Clinical trials: Family
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
Puberty, Precocious
Pediatrics Testotoxicosis Study [Bicalutamide Anastrozole Treatment for Testotoxicosis]
The primary objective of this study is to investigate whether bicalutamide given in combination with anastrozole once daily for 12 months is effective in treating testotoxicosis in boys.
- Trial name
- BATT (NCT00094328)
- Testing
- Bicalutamide, Anastrozole
- Where
- 15 sites in United States, Canada and 4 more
- Volunteers
- 14 people
- Sponsor
- AstraZeneca
Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency
The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome
An open-label study of volanesorsen (ISIS 304801) administered subcutaneously to participants with FCS.
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Trial name
- NCT02658175
- Testing
- Volanesorsen
- Where
- 34 sites in United States, Brazil and 9 more
- Volunteers
- 68 people
- Sponsor
- Akcea Therapeutics
Hypercholesterolemia
Evolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study Enrollment
To evaluate the efficacy of subcutaneous (SC) evolocumab, compared to regularly scheduled low-density lipoprotein cholesterol (LDL-C) apheresis, on reducing the need for future apheresis.
- Trial name
- NCT02585895
- Testing
- Evolocumab, Low-density Lipoprotein Cholesterol (LDL-C) Apheresis
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 39 people
- Sponsor
- Amgen
Familial Hypercholesterolemia - Heterozygous
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol…
- Trial name
- ORION-16 (NCT04652726)
- Testing
- Inclisiran
- Where
- 59 sites in United States, Argentina and 24 more
- Volunteers
- 141 people
- Sponsor
- Novartis Pharmaceuticals
PFIC1, PFIC2
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC)…
- Trial name
- PEDFIC 1 (NCT03566238)
- Testing
- A4250 (odevixibat)
- Where
- 45 sites in United States, Australia and 13 more
- Volunteers
- 62 people
- Sponsor
- Albireo
Dyslipidemias, High Cholesterol
Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies
This study will be a placebo-controlled, double-blind, randomized, phase 3 study in participants with underlying heterozygous familial hypercholesterolemia (HeFH) and/or ASCVD to evaluate the efficacy, safety, and tolerability of…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROADWAY (NCT05142722)
- Testing
- Obicetrapib
- Where
- 181 sites in United States, China and 6 more
- Volunteers
- 2,530 people
- Sponsor
- NewAmsterdam Pharma
Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
- Trial name
- REALIZE (NCT01524289)
- Testing
- Anacetrapib
- Volunteers
- 306 people
- Sponsor
- Merck Sharp & Dohme LLC
Homozygous Familial Hypercholesterolemia
Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities
A study to determine the safety, tolerability, and efficacy of evolocumab (AMG 145) in patients with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- TESLA (NCT01588496)
- Testing
- Evolocumab
- Where
- 21 sites in United States, Belgium and 10 more
- Volunteers
- 58 people
- Sponsor
- Amgen
Hypercholesterolemia, Heterozygous Familial
A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
Primary objective: Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein…
- Trial name
- FOCUS FH (NCT01475825)
- Testing
- mipomersen sodium 200 mg, mipomersen sodium 70 mg
- Where
- 113 sites in United States, Argentina and 28 more
- Volunteers
- 309 people
- Sponsor
- Kastle Therapeutics, LLC
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
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.
Heart Failure With Preserved Ejection Fraction
A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT
This is a Phase 2a proof-of-concept study to assess safety, tolerability, and preliminary efficacy of mavacamten treatment on biomarker levels in participants with heart failure with preserved ejection fraction (HFpEF) and…
- Trial name
- EMBARK-HFpEF (NCT04766892)
- Testing
- mavacamten
- Where
- 21 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Bristol-Myers Squibb
Homozygous Familial Hypercholesterolemia
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
The goal of this trial is to study the effects of AEGR-733 on LDL cholesterol, other lipids as well as measures of safety over the long-term.
- Trial name
- NCT00730236
- Testing
- AEGR-733
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
X-linked Hypophosphatemia
A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH).
- Trial name
- NCT01340482
- Testing
- KRN23
- Where
- 6 sites in United States, Canada
- Volunteers
- 29 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Transthyretin (TTR) Amyloid Cardiomyopathy
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy
- Trial name
- NCT02791230
- Testing
- Tafamidis
- Where
- 80 sites in United States, Argentina and 15 more
- Volunteers
- 1,733 people
- Sponsor
- Pfizer
Amyloidosis, Hereditary, Transthyretin Amyloidosis
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis).
- Trial name
- NCT03759379
- Testing
- Patisiran, Vutrisiran
- Where
- 64 sites in United States, Argentina and 20 more
- Volunteers
- 164 people
- Sponsor
- Alnylam Pharmaceuticals
Dyslipidemias, Hypercholesterolemia
Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies
The study is a placebo-controlled, double-blind, randomized, phase 3 study in participants with heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (ASCVD) or multiple ASCVD risk…
- Conditions
- Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD
- Trial name
- TANDEM (NCT06005597)
- Testing
- Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe
- Where
- 45 sites in United States
- Volunteers
- 407 people
- Sponsor
- NewAmsterdam Pharma
Infections, Rotavirus
A Study to Explore the Existence of Horizontal Transmission of the RIX4414 Vaccine Strain Between Twins Within a Family.
The aim of this study is to explore horizontal transmission of the HRV (Human Rotavirus) vaccine strain within a family from the twin vaccinated with Rotarix to the twin receiving placebo.
- Trial name
- NCT00396630
- Testing
- Rotarix
- Where
- Dominican Republic
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Acute Myeloid Leukemia, Leukemia
A Phase I Study of AC220 in Patients With Relapsed/Refractory Acute Myeloid Leukemia Regardless of FLT3 Status
Patients received oral AC220 daily for 14 days to study the side effects, tolerability and best dose for treating relapsed or refractory acute myeloid leukemia, regardless of FLT3 status.
- Conditions
- Acute Myeloid Leukemia, Leukemia, Myelodysplastic Syndrome, AML
- Trial name
- NCT00462761
- Testing
- AC220
- Where
- 5 sites in United States, Georgia
- Volunteers
- 76 people
- Sponsor
- Daiichi Sankyo