Search clinical trials for: chromosome 16p11.2

Total 2698 results

Pfizer

Not yet recruiting

A Study to Learn About the Study Medicine Bosulif in Adult Patients With Chronic Myeloid Leukemia(CML).

Chronic Leukemia Myelogenous
Lise Aksglæde

Not yet recruiting

Klinefelter Syndrome and Testosterone Treatment in Puberty (TiPY)

Klinefelter Syndrome
Istituto Auxologico Italiano

Recruiting

Evaluation of the Psychological Profile of Adult Patients With Prader-Willi Syndrome (PROPSICOPWS)

Prader-Willi Syndrome

Italy
IRCCS Fondazione Stella Maris

Recruiting

Optical Imaging as a Tool for Monitoring Brain Function in Fragile X Syndrome

Fragile X Syndrome

Italy
IRCCS Fondazione Stella Maris

Recruiting

Optical Imaging as a Tool for Monitoring Brain Function in Creatine Deficiency Syndromes

Creatine Deficiency, X-linked | Creatine Deficiency Syndrome Due to Gamt Deficiency

Italy
University of Florida
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Not yet recruiting

Vasodilator and Exercise Study for DMD (VASO-REx)

Muscular Dystrophy | Exercise | Duchenne Muscular Dystrophy | Vasodilation | DMD | Muscular Dystrophy in Children | Duchenne Disease
Memorial Sloan Kettering Cancer Center

Recruiting

A Study of 19-28z/IL-18 in People With Acute Lymphoblastic Leukemia (ALL)

Refractory Acute Lymphoblastic Leukemia | Philadelphia-Negative ALL | Philadelphia-Positive ALL | Refractory Acute Lymphoblastic Leukemia (ALL) | Relapsed ALL, Adult | Refractory Acute Lymphoid Leukemia in Relapse

United States
InnoVec Biotherapeutics Inc.
Peking Union Medical College Hospital

Recruiting

Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis

Eye Diseases | Retinal Degeneration | Retinal Disease | Retinoschisis

China
Specila hospital for cerebral palsy and developmental...
FRAXA Research Foundation

Recruiting

Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome

Fragile X Syndrome

Serbia
BioMarin Pharmaceutical

Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Spain, United Kingdom
Perha Pharmaceuticals
Göteborg University; Proteas Bioanalytics

Recruiting

Phosphoproteomic Profile of Children With Down Syndrome (PEPS)

Down Syndrome

France
University Hospital, Toulouse

Not yet recruiting

Adolescent Projections During Transition in Prader-Willi Syndrome (APT-WILL)

Prader-Willi Syndrome

France
UniQure Biopharma B.V.

Recruiting

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Fabry Disease

United States
Sarepta Therapeutics, Inc.

Recruiting

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy (ENDURE)

Duchenne Muscular Dystrophy

United States
Université de Sherbrooke
Canadian Institutes of Health Research (CIHR); Centre de recherche du Centre... and other collaborators

Not yet recruiting

Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals With Fragile-X Syndrome (CANAX)

Fragile X Syndrome
Université de Sherbrooke
Canadian Institutes of Health Research (CIHR); Centre de recherche du Centre... and other collaborators

Not yet recruiting

Effect of CBD on the Brain

Fragile X Syndrome
Brunel University
Royal Free Hospital NHS Foundation Trust

Recruiting

A Co-designed Physical Activity Intervention in Fabry Disease

Fabry Disease

United Kingdom
iECURE, Inc.

Recruiting

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE)

Urea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)

United Kingdom
Hospices Civils de Lyon

Not yet recruiting

Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets FLAM-XLH (FLAM-XLH)

X-Linked Hypophosphatemic Rickets
Riphah International University

Recruiting

Effects of Maze Activities in Down's Syndrome

Down Syndrome

Pakistan
Centre Hospitalier Universitaire de Liege
Centre Hospitalier Régional de la Citadelle; SYSNAV

Recruiting

Natural History Study for Patients With Angelman Syndrome (NatHisAngelman)

Angelman Syndrome

Belgium
Children's Hospital Medical Center, Cincinnati
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders (ENTRAIN)

Autism Spectrum Disorder | Autistic Disorder | Fragile X Syndrome | Asperger Syndrome

United States
University of Cambridge
Sanofi

Recruiting

Molecular Imaging in Fabry Disease of the Heart

Fabry Disease, Cardiac Variant

United Kingdom
Sardocor Corp.

