Search clinical trials for: Genetic lipids disorders

Total 157712 results

Steminent US, Inc.

Not yet recruiting

Stemchymal® for Polyglutamine Spinocerebellar Ataxia

Spinocerebellar Ataxias
Alnylam Pharmaceuticals

Recruiting

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy (cAPPricorn-1)

Cerebral Amyloid Angiopathy

Canada
Natural Resources Institute Finland
Tampere University

Recruiting

Sand Play - the Effect of Biodiversity Exposure on Atopic Dermatitis (BIWE)

Microbial Colonization | Atopic Dermatitis | Immune System and Related Disorders | Nature, Human

Finland
Peking Union Medical College Hospital

Not yet recruiting

A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (GEN6050XIIT)

Duchenne Muscular Dystrophy (DMD)
AbbVie

Not yet recruiting

A Study to Assess the Effect of Upadacitinib in Atopic Dermatitis-induced Sleep Disturbance in Adults With Moderate to Severe Atopic Dermatitis (Sleep Up)

Sleep Disturbance | Atopic Dermatitis
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts (CALNATHIS)

Limb Girdle Muscular Dystrophy
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
University College, London
London School of Hygiene and Tropical Medicine; King's College London; University... and other collaborators

Recruiting

Deep Brain Stimulation in Tourette Syndrome (Op-TICS)

Tourette Syndrome | Deep Brain Stimulation

United Kingdom
Laval University

Recruiting

Unravelling the Impact of Diet on Cardiovascular Health in Treated Heterozygous Familial Hypercholesterolemia.

Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy

Canada
Gene Solutions
Medical Genetics Institute (MGI)

Not yet recruiting

Developing & Evaluating Models for Early Predicting Obstetrical Diseases in Pregnant Women by Non-invasive Prenatal Test

Preterm Birth | Preeclampsia | Gestational Diabetes | Pregnant With Complication
University of Utah

Recruiting

Clinical Characteristics and Temporal Properties of Individual Tics in Persistent Tic Disorder (PTD)

Tourette Syndrome | Tic Disorders | Tics | Tourette Syndrome in Children | Tourette Syndrome in Adolescence | Vocal Tic | Motor Tic | Tics/Tremor

United States
Washington University School of Medicine
Alzheimer's Association; Eisai Inc.

Not yet recruiting

DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease (DIAN-TU)

Dementia | Alzheimer's Disease | Alzheimer's Disease, Familial

United States, Australia, United Kingdom
Air Force Military Medical University, China

Not yet recruiting

Diagnosis of Depressive Disorder Malinger Using the Niacin Skin Response Test (NSRT)

Depressive Disorder | Malingering

China
University of Salerno

Not yet recruiting

Efficacy and Safety of Bempedoic Acid in Association With Anti-PCSK9 and Ezetimibe in Statin-intolerant Patients (BESAFE)

Cardiovascular Diseases | Dyslipidemias | Lipid Metabolism Disorders | Statin Adverse Reaction
Shanghai Ninth People's Hospital Affiliated to...

Recruiting

A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)

Neurofibromatosis 1 | Plexiform Neurofibroma

China
The First Affiliated Hospital of Zhejiang Chinese...

Recruiting

Peripheral Mechanisms of Humoral Immune Effector-mediated Tourette's Syndrome and the Role of Tuina Intervention

Inflammatory Response | Tourette Syndrome in Children | Food Intolerance

China
University of Colorado, Denver

Not yet recruiting

Maternal Choline Supplementation and Cannabis Use During Pregnancy: Impact on Early Brain Development

Child Development | Cannabis Use

United States
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
IRCCS Eugenio Medea

Recruiting

Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe] (MDCJ-NeuBeRe)

Myotonic Dystrophy 1

Italy
University of Pennsylvania

Not yet recruiting

Using the EHR to Advance Genomic Medicine Across a Diverse Health System

Parkinson Disease | Alzheimer Disease | ALS | Polyneuropathies | Frontotemporal Dementia | Pheochromocytoma | Thoracic Aortic Aneurysm | Paraganglioma | Genetic Predisposition | Cardiomyopathy Non-ischemic
University Hospital Schleswig-Holstein

Recruiting

Evaluation of Safety and Pharmacokinetics of Oral Controlled-ileal-release Nicotinic Acid (CIR-NA) Compared to Immediate-release Nicotinic Acid and Placebo in Healthy Subjects and Subjects With Prediabetes

Safety Issues | Pharmacokinetic

Germany
IRCCS Eugenio Medea

Recruiting

Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI] (RIMUDI)

Muscular Dystrophies | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy | Facio-Scapulo-Humeral Dystrophy

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Next-Generation alzheImer'S Therapeutics (ENERGISE)

Alzheimer Disease

Italy
Enzyre B.V.
Indiana Hemophilia & Thrombosis Center

Not yet recruiting

Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia A Monitoring

Hemophilia A

United States
Chugai Pharmaceutical

Not yet recruiting

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS) | Immune Thrombocytopenia (ITP) | Dermatomyositis (DM) | Bullous Pemphigoid (BP) | Behçet's Syndrome (BS) | Immune-mediated Necrotizing Myopathy (IMNM)

