Heterozygous Familial Hypercholesterolemia Clinical Trial
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
| Verified date | May 2021 |
| Source | Regeneron Pharmaceuticals |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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
| Status | Terminated |
| Enrollment | 1389 |
| Est. completion date | April 8, 2020 |
| Est. primary completion date | April 8, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 40 Years to 85 Years |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Bulgaria | Regeneron Research Site | Pleven | |
| Bulgaria | Regeneron Research Site | Plovdiv | |
| Bulgaria | Regeneron Research Site | Sofia | |
| Bulgaria | Regeneron Research Site | Sofia | |
| Bulgaria | Regeneron Research Site #2 | Sofia | |
| Bulgaria | Regeneron Research Site | Stara Zagora | |
| Bulgaria | Regeneron Research Site | Varna | |
| Estonia | Regeneron Research Site | Paide | |
| Estonia | Regeneron Research Site | Tallinn | Harjumaa |
| Estonia | Regeneron Research Site | Tallinn | |
| Estonia | Regeneron Research Site #2 | Tallinn | |
| Estonia | Regeneron Research Site | Tartu | |
| Russian Federation | Regeneron Research Site | Ekaterinburg | |
| Russian Federation | Regeneron Research Site | Ivanovo | |
| Russian Federation | Regeneron Research Site | Kirov | |
| Russian Federation | Regeneron Research Site | Moscow | |
| Russian Federation | Regeneron Research Site | Moscow | |
| Russian Federation | Regeneron Research Site | Moscow | |
| Russian Federation | Regeneron Research Site | Novosibirsk | |
| Russian Federation | Regeneron Research Site | Rostov-na-Donu | |
| Russian Federation | Regeneron Research Site | Saint Petersburg | |
| Russian Federation | Regeneron Research Site | Saint-Petersburg | |
| Russian Federation | Regeneron Research Site | Saint-Petersburg | |
| Russian Federation | Regeneron Research Site | Saratov | |
| Russian Federation | Regeneron Research Site | St. Petersburg | |
| Russian Federation | Regeneron Research Site | St. Petersburg | |
| Russian Federation | Regeneron Research Site | St. Petersburg | |
| Russian Federation | Regeneron Research Site | St. Petersburg | |
| Russian Federation | Regeneron Research Site #2 | St. Petersburg | |
| Russian Federation | Regeneron Research Site | Sverdlovskaya | |
| Russian Federation | Regeneron Research Site | Tyumen | |
| Russian Federation | Regeneron Research Site 1 | Yaroslavl | |
| Russian Federation | Regeneron Research Site 2 | Yaroslavl | |
| South Africa | Regeneron Research Site | Bloemfontein | |
| South Africa | Regeneron Research Site | Cape Town | Western Cape |
| South Africa | Regeneron Research Site | Cape Town | Western Cape |
| South Africa | Regeneron Research Site | Claremont | |
| South Africa | Regeneron Research Site | George | Western Cape |
| South Africa | Regeneron Research Site | Halfway House | Gauteng |
| South Africa | Regeneron Research Site | Johannesburg | |
| South Africa | Regeneron Research Site | Kempton Park | Johannesburg |
| South Africa | Regeneron Research Site | Kuilsrivier | Cape Town |
| South Africa | Regeneron Research Site | Paarl | Western Cape |
| South Africa | Regeneron Research Site | Parow | Cape Town |
| South Africa | Regeneron Research Site | Port Elizabeth | Eastern Cape |
| South Africa | Regeneron Research Site | Pretoria | Gauteng |
| South Africa | Regeneron Research Site | Pretoria | Gauteng |
| South Africa | Regeneron Research Site | Pretoria | Gauteng |
| South Africa | Regeneron Research Site | Pretoria West | Gauteng |
| South Africa | Regeneron Research Site | Somerset West | Western Cape |
| South Africa | Regeneron Research Site | Soweto | Johannesburg |
| South Africa | Regeneron Research Site | Worcester | Western Cape |
| Ukraine | Regeneron Research Site #2 | Kharkiv | |
| Ukraine | Regeneron Research Site | Kharkov | |
| Ukraine | Regeneron Research Site | Kiev | |
| Ukraine | Regeneron Research Site | Kyiv | |
| Ukraine | Regeneron Research Site | Kyiv | |
| Ukraine | Regeneron Research Site | Kyiv | |
| Ukraine | Regeneron Research Site | Kyiv | |
| Ukraine | Regeneron Research Site | Kyiv | |
| Ukraine | Regeneron Research Site | Kyiv | |
| Ukraine | Regeneron Research Site #2 | Kyiv | |
| Ukraine | Regeneron Research Site | Lviv | |
| Ukraine | Regeneron Research Site | Uzhorod | |
| Ukraine | Regeneron Research Site | Vinnitsa | |
| Ukraine | Regeneron Research Site | Vinnitsya | |
| Ukraine | Regeneron Research Site | Vinnytsya | |
| United States | Regeneron Research Site | Ames | Iowa |
| United States | Regeneron Research Site | Auburn | Alabama |
| United States | Regeneron Research Site | Aurora | Colorado |
| United States | Regeneron Research Site | Baltimore | Maryland |
| United States | Regeneron Research Site | Bangor | Maine |
| United States | Regeneron Research Site | Beverly Hills | California |
| United States | Regeneron Research Site | Bristol | Tennessee |
