Clinical trials: Hypercholesterolemia, Autosomal Dominant
- Condition Hypercholesterolemia, Autosomal Dominant
- Age group Children (under 18)
- Has posted results yes
Hypercholesterolemia
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Primary Objective: To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment…
- Trial name
- NCT03510715
- Testing
- Alirocumab SAR236553 (REGN727), Atorvastatin, Simvastatin
- Where
- 10 sites in Brazil, Canada and 8 more
- Volunteers
- 18 people
- Sponsor
- Sanofi
Pediatric Heterozygous Hypercholesterolemia
8-Week PK/PD Atorvastatin Study In Children And Adolescents With Heterozygous Familial Hypercholesterolemia
To evaluate pharmacokinetics, pharmacodynamics, safety and tolerability of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia
- Trial name
- NCT00739999
- Testing
- Atorvastatin
- Where
- 3 sites in Canada, Greece and 1 more
- Volunteers
- 39 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Hypercholesterolemia, Familial, Heterozygous Familial Hypercholesterolemia
A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0524A-158)
The purpose of this study is to determine the pharmacokinetics of laropiprant following administration of a single dose of 1 (Panel A) and 2 (Panel B) combination tablets of MK-0524A in adolescents with heterozygous familial…
- Trial name
- NCT01583647
- Testing
- MK-0524A
- Volunteers
- 10 people
- Sponsor
- Merck Sharp & Dohme LLC
Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome
Pediatric Reporting of Adult-Onset Genomic Results
The Investigators will conduct a longitudinal, mixed-methods cohort study to assess primary and secondary psychosocial outcomes among MyCode adolescent participants and their parents, and health behaviors of children who received…
- Conditions
- Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome, Familial Hypercholesterolemia
- Trial name
- NCT03832985
- Testing
- Child(ren) receive an adult-onset result, Child(ren) received a pediatric-onset result, Control - Negative Result
- Where
- United States
- Volunteers
- 162 people, healthy volunteers welcome
- Sponsor
- Geisinger Clinic
Hypercholesteremia
Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)
The purpose of this study was to assess the efficacy, safety, and tolerability of multiple doses of Gemcabene in patients with HoFH on stable, lipid-lowering therapy.
- Trial name
- NCT02722408
- Testing
- Gemcabene
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 8 people
- Sponsor
- NeuroBo Pharmaceuticals Inc.
Homozygous Familial Hypercholesterolemia
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objectives of the study are: To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH) To evaluate the long-term safety and tolerability of…
- Trial name
- NCT03409744
- Testing
- evinacumab
- Where
- 38 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.
- Trial name
- ORION-2 (NCT02963311)
- Testing
- ALN-PCSSC, Standard of Care
- Where
- 3 sites in United States, Netherlands and 1 more
- Volunteers
- 9 people
- Sponsor
- The Medicines Company
Familial Hypercholesterolemia
Atorvastatin Three Year Pediatric Study
The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial…
- Trial name
- NCT00827606
- Testing
- atorvastatin
- Where
- 40 sites in United States, Belgium and 13 more
- Volunteers
- 272 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Homozygous Familial Hypercholesterolemia
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks.
- Trial name
- Gateway (NCT05217667)
- Testing
- ARO-ANG 3 Injection
- Where
- 7 sites in United States, Australia and 2 more
- Volunteers
- 18 people
- Sponsor
- Arrowhead Pharmaceuticals
Hypercholesterolaemia
An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
Primary Objective: To evaluate the effect of alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 8 weeks of treatment in heterozygous familial hypercholesterolemia (heFH) participants aged of 8 to 17 years…
- Trial name
- ODYSSEY KIDS (NCT02890992)
- Testing
- alirocumab SAR236553 (REGN727), statins, ezetimibe
- Where
- 16 sites in United States, Canada and 8 more
- Volunteers
- 42 people
- Sponsor
- Sanofi
Familial Hypercholesterolemia
PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin
The primary objective of this study is to determine the efficacy of once-daily rosuvastatin in reducing LDL-C in children and adolescents aged 10-17 years with HeFH from baseline (Day 0) to the end of the 12-week double-blind…
- Trial name
- NCT00355615
- Testing
- Rosuvastatin
- Where
- 20 sites in United States, Canada and 3 more
- Volunteers
- 173 people
- Sponsor
- AstraZeneca
Hypercholesterolemia
A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)
Multiple-dose study to measure pharmacokinetics, pharmacodynamics and safety of bempedoic acid in pediatric participants 6 to 17 years of age with HeFH.
- Trial name
- CLEAR Path 1 (NCT05694260)
- Testing
- Bempedoic acid
- Where
- 24 sites in United States, Canada and 4 more
- Volunteers
- 31 people
- Sponsor
- Esperion Therapeutics, Inc.
Hypercholesterolemia
Efficacy and Safety of Colesevelam in Pediatric Patients With Genetic High Cholesterol
This study will evaluate the lipid-lowering effect and safety of colesevelam therapy administered to heterozygous familial pediatric patients 10 through 17 years of age who are on a stable dose of a pediatric-approved statin…
- Trial name
- NCT00145574
- Testing
- colesevelam HCl
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 194 people
- Sponsor
- Daiichi Sankyo
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
To evaluate the safety and efficacy of extended dosing with mipomersen (ISIS 301012) in participants with familial hypercholesterolemia or severe hypercholesterolemia on lipid-lowering therapy who had completed either the…
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- NCT00694109
- Testing
- Mipomersen Sodium
- Where
- 33 sites in United States, Brazil and 5 more
- Volunteers
- 144 people
- Sponsor
- Kastle Therapeutics, LLC
Primary Hypercholesterolemia
Pediatric Study to Evaluate the Efficacy and Safety of Ezetimibe Monotherapy in Children With Primary Hypercholesterolemia (P05522)
The purpose of this study is to determine the effect of ezetimibe 10 mg/day compared to placebo on the reduction of low-density lipoprotein cholesterol (LDL-C) from baseline to 12 weeks of treatment in children >=6 to <=10 years…
- Trial name
- NCT00867165
- Testing
- ezetimibe
- Volunteers
- 138 people
- Sponsor
- Organon and Co
Homozygous Familial Hypercholesterolemia HoFH
Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
- Trial name
- RAMAN (NCT03403374)
- Testing
- evolocumab
- Where
- 12 sites in India
- Volunteers
- 30 people
- Sponsor
- Amgen
Hypercholesterolaemia
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
Primary Objective: To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks (Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein cholesterol (LDL-C) levels…
- Trial name
- NCT03510884
- Testing
- Alirocumab SAR236553 (REGN727), Rosuvastatin, Atorvastatin
- Where
- 43 sites in United States, Argentina and 22 more
- Volunteers
- 153 people
- Sponsor
- Sanofi
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- RADICHOL 1 (NCT00607373)
- Testing
- mipomersen
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 51 people
- Sponsor
- Kastle Therapeutics, LLC
Homozygous Familial Hypercholesterolaemia (HoFH)
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein…
- Trial name
- NCT04681170
- Testing
- Lomitapide
- Where
- 12 sites in Germany, Israel and 4 more
- Volunteers
- 46 people
- Sponsor
- Amryt Pharma
Homozygous Familial Hypercholesterolemia
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial…
- Trial name
- NCT03399786
- Testing
- evinacumab
- Where
- 30 sites in United States, Australia and 9 more
- Volunteers
- 65 people
- Sponsor
- Regeneron Pharmaceuticals