Clinical trials: Families
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited
Cardiovascular, Metabolic Disease
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
- Conditions
- Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder
- Trial name
- NCT07491172
- Testing
- CTX310
- Where
- 17 sites in United States, Australia and 2 more
- Volunteers
- 90 people
- Sponsor
- CRISPR Therapeutics AG
Amyotrophic Lateral Sclerosis, Cytochrome P-450 Enzyme System
A Study of LY3873862 in Participants With Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS).
The main purpose of this study in participants with Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS) is to test the safety of different doses and to determine the amount of LY3873862 that gets into the bloodstream and how…
- Trial name
- NCT07883590
- Testing
- LY3873862, CYP450 Drug Cocktail
- Where
- 4 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Eli Lilly and Company
Hyperlipoproteinemia Type II, Homozygous Familial Hypercholesterolemia
Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)
This study will evaluate the safety and effect of anacetrapib on low-density lipoprotein-cholesterol (LDL-C) when added to ongoing lipid-lowering therapy.
- Trial name
- NCT01841684
- Testing
- Anacetrapib
- Volunteers
- 2 people
- Sponsor
- Merck Sharp & Dohme LLC
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
Heart Failure, Tachyarrhythmia
Iperia/Sentus QP Study
The objective of this study is to confirm the safety and efficacy of the new Sentus OTW QP LV lead and Iperia ICD family. The study focuses on the safety and efficacy of the QP device system.
- Trial name
- NCT02181686
- Where
- 24 sites in Austria, Denmark and 9 more
- Volunteers
- 152 people
- Sponsor
- Biotronik SE & Co. KG
NSCLC, Stage IV, NSCLC Stage IIIB
Study of Tarloxotinib in Pts With NSCLC (EGFR Exon 20 Insertion, HER2-activating Mutations) & Other Solid Tumors With NRG1/ERBB Gene Fusions
Open-label, Phase 2, single treatment arm, 3 cohorts
- Conditions
- NSCLC, Stage IV, NSCLC Stage IIIB, NSCLC, Stage IIIC, NSCLC, Recurrent
- Trial name
- RAIN (NCT03805841)
- Testing
- tarloxotinib bromide
- Where
- 14 sites in United States, Canada and 1 more
- Volunteers
- 41 people
- Sponsor
- Rain Oncology Inc
Heterozygous Familial Hypercholesterolemia
Safety and Efficacy of IBI306 in HeFH Patients
IBI306 is a fully human monoclonal antibody that binds proprotein convertase substilisin/kexin type 9 (PCSK-9), preventing its interaction with the low-density lipoprotein cholesterol receptor (LDL-R) and thereby restoring LDL-R…
- Trial name
- NCT04179669
- Testing
- IBI306
- Where
- China
- Volunteers
- 149 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Clear Cell Renal Cell Carcinoma, Solid Tumors
A Phase 1 Study of AB521 Monotherapy and Combination Therapies in Renal Cell Carcinoma and Other Solid Tumors
The purpose of this study is to evaluate the safety and tolerability of: casdatifan when taken alone in participants with advanced solid tumor malignancies and clear cell renal cell carcinoma (ccRCC) during the dose escalation…
- Trial name
- ARC-20 (NCT05536141)
- Testing
- casdatifan, Cabozantinib, Zimberelimab
- Where
- 25 sites in United States, Australia and 2 more
- Volunteers
- 362 people
- Sponsor
- Arcus Biosciences, Inc.