Not yet recruiting

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (MUSIC-DMD)

DMD-Associated Dilated Cardiomyopathy
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

Cerebral Adrenoleukodystrophy (CALD)

United States
Nanfang Hospital, Southern Medical University

Recruiting

A Single-arm, Open-label Study of Olverembatinib, CD3/CD19 Bispecific T-cell Engager, and Chidamide in Patients With Newly Diagnosed Ph+ALL (ABC)

Acute Lymphoblastic Leukemia | Philadelphia Chromosome | Adult ALL | Philadelphia-Positive ALL | IKZF1 Gene Mutation

China
Boston University Charles River Campus
Boston Children's Hospital

Not yet recruiting

Harness-based Mobility Intervention for Infants With Down Syndrome

Down Syndrome

United States
Rocket Pharmaceuticals Inc.

Recruiting

Danon Disease Natural History Study

Danon Disease

United States, Germany
Richard Frye
Autism Discovery and Treatment Foundation

Recruiting

Patterns of Neurodevelopmental Disorders

Epilepsy | Neurodevelopmental Disorders | Autism Spectrum Disorder | Down Syndrome | Mitochondrial Encephalomyopathies | Pediatric Acute-Onset Neuropsychiatric Syndrome | Cerebral Folate Deficiency | Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection

United States
Children's Hospital of Fudan University

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Fabry Disease

China
Perha Pharmaceuticals
Fondation Jérôme Lejeune; European Innovation Council; France 2030 program

Recruiting

Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease (LEUCETTA)

Healthy Volunteers | Down Syndrome | Alzheimer's Disease

France
University Hospital, Strasbourg, France

Recruiting

Identification of Y Chromosome From Free Circulating DNA in Patients With Turner Syndrome (Turner-Ylc)

Turner Syndrome

France
University College Cork

Not yet recruiting

Assessment of Diastolic Function During the Transitional Period and Infancy Using Serial Echocardiography (DiFuSE)

Gestational Diabetes | Neonatal Encephalopathy | Diastolic Dysfunction | Trisomy 21 | Transient Tachypnea of the Newborn | Small for Gestational Age at Delivery

Ireland
Children's Hospital of Fudan University

Recruiting

Esketamine for the Treatment of Rett Syndrome

Rett Syndrome

China
Riphah International University

Recruiting

Action Observation Therapy on Balance in Children With Down Syndrome

Down Syndrome

Pakistan
Riphah International University

Recruiting

Effects of Rhythmic Aerobic Exercises on Functional Balance in Children With Down Syndrome

Down Syndrome

Pakistan
University of Seville

Recruiting

The Coping With and Caring for Infants With Special Needs Intervention in Down Syndrome Infants

Down Syndrome | Physical Disability | Family

Spain
Azienda Ospedaliero-Universitaria di Modena

Recruiting

Role of Cardiac AngioMR in Diagnosis of Cardiac and Vascular Anomalies in Adult Patients With Turner Syndrome

Turner Syndrome | Anomaly Heart

Italy
Minoryx Therapeutics, S.L.

Recruiting

Disease Progression in Women With X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

France
ACADIA Pharmaceuticals Inc.

Recruiting

Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

Hyperphagia in Prader-Willi Syndrome

United States, Canada, United Kingdom
Qilu Hospital of Shandong University

Recruiting

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy (SEARCH)

Fabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular Hypertrophy

China
Gianni Soraru
Mario Negri Institute for Pharmacological Research

Recruiting

A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy (BetaSBMA)

Spinal and Bulbar Muscular Atrophy

Italy
National Institute for Mother and Child Health...
Marie Curie Emergency Children's Hospital, Bucharest; Victor Gomoiu Children...

Recruiting

Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease

Chronic Granulomatous Disease

Romania
Hospices Civils de Lyon

Recruiting

Study of Surgical Practices in Patients With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®) (CHALE)

Hemophilia A | Hemophilia B

France
Changhai Hospital

Not yet recruiting

Analyzing the Urine During BCG Instillation in Bladder Cancer Patients for Disease Followup

Bladder Cancer

China
Taysha Gene Therapies, Inc.

Recruiting

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Rett Syndrome

United States, United Kingdom
Ionis Pharmaceuticals, Inc.

Recruiting

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Pelizaeus-Merzbacher Disease

France, Netherlands
safia Darweesh halwsh

Not yet recruiting

A Comparison of Cognitive-Motor Dual-Task Exercise and Exergaming on Balance, Functional Mobility, and Executive Function in Down Syndrome Children

Down Syndrome

Saudi Arabia
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders (DANNIgene)

Hemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditions

France
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands

Search clinical trials for: chromosome 16p11.2

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