Search clinical trials for: genetic disorders

Total 155433 results

Inozyme Pharma

Not yet recruiting

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Pseudoxanthoma Elasticum | Arterial Calcification | Gene Mutations | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | Autosomal Recessive Hypophosphatemic Rickets Type 2
First Affiliated Hospital Bengbu Medical College

Recruiting

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Familial Hypercholesterolemia

China
Sahlgrenska University Hospital, Sweden

Not yet recruiting

HIBOC = Hepatic Imaging Biomarkers in Obese Children

Fibrosis, Liver | Steatosis | Elastography | Diagnosis | Child Obesity | Metabolic Disease | Shear Wave Elastography | Shear Wave Dispersion | Attenuation Imaging | Metabolic and Genetic Disorder Affecting the Liver | Inflammation; Liver
Acibadem University
Boston Children's Hospital; Ege University

Recruiting

PTEN Hamartoma Tumor Syndrome Pediatric Patient Registry

PTEN Hamartoma Tumor Syndrome | Macrocephaly Autism Syndrome

Turkey
Riphah International University

Not yet recruiting

Effects of Cuevas Medek Exercises on Trunk Control and Balance in Children With Down Syndrome

Down Syndrome

Pakistan
Takeda

Recruiting

A Survey of Susoctocog Alfa (Genetical Recombination) in Participants With Acquired Haemophilia A

Acquired Hemophilia A

Japan
Riphah International University

Not yet recruiting

Effect of Group-Task-Oriented Training on GMFM and ADL in Children With DS

Downs Syndrome

Pakistan
PYC Therapeutics

Not yet recruiting

SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

Autosomal Dominant Optic Atrophy | Kjer Optic Atrophy | OPA1 Gene Mutation | Hereditary Optic Atrophies
University of Monastir

Not yet recruiting

mRNA Expression and Genetic Polymorphisms Affecting DRD3 (rs6280) and HTR2A (rs6313) in Bruxism

Bruxism
RenJi Hospital

Recruiting

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Familial Hypercholesterolemia

China
Unity Health Toronto

Not yet recruiting

The Genetics Navigator: Evaluating a Digital Platform for Genomics Health Services

Connective Tissue Diseases | Neurodevelopmental Disorders | Retinal Disease | Epilepsy in Children | Cardiac Conditions

Canada
Khondrion BV

Not yet recruiting

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases (KH176-301)

Mitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
Hoffmann-La Roche

Not yet recruiting

An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (SHIELD DMD)

Duchenne Muscular Dystrophy

United States
Arizona State University
American Speech-Language-Hearing Foundation

Recruiting

Proactive Speech and Language Intervention for Infants With Down Syndrome (BBC-DS)

Down Syndrome

United States
Hacettepe University

Recruiting

Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAH

Quality of Life | Congenital Adrenal Hyperplasia | Physical Activity | Exercise Capacity

Turkey
London North West Healthcare NHS Trust

Not yet recruiting

PSYLIVED: the Psychological Impacts of Living With an Inherited Colorectal Cancer Predisposition Syndrome (PSYLIVED)

Lynch Syndrome | Polyposis Syndrome, Hereditary Mixed, 1 | Polyposis Syndrome, Hereditary Mixed, 2

United Kingdom
Clinica Universidad de Navarra, Universidad de...
Biobizkaia; Genbioma Aplicaciones S.L.

Not yet recruiting

Effect of a Postbiotic Intake on Glucose Control and Microbiota Composition of Type 2 Diabetic Subjects: a Randomized Controlled Trial. (Diabet2Predict)

Diabetes Mellitus, Type 2

Spain
University of Central Florida
University of South Florida

Not yet recruiting

Gut Microbiomes in HD

Huntington Disease

United States
Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical...

Recruiting

Ultrasound to Assess Sarcopenia in Prader Willi Syndrome (PWS)

Metabolic Syndrome | Sarcopenia | Prader-Willi Syndrome

Taiwan
Lancashire Teaching Hospitals NHS Foundation Trust
Duchenne UK

Recruiting

Hydrotherapy in Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United Kingdom
Dacima Consulting
Société Tunisienne D'endocrinologie De Diabétologie Et Des Maladies Métaboliques

Not yet recruiting

Tunisian Clinical Registry on Hypoparathyroidism and Pseudo-hypoparathyroidism. (THYPARS)

Hypoparathyroidism | Pseudo Hypoparathyroidism

Tunisia
University Hospital, Angers

Not yet recruiting

Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD (FibroTG-HD)

Huntington Disease

France
Nantes University Hospital
University of Angers - Pays de la Loire psychology laboratory

Not yet recruiting

Characterization and Support for Neurodevelopmental Disorders Associated With Congenital Heart Defects (CATAMARAN Ped)

Congenital Heart Defects | Neurodevelopmental Disorder

France
Jay Alberts
National Institutes of Health (NIH)

Not yet recruiting

Genetics and Aerobic Exercise to Slow Parkinson's Disease Trial (GEARS)

Parkinson Disease
Novartis Pharmaceuticals

Not yet recruiting

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (SPARKLE)

Sickle Cell Disease
Atlantic Health System
Regeneron Pharmaceuticals

Not yet recruiting

Atlantic Lipid Lowering Treatment Optimization Program (ALLTOP)

Familial Hypercholesterolemia | High Density Lipoprotein Deficiency | Lipoprotein Types--Lp System Lp(A) Hyperlipoproteinemia | Apolipoprotein B 100, Familial Defective | Low-Density-Lipoid-Type Hyperlipoproteinemia

United States
Firalis SA

Recruiting

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

Genetic Health Risks

France
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
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.

Recruiting

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

United States
Avidity Biosciences, Inc.

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

United States
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain, Canada, France, Germany, Sweden, United Kingdom
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

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

Search clinical trials for: genetic disorders

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