Thyroid Cancer
An Efficacy Study Comparing ZD6474 to Placebo in Medullary Thyroid Cancer
The purpose of this study is to learn how hereditary or sporadic medullary thyroid cancer patients, treated with ZD6474, react to the drug, what happens to ZD6474 in the human body, about the side effects of ZD6474, and if ZD6474…
- Trial name
- NCT00410761
- Testing
- ZD6474 (Vandetanib)
- Where
- 127 sites in United States, Australia and 22 more
- Volunteers
- 331 people
- Sponsor
- Genzyme, a Sanofi Company
Familial Chylomicronemia Syndrome
The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome
- Trial name
- NCT02211209
- Testing
- Volanesorsen
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 67 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Amyloidosis, Amyloid Cardiomyopathy
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- ATTRibute-CM (NCT03860935)
- Testing
- acoramidis
- Where
- 104 sites in United States, Australia and 16 more
- Volunteers
- 632 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Heartfailure
Maximizing CRT Delivery by Using MultipolAr Coronary Sinus Lead FamiLy ACUITY® X4 - RALLY X4 Study
The objective of this study is to collect clinical data on safety and performance of ACUITY X4® leads when used in a standard clinical setting.
- Trial name
- RallyX4 (NCT02066467)
- Testing
- Left Ventricular lead implant: ACUITY X4® Lead Family
- Where
- 83 sites in Austria, Belgium and 16 more
- Volunteers
- 863 people
- Sponsor
- Boston Scientific Corporation
Contact Lens Care
OPTI-FREE Family STUDY
The purpose of this post-market clinical follow-up (PMCF) study is to evaluate the long-term safety and performance of 3 commercially available contact lens cleaning and disinfecting solutions in a pediatric population.
- Trial name
- NCT06592521
- Testing
- OPTI-FREE EXPRESS MPDS and Alcon contact lens case, OPTI-FREE PureMoist MPDS and Alcon contact lens case, OPTI-FREE REPLENISH MPDS and Alcon contact lens case
- Where
- United States
- Sponsor
- Alcon Research
Pancreatic Ductal Adenocarcinoma
Utilizing a Multi-gene Testing Approach to Identify Hereditary Pancreatic Cancer
The primary objective of the study will be to estimate the prevalence of germline mutations in patients who present consecutively within 12 weeks of a confirmed diagnosis of pancreatic ductal adenocarcinoma.
- Trial name
- NCT02790944
- Testing
- Multi-gene Next Generation Sequencing Panel
- Where
- 3 sites in United States
- Volunteers
- 300 people
- Sponsor
- Ambry Genetics
Heart Failure, Tachyarrhythmia
Post Market Clinical Follow-up Study for the Pamira ICD Lead Family
Confirm clinical safety and performance of the Pamira lead to support the regulatory post market strategy in Europe and other regions and validating promotional claims by demonstrating clinical safety evaluating performance based…
- Trial name
- NCT05621187
- Testing
- Pamira ICD lead family
- Where
- 23 sites in Australia, Austria and 6 more
- Volunteers
- 221 people
- Sponsor
- Biotronik SE & Co. KG
Hypercholesterolemia, Heterozygous Familial Hypercholesterolemia
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)
The primary objective of the study was to assess the long-term safety and tolerability of alirocumab in patients with heFH who were receiving concomitant treatment with hydroxymethyl glutaryl-coenzyme A (HMG-CoA) reductase…
- Trial name
- NCT01576484
- Testing
- Alirocumab
- Where
- 14 sites in United States, Canada
- Volunteers
- 58 people
- Sponsor
- Regeneron Pharmaceuticals
Hypercholesterolemia, Heterozygous Familial Hypercholesterolemia
Study of Efficacy and Safety of Inclisiran in Japanese Participants With High Cardiovascular Risk and Elevated LDL-C
This was a placebo-controlled, double-blind, randomized trial in Japanese participants with history of coronary artery disease (CAD) or participants categorized in 'high risk' by JAS 2017 guideline, or Japanese participants with…
- Trial name
- ORION-15 (NCT04666298)
- Testing
- Inclisiran sodium
- Where
- 42 sites in Japan
- Volunteers
- 312 people
- Sponsor
- Novartis Pharmaceuticals
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis.
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT01960348
- Testing
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Where
- 52 sites in United States, Argentina and 19 more
- Volunteers
- 225 people
- Sponsor
- Alnylam Pharmaceuticals