| United States | Regeneron Research Site | Buffalo | New York |
| United States | Regeneron Research Site | Cary | North Carolina |
| United States | Regeneron Research Site | Champaign | Illinois |
| United States | Regeneron Research Site | Charleston | South Carolina |
| United States | Regeneron Research Site | Charlotte | North Carolina |
| United States | Regeneron Research Site | Charlotte | North Carolina |
| United States | Regeneron Research Site | Cincinnati | Ohio |
| United States | Regeneron Research Site | Cleveland | Ohio |
| United States | Regeneron Research Site | Colorado Springs | Colorado |
| United States | Regeneron Research Site | Dallas | Texas |
| United States | Regeneron Research Site | Dayton | Ohio |
| United States | Regeneron Research Site | Evansville | Indiana |
| United States | Regeneron Research Site | Falls Church | Virginia |
| United States | Regeneron Research Site | Greensboro | North Carolina |
| United States | Regeneron Research Site | Hickory | North Carolina |
| United States | Regeneron Research Site | Houston | Texas |
| United States | Regeneron Research Site | Indianapolis | Indiana |
| United States | Regeneron Research Site | Iowa City | Iowa |
| United States | Regeneron Research Site | Knoxville | Tennessee |
| United States | Regeneron Research Site 2 | Knoxville | Tennessee |
| United States | Regeneron Research Site 3 | Knoxville | Tennessee |
| United States | Regeneron Research Site | Lake Worth | Florida |
| United States | Regeneron Research Site | Los Gatos | California |
| United States | Regeneron Research Site | Louisville | Kentucky |
| United States | Regeneron Research Site | Manitowoc | Wisconsin |
| United States | Regeneron Research Site | Miami Springs | Florida |
| United States | Regeneron Research Site | Mobile | Alabama |
| United States | Regeneron Research Site | New Hyde Park | New York |
| United States | Regeneron Research Site | Newton | Kansas |
| United States | Regeneron Research Site | North Hollywood | California |
| United States | Regeneron Research Site | Olive Branch | Mississippi |
| United States | Regeneron Research Site | Oxon Hill | Maryland |
| United States | Regeneron Research Site | Port Hueneme | California |
| United States | Regeneron Research Site | Powell | Tennessee |
| United States | Regeneron Research Site | Raleigh | North Carolina |
| United States | Regeneron Research Site | Rapid City | South Dakota |
| United States | Regeneron Research Site | Rocky Mount | North Carolina |
| United States | Regeneron Research Site | Salisbury | North Carolina |
| United States | Regeneron Research Site | Schertz | Texas |
| United States | Regeneron Research Site | Shavano Park | Texas |
| United States | Regeneron Research Site | Shavano Park | Texas |
| United States | Regeneron Research Site | Statesville | North Carolina |
| United States | Regeneron Research Site | Summerville | South Carolina |
| United States | Regeneron Research Site | Tacoma | Washington |
| United States | Regeneron Research Site | Walla Walla | Washington |
| United States | Regeneron Research Site | Washington | Missouri |
| United States | Regeneron Research Site | Waterloo | Iowa |
| United States | Regeneron Research Site | Wilmington | North Carolina |
| United States | Regeneron Research Site | Winchester | Virginia |
| United States | Regeneron Research Site | Winston-Salem | North Carolina |
| Lead Sponsor | Collaborator |
|---|---|
| Regeneron Pharmaceuticals | Sanofi |
United States, Bulgaria, Estonia, Russian Federation, South Africa, Ukraine,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AE) After First Administration of Study Drug Through the Last Dose of Study Drug Plus 2 Weeks | An AE is any untoward medical occurrence in a participant administered a study drug which may or may not have a causal relationship with the study drug. AEs include serious adverse events (SAEs), AEs leading to treatment discontinuation, and adverse events of special interest (AESI). AESI include local injection site reactions, general allergic events, elevated alanine aminotransferase (ALT) levels greater than or equal to (=) 3 upper limit normal (ULN) (if baseline is less than (<) ULN)/ALT =2 x ULN (if baseline = ULN), neurologic events, neurocognitive events (according to Customized Medical Dictionary for Regulatory Activities [MedDRA] Query [CMQ] by Sponsor grouping and CMQ by FDA grouping), cataract, new onset diabetes (NOD), hepatic disorders, and diabetes mellitus (DM)/diabetic complications. | After first administration of study drug through the last dose of study drug plus 2 weeks, up to 80 weeks | |
| Secondary | Calculated Low-density Lipoprotein Cholesterol (LDL-C) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change in LDL-C From Baseline Over Time | Up to week 72 | ||
| Secondary | Total Cholesterol (Total-C) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in Total-C Over Time | Up to week 72 | ||
