Clinical trial NCT03694197: Long Term Safety Study of PRALUENT
Official title: Long Term Safety Study of PRALUENT in Patients With Heterozygous Familial Hypercholesterolemia or With Non-Familial Hypercholesterolemia at High and Very High Cardiovascular Risk and Previously Enrolled in the Neurocognitive Function Trial
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Heterozygous Familial Hypercholesterolemia
Non-familial Hypercholesterolemia
Lead sponsor: Regeneron Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Sanofi
First posted: 2018-10-03
Enrollment: 1389
Sex: ALL
Minimum age (years): 40
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of the study was to evaluate the long term safety of PRALUENT in participants with heterozygous familial hypercholesterolemia (heFH) or non-familial hypercholesterolemia (FH) participants at high or very high cardiovascular risk who completed the neurocognitive function study R727-CL-1532 (NCT02957682).

The secondary objectives of the study were:

* To evaluate the effect of PRALUENT on low-density lipoprotein cholesterol (LDL-C)
* To evaluate the effect of PRALUENT on other lipid parameters
* To evaluate the effect of PRALUENT on gonadal steroid hormones


Eligibility criteria: Key Inclusion Criteria:

Participants randomized into the neurocognitive function study (R727-CL-1532) who completed treatment and the end of study (EOS) visit with no premature or permanent discontinuation of study drug.

Key Exclusion Criteria:

1. Significant protocol deviation in the parent study (neurocognitive function study R727-CL-1532: NCT02957682) based on the investigator's or sponsor's judgment, such as noncompliance
2. Any participant who experienced an AE leading to permanent discontinuation from the neurocognitive function study R727-CL-1532 (NCT02957682).
3. Any new condition or worsening of an existing condition which, in the opinion of the investigator or per the PRALUENT local label, would make the participant unsuitable for enrollment or could interfere with the participant participating in or completing the open-label extension (OLE) study
4. Known hypersensitivity to monoclonal antibody or any component of the drug product
5. Pregnant or breastfeeding women

