Clinical trials: Family
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Amyloidosis, ATTR-CM
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes.
- Conditions
- Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR
- Trial name
- MaesTTRo (NCT06465810)
- Testing
- Treatment of transthyretin (ATTR) amyloidosis in observational study setting
- Where
- 89 sites in United States, Canada and 4 more
- Volunteers
- 1,850 people
- Sponsor
- AstraZeneca
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.
Homozygous Familial Hypercholesterolemia, HoFH
HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
Assess the effect on coronary atheroma of serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System
- Trial name
- NCT03135184
- Testing
- HDL Therapeutics PDS-2 System
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- HDL Therapeutics
Fertility Disorders
Quantum Natural Family Planning Pilot
•The Pilot study will involve current Marquette Method users who own and use a ClearBlue Fertility monitor and who will be asked to simultaneously use the Mira monitor for the sake of comparing the two devices to monitor the…
- Trial name
- QNFP (NCT03908697)
- Testing
- Mira monitor comparison to ClearBlue monitor
- Where
- Canada
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Quanovate Tech Inc.
Familial Adenomatous Polyposis
Evaluate REC-4881 in Participants With Familial Adenomatous Polyposis (FAP)
This is a multicenter, two-part trial in participants with FAP.
- Trial name
- TUPELO (NCT05552755)
- Testing
- REC-4881
- Where
- 17 sites in United States
- Volunteers
- 67 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Hyperlipidemia, Familial Combined
Phase 3 Study to Evaluate the Efficacy and Safety of Omega-3-acids Ethylesters 90 in Type Ⅱb Hyperlipidemia
The purpose of this study is to evaluate the efficacy and safety of combination therapy of omega-3-acids ethylesters 90/Atorvastatin calcium in type Ⅱb hyperlipidemia
- Trial name
- NCT02035215
- Testing
- Atorvastatin 20mg, Omega-3-acids ethylesters 90 4g
- Where
- 4 sites in South Korea
- Volunteers
- 80 people
- Sponsor
- Kuhnil Pharmaceutical Co., Ltd.
Atherosclerosis, Cardiovascular Disease
A Study of VERVE-102 (LY4636581) in Adults With or at High Risk for Cardiovascular Disease
The purpose of this study is to measure the change in bad cholesterol (LDL-C) with VERVE-102 compared to placebo in adults who have plaque buildup in their arteries or are at high risk of developing it.
- Conditions
- Atherosclerosis, Cardiovascular Disease, Hypercholesterolemia
- Trial name
- HEART-3 (NCT07839936)
- Testing
- VERVE-102
- Where
- United States
- Volunteers
- 175 people
- Sponsor
- Verve Therapeutics, 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
Hypercholesterolemia, Familial Hypercholesterolemia
A 52-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05245)
This study is a non-interventional (observational) study in Japan to confirm the safety and efficacy of Zetia when administered alone or in combination with other lipid-lowering drugs in daily medical practice throughout a…
- Conditions
- Hypercholesterolemia, Familial Hypercholesterolemia, Homozygous Sitosterolemia
- Trial name
- NCT00705211
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 1,794 people
- Sponsor
- Organon and Co
Primary Pulmonary Hypertension
Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Dry Powder Inhalation of Treprostinil
The primary objective of this study is to evaluate the long-term safety of LIQ861 in patients with pulmonary arterial hypertension (PAH).
- Trial name
- NCT03992755
- Testing
- LIQ861 Inhaled Treprostinil
- Where
- 25 sites in United States
- Volunteers
- 92 people
- Sponsor
- Liquidia Technologies, Inc.
Dementia, Alzheimer Disease 1
Study of APH-1105 in Patients With Mild to Moderate Alzheimer's Disease
This is a Phase 2 study assessing the safety, tolerability and efficacy of intranasal delivery of APH-1105 for the treatment of mild to moderate Alzheimer's in adult.
- Conditions
- Dementia, Alzheimer Disease 1, Alzheimer Disease 2, Alzheimer Disease 3
- Trial name
- NCT03806478
- Testing
- APH-1105
- Volunteers
- 60 people
- Sponsor
- Aphios
Non Familial Chylocmicronemia Syndrome (Non-FCS)
A Pilot Study to Assess the Efficacy and Safety of LCQ908 Alone and in Combination With Fenofibrate or Lovaza® in Patients With Severe Hypertriglyceridemia
This study is to determine a dose response signal for LCQ908 monotherapy and to assess the efficacy and safety of adding LCQ908 to Lovaza or fenofibrate.
- Trial name
- NCT01594983
- Testing
- LCQ908, Fenofibrate, Fish Oil
- Where
- 32 sites in United States, Canada and 1 more
- Volunteers
- 58 people
- Sponsor
- Novartis Pharmaceuticals
Atherosclerotic Cardiovascular Disease, Heterozygous Familial Hypercholesterolemia
Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease
LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy.
- Conditions
- Atherosclerotic Cardiovascular Disease, Heterozygous Familial Hypercholesterolemia, Hypercholesterolemia
- Trial name
- NCT06112327
- Where
- 3 sites in New Zealand, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Verve Therapeutics, Inc.
Heart Failure
Model 4396 Left Ventricular (LV) Lead Chronic Performance Study
Lead survivability will be summarized.
- Trial name
- NCT01361685
- Where
- 52 sites in United States, Canada and 1 more
- Volunteers
- 366 people
- Sponsor
- Medtronic
Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism
Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Homozygous familial Hypercholesterolemia from blood
- Conditions
- Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism, Corneal Arcus
- Trial name
- BioHoFH (NCT03198897)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Ear Deformities, Acquired, Ear; Deformity, Congenital
Evaluation of the Auryzon Devices in the Optimization of Ear and Nose Reconstruction Procedures
This study evaluates the efficacy of the AuryzoN devices in the ear and nose reconstruction surgeries, both in terms of operative time and overall quality of reconstruction.
- Conditions
- Ear Deformities, Acquired, Ear; Deformity, Congenital, Microtia, Nose Deformity
- Trial name
- NCT03624608
- Testing
- AuryzoN Family of Devices
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Reconstrata, LLC
Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Left Ventricular Cardiomyopathy
IC14 (Atibuclimab) in Arrhythmogenic Cardiomyopathy
The goal of this clinical trial is to test IC14 (atibuclimab) in patients with arrhythmogenic cardiomyopathy (ACM) and who have an implantable cardoverter/defibrillator in place.
- Conditions
- Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Left Ventricular Cardiomyopathy, Arrhythmogenic Right Ventricular Cardiomyopathy 1, Arrhythmogenic Cardiomyopathy
- Trial name
- NCT06275893
- Testing
- IC14
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Implicit Bioscience