Search clinical trials for: Genetic Disorders

Total 154695 results

Ascendis Pharma Growth Disorders A/S

Not yet recruiting

A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (COACH)

Achondroplasia

Denmark, Ireland, United Kingdom
Firalis SA

Recruiting

A Prospective, Monocentric Clinical Study for the Validation of in Vitro Diagnostic Tests Developed by Firalis (ApoDiag)

Genetic Health Risks

France
Ionis Pharmaceuticals, Inc.

Not yet recruiting

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Hospices Civils de Lyon

Not yet recruiting

Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients (VARCUWIC)

Wilson Disease

France
Novartis Pharmaceuticals

Not yet recruiting

Clinical Study of Asciminib in Previously Treated Indian Patients With Ph+ CML-CP Without T315I Mutation and in Patients With Ph+ CML-CP With T315I Mutation (ASC4INDIA)

Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic Phase
University of North Carolina, Chapel Hill
North Carolina Translational and Clinical Sciences Institute

Not yet recruiting

PeLear CCC: Proyecto Latino Contra Cancer Colorectal (PeLear CCC)

Colorectal Cancer | Rectal Cancer | Rectal Neoplasms | Colon Cancer | Colon Adenocarcinoma | Colon Rectal Cancer | Hereditary Nonpolyposis Colon Cancer

United States
University of Mons

Recruiting

Quantitative Assessment of Orofacial Muscle Function in FSHD

Facioscapulohumeral Muscular Dystrophy

Belgium
Leiden University Medical Center
The Dutch Brain Foundation (funding)

Not yet recruiting

Stimulating Amyloid Clearance in Cerebral Amyloid Angiopathy (Clear-Brain)

Cerebral Amyloid Angiopathy
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)
University of Haifa
Bnai Zion Medical Center; Clalit Health Services, Haifa and West Galilee

Not yet recruiting

Risk and Clinical Consequences of Low Count Monoclonal B-cell Lymphocytosis (LC MBL) (MBL RiskConseq)

Cardiovascular Diseases | Infections | Autoimmune Diseases | Parkinson Disease | Dementia | Alzheimer Disease | Chronic Lymphocytic Leukemia | Monoclonal B-Cell Lymphocytosis | Cancers
AstraZeneca

Recruiting

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia (LOCUS)

Carpal Tunnel Syndrome | Amyloidosis | Polyneuropathy | Hereditary Transthyretin Amyloidosis

Russian Federation
Ultragenyx Pharmaceutical Inc

Not yet recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

United States, Australia, Canada, Germany, United Kingdom, Israel, France, Spain
Poitiers University Hospital

Recruiting

Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington's Disease (MUSIC-HD) (MUSIC-HD)

Huntington Disease

France
Spinogenix
Avance Clinical Pty Ltd.

Not yet recruiting

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

United States
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
Avidity Biosciences, Inc.

Not yet recruiting

Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Myotonic Dystrophy | Myotonic Disorders | Myotonia | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert Disease | Steinert
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain
Nido Biosciences, Inc.

Recruiting

A Study of NIDO-361 in Patients With SBMA (PIONEER KD)

Spinal and Bulbar Muscular Atrophy | Kennedy's Disease

Korea, Republic of, Denmark, Italy, United Kingdom
Johns Hopkins University
National Center for Complementary and Integrative Health (NCCIH); Bowdoin College

Recruiting

Remote Delivery of a Mindfulness-based Intervention for Tics (MBIT)

Tourette Syndrome | Tic Disorders | Tourette's Disorder | Persistent Tic Disorder | Chronic Motor Tic Disorder | Chronic Vocal Tic Disorder | Persistent Motor Tic Disorder | Persistent Vocal Tic Disorder

United States
Riphah International University

Not yet recruiting

Effects of Lower Limb Fitness Program in Children With DS

Down Syndrome

Pakistan
Tisento Therapeutics

Not yet recruiting

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

United States, Australia, Italy, Germany, Canada, United Kingdom
Başak Çağla Arslan
Hacettepe University

Recruiting

Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy | Occupational Therapy | Gamification

Turkey
Assiut University

Not yet recruiting

The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy

Limb-girdle Muscular Dystrophy
M.D. Anderson Cancer Center
Ascentage Pharma Group Inc.

Not yet recruiting

A Phase I Study of Decitabine, Lisaftoclax, and Olverembatinib in Patients With Advanced Chronic Myeloid Leukemia and Philadelphia Chromosome-Positive Acute Myeloid Leukemia

Advanced Chronic Myeloid Leukemia | Philadelphia Chromosome-Positive Acute Myeloid Leukemia

United States
Istanbul University - Cerrahpasa (IUC)

Not yet recruiting

Comparing Efficacies of Median Nerve Hydrodissection With Dexamethasone and Dextrose in Carpal Tunnel Syndrome

Carpal Tunnel Syndrome | Entrapment Neuropathy | Median Nerve Entrapment

Turkey
CSL Behring

Not yet recruiting

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Hemophilia B
Vanderbilt University Medical Center
Aminu Kano Teaching Hospital; Murtala Muhammed Specialist Hospital

Not yet recruiting

Prevention of Malnutrition in Children With Sickle Cell Disease (PMC-SCD)

Malnutrition | Sickle Cell Disease
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
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
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
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
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

Search clinical trials for: Genetic Disorders

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