Note: Other inclusion/ exclusion criteria apply

Primary outcomes:
measure: Number of Participants With Adverse Events (AE) After First Administration of Study Drug Through the Last Dose of Study Drug Plus 2 Weeks
timeFrame: After first administration of study drug through the last dose of study drug plus 2 weeks, up to 80 weeks
Secondary outcomes:
measure: Calculated Low-density Lipoprotein Cholesterol (LDL-C) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change in LDL-C From Baseline Over Time
timeFrame: Up to week 72
measure: Total Cholesterol (Total-C) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in Total-C Over Time
timeFrame: Up to week 72
measure: Lipoprotein a (Lp(a)) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in Lp(a) Over Time
timeFrame: Up to week 72
measure: Non-high-density Lipoprotein Cholesterol (Non-HDL-C) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in Non-HDL-C Over Time
timeFrame: Up to week 72
measure: High-density Lipoprotein Cholesterol (HDL-C) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in HDL-C Over Time
timeFrame: Up to week 72
measure: Fasting Triglycerides (TGs) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in Fasting TGs Over Time
timeFrame: Up to week 72
measure: Apolipoprotein B (Apo B) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in Apo B Over Time
timeFrame: Up to week 72
measure: Apolipoprotein-A1 (Apo A1) Values From Baseline Over Time
timeFrame: Up to week 72
measure: Percent Change From Baseline in Apo A1 Over Time
timeFrame: Up to week 72
measure: Gonadal Hormone (Follicle Stimulating Hormone [FSH] and Luteinizing Hormone [LH]) Values for Female Participants From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Gonadal Hormones (FSH and LH) for Female Participants Over Time
timeFrame: Up to week 72
measure: Gonadal (FSH and LH) Hormone Values for Male Participants From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Gonadal Hormones (FSH and LH) for Male Participants Over Time
timeFrame: Up to week 72
measure: Gonadotropin (Estradiol) Values for Female Participants From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Gonadotropins (Estradiol) for Female Participants Over Time
timeFrame: Up to week 72
measure: Gonadotropin (Testosterone) Values for Male Participants From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Gonadotropins (Testosterone) for Male Participants Over Time
timeFrame: Up to week 72
measure: Alanine Aminotransferase Values From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Alanine Aminotransferase Over Time
timeFrame: Up to week 72
measure: Aspartate Aminotransferase Values From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Aspartate Aminotransferase Over Time
timeFrame: Up to week 72
measure: Alkaline Phosphatase Values From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Alkaline Phosphatase Over Time
timeFrame: Up to week 72
measure: Total Bilirubin Values From Baseline Over Time
timeFrame: Up to week 72
measure: Change From Baseline in Total Bilirubin Over Time
timeFrame: Up to week 72
Other outcomes:
Locations:
city: Auburn
country: United States
facility: Regeneron Research Site
state: Alabama
city: Mobile
country: United States
facility: Regeneron Research Site
state: Alabama
city: Beverly Hills
country: United States
facility: Regeneron Research Site
state: California
city: Los Gatos
country: United States
facility: Regeneron Research Site
state: California
city: North Hollywood
country: United States
facility: Regeneron Research Site
state: California
city: Port Hueneme
country: United States
facility: Regeneron Research Site
state: California
city: Aurora
country: United States
facility: Regeneron Research Site
state: Colorado
city: Colorado Springs
country: United States
facility: Regeneron Research Site
state: Colorado
city: Lake Worth
country: United States
facility: Regeneron Research Site
state: Florida
city: Miami Springs
country: United States
facility: Regeneron Research Site
state: Florida
city: Champaign
country: United States
facility: Regeneron Research Site
state: Illinois
city: Evansville
country: United States
facility: Regeneron Research Site
state: Indiana
city: Indianapolis
country: United States
facility: Regeneron Research Site
state: Indiana
city: Ames
country: United States
facility: Regeneron Research Site
state: Iowa
city: Iowa City
country: United States
facility: Regeneron Research Site
state: Iowa
city: Waterloo
country: United States
facility: Regeneron Research Site
state: Iowa
city: Newton
country: United States
facility: Regeneron Research Site
state: Kansas
city: Louisville
country: United States
facility: Regeneron Research Site
state: Kentucky
city: Bangor
country: United States
facility: Regeneron Research Site
state: Maine
city: Baltimore
country: United States
facility: Regeneron Research Site
state: Maryland
city: Oxon Hill
country: United States
facility: Regeneron Research Site
state: Maryland
city: Olive Branch
country: United States
facility: Regeneron Research Site
state: Mississippi
city: Washington
country: United States
facility: Regeneron Research Site
state: Missouri
city: Buffalo
country: United States
facility: Regeneron Research Site
state: New York
city: New Hyde Park
country: United States
facility: Regeneron Research Site
state: New York
city: Cary
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Charlotte
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Charlotte
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Greensboro
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Hickory
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Raleigh
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Rocky Mount
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Salisbury
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Statesville
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Wilmington
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Winston-Salem
country: United States
facility: Regeneron Research Site
state: North Carolina
city: Cincinnati
country: United States
facility: Regeneron Research Site
state: Ohio
city: Cleveland
country: United States
facility: Regeneron Research Site
state: Ohio
city: Dayton
country: United States
facility: Regeneron Research Site
state: Ohio
city: Charleston
country: United States
facility: Regeneron Research Site
state: South Carolina
city: Summerville
country: United States
facility: Regeneron Research Site
state: South Carolina
city: Rapid City
