (42)

  • Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment

    Baseline and End of the Treatment (Week 27)

  • Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment

    Baseline and End of the Treatment (Week 27)

  • Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment

    Baseline and End of the Treatment (Week 27)

  • Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment

    Baseline and End of the Treatment (Week 27)

  • Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment

    Baseline and End of the Treatment (Week 27)

  • +37 more secondary outcomes

Study Arms (1)

AKCEA-ANGPTL3-LRx 20 mg

EXPERIMENTAL

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

Drug: AKCEA-ANGPTL3-LRx

Interventions

AKCEA-ANGPTL3-LRxDRUG

AKCEA-ANGPTL3-LRx solution for SC injection.

Also known as: ISIS 703802
AKCEA-ANGPTL3-LRx 20 mg

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.
  • Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Site

Ann Arbor, Michigan, 48105, United States

Location

MeSH Terms

Conditions

Lipodystrophy, Familial PartialLipodystrophyLipid Metabolism DisordersDyslipidemiasDiabetes Mellitus, Lipoatrophic

Condition Hierarchy (Ancestors)

LaminopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, MetabolicSkin DiseasesSkin and Connective Tissue DiseasesLipid Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesDiabetes Mellitus, Type 2Diabetes MellitusGlucose Metabolism DisordersEndocrine System Diseases

Results Point of Contact

Title
Study Director
Organization
Akcea Therapeutics
clinicalstudies@akceatx.com
617-207-0289

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 21, 2018

First Posted

May 2, 2018

Study Start

June 15, 2018

Primary Completion

July 30, 2019

Study Completion

August 21, 2019

Last Updated

February 16, 2021

Results First Posted

February 16, 2021

Record last verified: 2021-01

Data Sharing

IPD Sharing
Will not share

Locations

US(1)