| Secondary | Lipoprotein a (Lp(a)) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in Lp(a) Over Time | Up to week 72 | ||
| Secondary | Non-high-density Lipoprotein Cholesterol (Non-HDL-C) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in Non-HDL-C Over Time | Up to week 72 | ||
| Secondary | High-density Lipoprotein Cholesterol (HDL-C) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in HDL-C Over Time | Up to week 72 | ||
| Secondary | Fasting Triglycerides (TGs) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in Fasting TGs Over Time | Up to week 72 | ||
| Secondary | Apolipoprotein B (Apo B) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in Apo B Over Time | Up to week 72 | ||
| Secondary | Apolipoprotein-A1 (Apo A1) Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Percent Change From Baseline in Apo A1 Over Time | Up to week 72 | ||
| Secondary | Gonadal Hormone (Follicle Stimulating Hormone [FSH] and Luteinizing Hormone [LH]) Values for Female Participants From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Gonadal Hormones (FSH and LH) for Female Participants Over Time | Up to week 72 | ||
| Secondary | Gonadal (FSH and LH) Hormone Values for Male Participants From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Gonadal Hormones (FSH and LH) for Male Participants Over Time | Up to week 72 | ||
| Secondary | Gonadotropin (Estradiol) Values for Female Participants From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Gonadotropins (Estradiol) for Female Participants Over Time | Up to week 72 | ||
| Secondary | Gonadotropin (Testosterone) Values for Male Participants From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Gonadotropins (Testosterone) for Male Participants Over Time | Up to week 72 | ||
| Secondary | Alanine Aminotransferase Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Alanine Aminotransferase Over Time | Up to week 72 | ||
| Secondary | Aspartate Aminotransferase Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Aspartate Aminotransferase Over Time | Up to week 72 | ||
| Secondary | Alkaline Phosphatase Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Alkaline Phosphatase Over Time | Up to week 72 | ||
| Secondary | Total Bilirubin Values From Baseline Over Time | The baseline value was defined as the last available value before the first dose of double-blind study treatment in study R727-CL-1532 (NCT02957682) | Up to week 72 | |
| Secondary | Change From Baseline in Total Bilirubin Over Time | Up to week 72 |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT03563547 -
Effects of Soy Protein on Cholesterol Levels in Children Affected With Familial Hypercholesterolemia
|
N/A | |
| Active, not recruiting |
NCT04759534 -
Application of PCSK9 Inhibitors in Patients With Heterozygous Familial Hypercholesterolemia
|
Phase 3 | |
| Completed |
NCT01968980 -
A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia
|
Phase 3 | |
| Completed |
NCT00171236 -
Efficacy and Safety of Fluvastatin in Children With Heterozygous Familial Hypercholesterolemia
|
Phase 3 | |
| Completed |
NCT03397121 -
Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)
|
Phase 3 | |
| Completed |
NCT04173793 -
A Study of PCSK9 Inhibitor AK102 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
|
Phase 2 | |
| Not yet recruiting |
NCT06164730 -
A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
|
Phase 1 | |
| Completed |
NCT02326220 -
Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy
|
Phase 3 | |
| Completed |
NCT02392559 -
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
|
Phase 3 | |
| Completed |
NCT02460159 -
A Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653C in Japanese Participants With Hypercholesterolemia (MK-0653C-384)
|
Phase 3 | |
| Terminated |
NCT01583647 -
A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0524A-158)
|
Phase 1 | |
| Active, not recruiting |
NCT05398029 -
A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease
|
Phase 1 | |
| Completed |
NCT00706849 -
Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease
|
Phase 3 | |
| Completed |
NCT04666298 -
Study of Efficacy and Safety of Inclisiran in Japanese Participants With High Cardiovascular Risk and Elevated LDL-C
|
Phase 2 | |
| Completed |
NCT01515241 -
Exploratory Study of Plaque Regression
|
Phase 2 | |
| Completed |
NCT03038022 -
Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
|
Phase 2 | |
| Completed |
NCT01576484 -
Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)
|
Phase 2 | |
| Terminated |
NCT00151788 -
Efficacy and Safety of the ACAT Inhibitor CS-505 (Pactimibe) for Reducing the Progression of Carotid Artery Disease. This Study is Also Known as CAPTIVATE.
|
Phase 2/Phase 3 | |
| Completed |
NCT05325203 -
A Study to Evaluate the Efficacy and Safety of JS002 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH).
|
Phase 3 | |
| Completed |
NCT01709500 -
Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)
|
Phase 3 |