NCT00943306

Brief Summary

This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
19

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Oct 2009

Longer than P75 for phase_3

Geographic Reach
4 countries

10 active sites

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

July 21, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 22, 2009

Completed
3 months until next milestone

Study Start

First participant enrolled

October 29, 2009

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 17, 2012

Completed
2.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
3.4 years until next milestone

Results Posted

Study results publicly available

May 11, 2018

Completed
Last Updated

June 13, 2018

Status Verified

May 1, 2018

Enrollment Period

2.9 years

First QC Date

July 21, 2009

Results QC Date

February 29, 2016

Last Update Submit

May 14, 2018

Conditions

Familial Hypercholesterolemia

Keywords

HomozygousFamilialHypercholesterolemiaMTPApheresislomitapideHomozygous Familial Hypercholesterolemia (HoFH)

Outcome Measures

Primary Outcomes (1)

  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)

    Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

    Baseline and Week 126

Secondary Outcomes (53)

  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)

    Baseline and Week 174

  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)

    Baseline and Week 222

  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)

    Baseline and Week 246

  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)

    Baseline and Week 270

  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)

    Baseline and Week 294

  • +48 more secondary outcomes

Study Arms (1)

lomitapide

EXPERIMENTAL

Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

Drug: lomitapide

Interventions

lomitapideDRUG

5-60 mg po every day

Also known as: AEGR-733, BMS-201038
lomitapide

Eligibility Criteria

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

You may qualify if:

  • Completed UP1002 or 733-005.
  • Willing and able to provide consent and comply with the requirements of the study protocol.

You may not qualify if:

  • Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Robarts Research Institute

London, Ontario, N6A 5K8, Canada

Location

Lipid Clinic and University of Montreal Community Genomic Medicine Center

Chicoutimi, Quebec, G7H 5H6, Canada

Location

Medicina Interna Universitaria

Ferrara, Sicily, Italy

Location

Dipartimento di Medicina Clinica e Delle Patologie Emergenti

Palermo, Sicily, Italy

Location

Centro Universitario Dislipidemie

Milan, Italy

Location

DAI Ematologia, Oncologia, Anatomia Patologica e Medicina

Roma, Italy

Location

Cardiology Research

Bloemfontein, 9300, South Africa

Location

University of Capetown

Cape Town, 7925, South Africa

Location

Related Publications (3)

  • Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med. 2007 Jan 11;356(2):148-56. doi: 10.1056/NEJMoa061189.

    PMID: 17215532BACKGROUND

  • Cuchel M, Meagher E, Marais AD, et.al. Abstract 1077: A phase III study of microsomal triglyceride transfer protein inhibitor lomitapide (AEGR-733) in patients with homozygous familial hypercholesterolemia: interim results at 6 months. Circulation, Nov 2009; 120: S441

    BACKGROUND

  • Larrey D, D'Erasmo L, O'Brien S, Arca M; Italian Working Group on Lomitapide. Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. Liver Int. 2023 Feb;43(2):413-423. doi: 10.1111/liv.15497. Epub 2022 Dec 30.

    PMID: 36520008DERIVED

MeSH Terms

Conditions

Hyperlipoproteinemia Type IIHypercholesterolemiaHomozygous Familial Hypercholesterolemia

Interventions

BMS201038

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Alison Long, MD - VP Clinical
Organization
Aegerion Pharmaceuticals, Inc.
alison.long@aegerion.com
857-242-5142

Study Officials

  • Marina Cuchel, MD, PhD

    University of Pennsylvania

    PRINCIPAL INVESTIGATOR

  • Mark Sumeray, MD

    Aegerion Pharmaceuticals, Inc.

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 21, 2009

First Posted

July 22, 2009

Study Start

October 29, 2009

Primary Completion

September 17, 2012

Study Completion

December 1, 2014

Last Updated

June 13, 2018

Results First Posted

May 11, 2018

Record last verified: 2018-05

Locations

CA(2)IT(4)US(2)ZA(2)