United States, Japan
Sensorion

Not yet recruiting

A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (AUDIOGENE)

Otorhinolaryngologic Diseases | Ear Diseases | Hearing Disorders | Deafness | Hearing Loss, Sensorineural | Congenital Deafness | DFNB9 | OTOF Gene Mutation

France
Assiut University

Not yet recruiting

Statins Role in Acute Ischemic Stroke (observation)

Stroke, Acute
Hospices Civils de Lyon

Not yet recruiting

Circadian Rhythm Disorders in Children With Cystic Fibrosis Under CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators (CHRONO-MUCO)

Cystic Fibrosis

France
Johns Hopkins University
American Heart Association

Not yet recruiting

Improving Cardiovascular Health Risks in Adults With Epilepsy on a Modified Atkins Diet

Epilepsy | Dyslipidemia
University of Bologna

Not yet recruiting

Middle Term Effect of Red Yeast Rice on Plasma Lipids and Proteoma in Individuals With Suboptimal Cholesterolemia

Hypercholesterolemia

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

phenotypeS in Non Ambulant Duchenne Muscular Dystrophy (GUP21003)

Duchenne Muscular Dystrophy | Natural History | Motor Function; Retardation

Italy
Harmony Biosciences, LLC

Recruiting

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States
Dan Bing

Not yet recruiting

Genetic Feature of Congenital Hearing Loss in Chinese Population

Congenital Hearing Loss | Congenital Deafness
AstraZeneca

Recruiting

hATTR Polyneuropathy in Russia (PRIMER)

Hereditary Transthyretin Amyloidosis

Russian Federation
National Cancer Institute (NCI)

Not yet recruiting

Anti-CD19 Chimeric Antigen Receptor T-Cell Immunotherapy for Chronic Lymphocytic Leukemia (CLL)

Leukemia, Lymphocytic, Chronic, B-Cell | B-Cell Chronic Lymphocytic Leukemia | B-Lymphocytic Leukemia, Chronic

United States
Chang Gung University

Recruiting

Electrical Stimulation Cycling Training Effects on SCA

Spinocerebellar Ataxia (SCA)

Taiwan
University of Malaga

Not yet recruiting

Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. (DMDrespy2024)

Muscular Dystrophy | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Spain
Seoul National University Hospital

Not yet recruiting

The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne | Upper Extremity | Orthotic Devices

Korea, Republic of
AstraZeneca

Not yet recruiting

Real-World Treatment Study of Koselugo (Selumetinib)

Neurofibromatosis 1 | Neurofibroma, Plexiform
Xuanwu Hospital, Beijing

Not yet recruiting

Exploring DBS Efficacy in Treatment-refractory Tourette's Syndrome

Tourette's Syndrome

China
Kafrelsheikh University

Recruiting

Combining Use of Clopidogrel With Atorvastatin or Rosuvastatin in Patients With Large-vessel Ischemic Stroke

Ischemic Stroke

Egypt
Assistance Publique - Hôpitaux de Paris
Vaincre la Mucoviscidose

Not yet recruiting

Oral Health Status of Cystic Fibrosis Patients. An Online Survey in Collaboration With the Vaincre la Mucoviscidose Patient Association. (E-MUCODENT)

Cystic Fibrosis

France
Kafrelsheikh University

Recruiting

Concomitant Use of Clopidogrel With Atorvastatin or Rosuvastatin in Patients With Minor Stroke or TIA

Ischemic Stroke

Egypt
IRCCS Eugenio Medea

Recruiting

Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy

Epilepsy | Angelman Syndrome | Dup15q Syndrome

Italy
Institut de Myologie, France
Association Française contre les Myopathies (AFM), Paris

Not yet recruiting

Natural History Study of Children With LAMA2-related Dystrophies (LAMA2)

Merosin Deficient Congenital Muscular Dystrophy

France
CHU de Reims

Recruiting

Bronchiectasis Prevalence in Patients With Primary Humoral Immunodefiency in Champagne-Ardenne Region, France (PREDDICHA)

Primary Immunodeficiency | Common Variable Immunodeficiency | Igg Subclass Deficiency | Selective IgM Deficiency

France
Sensorion

Not yet recruiting

Cross-sectional and Prospective Study to Characterize Early-onset Presbycusis (SONG)

Sensorineural Hearing Loss, Bilateral

France
Huazhong University of Science and Technology

Not yet recruiting

Effects of Vitamin D3 and Prebiotics Supplementation on Cardiovascular Risk Factors in Patients With Type 2 Diabetes

Diabetes Mellitus, Type 2
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients (TRANSPLANTORN2)

Sickle Cell Disease
Hasan Kalyoncu University

Recruiting

Effects of Orofacial Therapy and Therapeutic Yoga in Children With Down Syndrome

Down Syndrome

Turkey

Search clinical trials for: Genetic lipids disorders

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