Search clinical trials for: Genetic lipids disorders

Total 157230 results

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
Hasan Kalyoncu University

Recruiting

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

Down Syndrome

Turkey
University Hospital, Bonn
Swedish Orphan Biovitrum

Recruiting

Prevalence of Synovitis in Patients With Haemophilia A (SynoPrev)

Haemophilia A | Synovitis | Hemophilia Arthropathy | Sonography

Germany
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
Equilibra Bioscience LLC

Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B)

Hemophilia A | Hemophilia B | Healthy Participants

United States
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...
Federico II University; Istituto Auxologico Italiano; Università degli Studi...

Recruiting

Maternal Genes and Epimutations: Beckwith-Wiedemann Syndrome & Reproductive Risks

Beckwith-Wiedemann Syndrome

Italy
University of Pavia
Foundation IRCCS San Matteo Hospital

Recruiting

Effect of the Association of Berberine Fitosoma® and Monacolin K MonaKoPure® on Glyco-metabolic Parameters in Dysglycemic and Dyslipidemic Patients.

Dyslipidemias | Dysglycemia

Italy
IRCCS Fondazione Stella Maris
Azienda USL Toscana Nord Ovest; University of Dublin, Trinity College; Meyer...

Recruiting

Biomarkers in Rett Syndrome (BIRS)

Rett Syndrome

Italy, Ireland
John Paul II Hospital, Krakow

Recruiting

New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis

Transthyretin Amyloidosis

Poland
Children's Hospital Colorado
University of Colorado, Denver; University of Pennsylvania; University of Alabama... and other collaborators

Recruiting

The Diagnostic Experience of Male Rett Syndrome

Rett Syndrome | RTT

United States
Poznan University of Life Sciences

Not yet recruiting

Betaine Supplementation and Lipid Metabolism in Overweight and Obese Pre-menopausal Women

Overweight and Obesity

Poland
Fondation Ophtalmologique Adolphe de Rothschild

Not yet recruiting

Functional Impairment in Albinism (PLAIB)

Albinism, Ocular
Roswell Park Cancer Institute

Not yet recruiting

Genetically Engineered Cells (EGFRt/19-28z/IL-12 CAR T Cells) for the Treatment of Relapsed or Refractory CD19+ Hematologic Malignancies

Recurrent Mantle Cell Lymphoma | Recurrent Diffuse Large B-Cell Lymphoma | Refractory Diffuse Large B-Cell Lymphoma | Refractory Chronic Lymphocytic Leukemia | Refractory Mantle Cell Lymphoma | Recurrent High Grade B-Cell Lymphoma | Recurrent Transformed Follicular Lymphoma to Diffuse Large B-Cell... and other conditions

United States
Huntington Study Group
Roche Products Limited

Recruiting

Project AWARE 2.0 to Improve Awareness, Willingness and Ability for Research and Enrollment in Clinical Research

Huntington Disease

United States
AstraZeneca

Recruiting

Transthyretin Amyloidosis Cardiomyopathy in Patients With HFpEF in Russia (TETRAMER)

Transthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection Fraction

Russian Federation
Samsung Medical Center

Recruiting

Optimal Attained LDL-Cholesterol Levels in Patients Who Achieved ≥50% LDL Reduction After PCI (Lipid-PCI)

Coronary Artery Disease | Hyperlipidemias

Korea, Republic of
Jirair Krikor Bedoyan
Ultragenyx Pharmaceutical Inc

Recruiting

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Pyruvate Dehydrogenase Complex Deficiency

United States
Washington University School of Medicine
National Cancer Institute (NCI); The Foundation for Barnes-Jewish Hospital

Recruiting

Washington University Participant Engagement and Cancer Genomic Sequencing Center (WU-PE-CGS)

Multiple Myeloma | Rectal Cancer | Cholangiocarcinoma | Colon Cancer

United States
Rett Syndrome Research Trust

Not yet recruiting

Validation of Innovative Biosensors for Rett Autonomic Symptom Tracking (VIBRANT)

Rett Syndrome

United States

Search clinical trials for: Genetic lipids disorders

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