Trial record 2 of 43 for:    regeneron | Open Studies

Study of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2015 by Regeneron Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02265952
First received: October 10, 2014
Last updated: March 10, 2015
Last verified: March 2015
  Purpose

This is an open-label, single-arm study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in patients with homozygous familial hypercholesterolemia (HoFH).


Condition Intervention Phase
Homozygous Familial Hypercholesterolemia
Drug: REGN1500
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia

Resource links provided by NLM:


Further study details as provided by Regeneron Pharmaceuticals:

Primary Outcome Measures:
  • Percent change in low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) to week 4. [ Time Frame: Week 0 to week 4 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The percent and absolute change in LDL-C from week 12 to week 16 [ Time Frame: Week 12 to week 16 ] [ Designated as safety issue: No ]
  • The percent and absolute change in LDL-C from week 2 to week 4 [ Time Frame: Week 2 to week 4 ] [ Designated as safety issue: No ]
  • The percent and absolute change in LDL-C from baseline (week 0) to week 16 [ Time Frame: Week 0 to week 16 ] [ Designated as safety issue: No ]
  • The absolute change in LDL-C from baseline (week 0) to week 4 [ Time Frame: Week 0 to week 4 ] [ Designated as safety issue: No ]
  • The percent and absolute change in apolipoprotein (Apo) B, non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (total-C) and lipoprotein (a) [Lp (a)], from baseline (week 0) to week 16 [ Time Frame: Week 0 to week 16 ] [ Designated as safety issue: No ]
  • The proportion of patients that achieve a reduction in LDL-C of >15% from baseline (week 0) to week 16 [ Time Frame: Week 0 to week 16 ] [ Designated as safety issue: No ]
  • The proportion of patients with ≥30% reduction in LDL-C from baseline (week 0) to week 16 [ Time Frame: Week 0 to week 16 ] [ Designated as safety issue: No ]
  • The percent and absolute change in HDL-C, triglycerides, and Apo A-1 from baseline (week 0) to week 16 [ Time Frame: Week 0 to week 16 ] [ Designated as safety issue: No ]
  • The percent and absolute change in Apo CIII from baseline (week 0) to week 16 [ Time Frame: Week 0 to week 16 ] [ Designated as safety issue: No ]
  • The incidence and severity of treatment-emergent adverse events (TEAEs) reported from the first dose of study drug to the end of study or the last visit in subjects treated with REGN1500 [ Time Frame: Week 0 to week 25 (End of study) ] [ Designated as safety issue: Yes ]
  • Serum concentration of REGN1500 over time and other PK parameters [ Time Frame: Week 0 to week 25 (End of study) ] [ Designated as safety issue: No ]
  • Presence and titer of anti-REGN1500 antibodies over time [ Time Frame: Week 0 to week 25 (End of study) ] [ Designated as safety issue: No ]

Estimated Enrollment: 8
Study Start Date: October 2014
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open-Label
Open-label REGN1500
Drug: REGN1500

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Men and women ≥18 years of age at the time of the screening visit
  2. Diagnosis of homozygous familial hypercholesterolemia (HoFH)
  3. Willing to consistently maintain usual diet for the duration of the study

Exclusion Criteria:

  1. Use of mipomersen within 6 months prior to the screening visit
  2. Background medical lipid modifying therapy that has not been stable for at least weeks (6 weeks for fenofibrate) prior to the screening visit
  3. Having undergone lipid apheresis within 4 weeks prior to the screening visit
  4. Use of fibrates, other than fenofibrate, within 6 weeks prior to the screening visit
  5. Use of another investigational drug or therapy within 30 days or at least 5 half-lives (whichever is longer) prior to the screening visit
  6. Previous participation in any clinical trial of REGN1500
  7. Women of childbearing potential

Note: The information listed above is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial therefore not all inclusion/ exclusion criteria are listed.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02265952

Contacts
Contact: Clinical Trials Administrator clinicaltrials@regeneron.com

Locations
United States, California
Not yet recruiting
Los Angeles, California, United States
United States, Pennsylvania
Recruiting
Philadelphia, Pennsylvania, United States
United States, Texas
Withdrawn
Dallas, Texas, United States
Canada, Quebec
Recruiting
Chicoutimi, Quebec, Canada
Not yet recruiting
Quebec City, Quebec, Canada
Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
Study Director: Clinical Trial Management Regeneron Pharmaceuticals
  More Information

No publications provided

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02265952     History of Changes
Other Study ID Numbers: R1500-CL-1331
Study First Received: October 10, 2014
Last Updated: March 10, 2015
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Dyslipidemias
Genetic Diseases, Inborn
Hyperlipidemias
Hyperlipoproteinemias
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on March 30, 2015