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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02265952
• Date of registration: 10/10/2014
• Prospective Registration: Yes
• Primary sponsor: Regeneron Pharmaceuticals
• Public title: Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
• Scientific 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
• Date of first enrolment: February 4, 2015
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02265952
• 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

Canada, Netherlands, United States

Contacts

• Name: Clinical Trial Management
• Affiliation: Regeneron Pharmaceuticals

Key inclusion & exclusion 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. Background medical lipid modifying therapy that has not been stable for at least 4
weeks (6 weeks for fibrates) prior to the screening visit

2. Having undergone lipid apheresis within 4 weeks prior to the screening visit

3. Use of another investigational drug or therapy within 30 days or at least 5 half-lives
(whichever is longer) prior to the screening visit

4. Previous participation in any clinical trial of REGN1500

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.



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

Health Condition(s) or Problem(s) studied

Homozygous Familial Hypercholesterolemia

Intervention(s)

Drug: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC

Primary Outcome(s)

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 4]

Secondary Outcome(s)

Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) Over Time in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 4]

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Week 2 to Week 4 in the Main Study Period [Time Frame: Week 2 to Week 4]

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of = 25% From Baseline (Week 0) in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

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 26) to Week 214 in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

Absolute Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 16]

Percent 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) up to Week 26 in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) Over Time in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of = 50% From Baseline (Week 26) in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

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) up to Week 26 in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

Percent 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 26) to Week 214 in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period [Time Frame: Baseline (Week 0) to Week 16]

Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period [Time Frame: Baseline (Week 0) up to Week 26]

Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of = 25% From Baseline (Week 26) in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) to Week 214]

Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period [Time Frame: Baseline (Week 26) up to Week 214]

Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Week 2 to Week 4 in the Main Study Period [Time Frame: Week 2 to Week 4]

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of = 50% From Baseline (Week 0) in the Main Study Period [Time Frame: Baseline (Week 0) to Week 26]

Secondary ID(s)

R1500-CL-1331

2016-000411-32

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 09/12/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02265952