country: United States
facility: Regeneron Research Site
state: South Dakota
city: Bristol
country: United States
facility: Regeneron Research Site
state: Tennessee
city: Knoxville
country: United States
facility: Regeneron Research Site 2
state: Tennessee
city: Knoxville
country: United States
facility: Regeneron Research Site 3
state: Tennessee
city: Knoxville
country: United States
facility: Regeneron Research Site
state: Tennessee
city: Powell
country: United States
facility: Regeneron Research Site
state: Tennessee
city: Dallas
country: United States
facility: Regeneron Research Site
state: Texas
city: Houston
country: United States
facility: Regeneron Research Site
state: Texas
city: Schertz
country: United States
facility: Regeneron Research Site
state: Texas
city: Shavano Park
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facility: Regeneron Research Site
state: Texas
city: Shavano Park
country: United States
facility: Regeneron Research Site
state: Texas
city: Falls Church
country: United States
facility: Regeneron Research Site
state: Virginia
city: Winchester
country: United States
facility: Regeneron Research Site
state: Virginia
city: Tacoma
country: United States
facility: Regeneron Research Site
state: Washington
city: Walla Walla
country: United States
facility: Regeneron Research Site
state: Washington
city: Manitowoc
country: United States
facility: Regeneron Research Site
state: Wisconsin
city: Pleven
country: Bulgaria
facility: Regeneron Research Site
city: Plovdiv
country: Bulgaria
facility: Regeneron Research Site
city: Sofia
country: Bulgaria
facility: Regeneron Research Site
city: Sofia
country: Bulgaria
facility: Regeneron Research Site
city: Sofia
country: Bulgaria
facility: Regeneron Research Site #2
city: Stara Zagora
country: Bulgaria
facility: Regeneron Research Site
city: Varna
country: Bulgaria
facility: Regeneron Research Site
city: Tallinn
country: Estonia
facility: Regeneron Research Site
state: Harjumaa
city: Paide
country: Estonia
facility: Regeneron Research Site
city: Tallinn
country: Estonia
facility: Regeneron Research Site #2
city: Tallinn
country: Estonia
facility: Regeneron Research Site
city: Tartu
country: Estonia
facility: Regeneron Research Site
city: Ekaterinburg
country: Russian Federation
facility: Regeneron Research Site
city: Ivanovo
country: Russian Federation
facility: Regeneron Research Site
city: Kirov
country: Russian Federation
facility: Regeneron Research Site
city: Moscow
country: Russian Federation
facility: Regeneron Research Site
city: Moscow
country: Russian Federation
facility: Regeneron Research Site
city: Moscow
country: Russian Federation
facility: Regeneron Research Site
city: Novosibirsk
country: Russian Federation
facility: Regeneron Research Site
city: Rostov-na-Donu
country: Russian Federation
facility: Regeneron Research Site
city: Saint Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: Saint-Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: Saint-Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: Saratov
country: Russian Federation
facility: Regeneron Research Site
city: St. Petersburg
country: Russian Federation
facility: Regeneron Research Site #2
city: St. Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: St. Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: St. Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: St. Petersburg
country: Russian Federation
facility: Regeneron Research Site
city: Sverdlovskaya
country: Russian Federation
facility: Regeneron Research Site
city: Tyumen
country: Russian Federation
facility: Regeneron Research Site
city: Yaroslavl
country: Russian Federation
facility: Regeneron Research Site 1
city: Yaroslavl
country: Russian Federation
facility: Regeneron Research Site 2
city: Kuilsrivier
country: South Africa
facility: Regeneron Research Site
state: Cape Town
city: Parow
country: South Africa
facility: Regeneron Research Site
state: Cape Town
city: Port Elizabeth
country: South Africa
facility: Regeneron Research Site
state: Eastern Cape
city: Halfway House
country: South Africa
facility: Regeneron Research Site
state: Gauteng
city: Pretoria West
country: South Africa
facility: Regeneron Research Site
state: Gauteng
city: Pretoria
country: South Africa
facility: Regeneron Research Site
state: Gauteng
city: Pretoria
country: South Africa
facility: Regeneron Research Site
state: Gauteng
city: Pretoria
country: South Africa
facility: Regeneron Research Site
state: Gauteng
city: Kempton Park
country: South Africa
facility: Regeneron Research Site
state: Johannesburg
city: Soweto
country: South Africa
facility: Regeneron Research Site
state: Johannesburg
city: Cape Town
country: South Africa
facility: Regeneron Research Site
state: Western Cape
city: Cape Town
country: South Africa
facility: Regeneron Research Site
state: Western Cape
city: George
country: South Africa
facility: Regeneron Research Site
state: Western Cape
city: Paarl
country: South Africa
facility: Regeneron Research Site
state: Western Cape
city: Somerset West
country: South Africa
facility: Regeneron Research Site
state: Western Cape
city: Worcester
country: South Africa
facility: Regeneron Research Site
state: Western Cape
city: Bloemfontein
country: South Africa
facility: Regeneron Research Site
city: Claremont
country: South Africa
facility: Regeneron Research Site
city: Johannesburg
country: South Africa
facility: Regeneron Research Site
city: Kharkiv
country: Ukraine
facility: Regeneron Research Site #2
city: Kharkov
country: Ukraine
facility: Regeneron Research Site
city: Kiev
country: Ukraine
facility: Regeneron Research Site
city: Kyiv
country: Ukraine
facility: Regeneron Research Site
city: Kyiv
country: Ukraine
facility: Regeneron Research Site
city: Kyiv
country: Ukraine
facility: Regeneron Research Site
city: Kyiv
country: Ukraine
facility: Regeneron Research Site
city: Kyiv
country: Ukraine
facility: Regeneron Research Site
city: Kyiv
country: Ukraine
facility: Regeneron Research Site #2
city: Kyiv
country: Ukraine
facility: Regeneron Research Site
city: Lviv
country: Ukraine
facility: Regeneron Research Site
city: Uzhorod
country: Ukraine
facility: Regeneron Research Site
city: Vinnitsa
country: Ukraine
facility: Regeneron Research Site
city: Vinnitsya
country: Ukraine
facility: Regeneron Research Site
city: Vinnytsya
country: Ukraine
facility: Regeneron Research Site
Study officials:
affiliation: Regeneron Pharmaceuticals
name: Clinical Trial Management
role: STUDY_DIRECTOR
References: