Search clinical trials for: Genetic causes

Total 18970 results

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, United States, Germany
University Hospital, Limoges
National Research Agency, France; Institut du Cerveau et de la Moelle épinière and other collaborators

Not yet recruiting

French-German Cohort Study to Determine Factors Associated With Weight Loss in Amyotrophic Lateral Sclerosis (FG-CoALS)

Amyotrophic Lateral Sclerosis

France
NYU Langone Health

Not yet recruiting

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
Perminder Sachdev
Melbourne Health; Royal Brisbane and Women's Hospital; The University of Queensland and other collaborators

Recruiting

AusCADASIL: An Australian Cohort of CADASIL

Cadasil

Australia
ModernaTX, Inc.

Recruiting

A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

Phenylketonuria

United States, Australia
Ad scientiam
Vertex Pharmaceuticals Incorporated

Not yet recruiting

Performances and Safety of MuCopilot, a Digital Tool for the Unsupervised Objective Assessment of Cystic Fibrosis (MuControl)

Cystic Fibrosis

France
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
Ganzhou Hemay Pharmaceutical Co., Ltd

Recruiting

A Study of Efficacy and Safety of Hemay005 Tablets in Patients With Behçet's Disease

Behçet's Disease

China
Sanofi

Recruiting

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Hemophilia A

Taiwan
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function

Sickle Cell Disease

United States
TESS Research Foundation
Brown University; Stanford University; University of Texas Southwestern Medical...

Recruiting

SLC13A5 Deficiency Natural History Study - United States Only

Epilepsy | Movement Disorders | Rare Diseases | Genetic Disorder | Citrate Transporter Deficiency | SLC13A5 Deficiency | EIEE25 | Kohlschutter-Tonz Syndrome (Non-ROGDI) | Citrate Transporter Disorder | DEE25

United States
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)

Spain, United States, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
Hillhurst Biopharmaceuticals, Inc.
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)

Anemia, Sickle Cell

United States
Kiminori Kimura, MD
Japan Agency for Medical Research and Development

Recruiting

A Phase 2 Trial of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia (OP-724-H201)

Liver Cirrhosis

Japan
Foundation for Prader-Willi Research
RTI International

Recruiting

A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS (VNS4PWS)

Prader-Willi Syndrome

United States
The Republican Research and Practical Center for...
Belarusian State Medical University

Recruiting

To Develop an Algorithm for Predicting the Unfavorable Course of Sepsis in Children Based on a Comprehensive Assessment of Immunological, Biochemical and Molecular Genetic Markers

Sepsis

Belarus
Genomic Prediction Inc.
Boston IVF

Recruiting

GEnetic Testing in Elective Single Embryo Transfer (GETSET): A Randomized Clinical Trial (GETSET)

Infertility, Female

United States
IRCCS San Raffaele

Not yet recruiting

WEST-KOaST Study: WES Analysis of Testicular Cancer Patientsand Their First-degree Family Members (West-KOaSt)

Testicular Cancer
Jiangsu Gensciences lnc.

Recruiting

Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein

Severe Hemophilia A

China
Jiangsu HengRui Medicine Co., Ltd.

Not yet recruiting

Clinical Study of HR091506 Tablets in Treatment of Gout With Hyperuricemia in Adults

Gout With Hyperuricemia in Adults
University Hospital, Clermont-Ferrand

Not yet recruiting

Epidemiology of Assertiveness and Emotion Management Disorders in People With Tourette's Syndrome (AFFIRMATICS)

Tourette Syndrome

France
Assistance Publique - Hôpitaux de Paris

Recruiting

Peripheral Serotonin and Albinism (SEPIAs)

Oculocutaneous Albinism

France
PYC Therapeutics

Recruiting

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

Optic Atrophies, Hereditary | Autosomal Dominant Optic Atrophy | Optic Atrophy, Autosomal Dominant | Kjer Optic Atrophy

Australia
Jiangsu Gensciences lnc.

Not yet recruiting

Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A

Severe Hemophilia A

China
Azitra Inc.

Recruiting

Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome

Netherton Syndrome

United States
Centre Hospitalier Universitaire de Nice

Not yet recruiting

A 44-week Monocentric Open Study Assessing the Efficacy and Safety of Deucravacitinib in Adults With Inflammatory Genodermatoses (GENEPID)

Inflammatory Epidermal Genodermatoses

France
Duke University
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

Predictors of Pain in Sickle Cell Disease

Chronic Pain | Sickle Cell Disease
Solid Biosciences Inc.

Recruiting

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Duchenne Muscular Dystrophy

United States
Nektar Therapeutics

Recruiting

A Phase 2b Study to Evaluate Rezpegaldesleukin (Rezpeg) in the Treatment of Adult Patients With Moderate-to-Severe Atopic Dermatitis (REZOLVE-AD)

Moderate to Severe Atopic Dermatitis

Spain, Czechia, United States, Australia, Croatia, Bulgaria, Poland, Canada, Germany, Hungary
Arrowhead Pharmaceuticals

Recruiting

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Myotonic Dystrophy 1

New Zealand, Australia
Azienda Ospedaliero-Universitaria di Modena

Recruiting

"Iron Overload and Endocrinological Diseases"

Iron Overload | Hemochromatosis | Thalassemia Major | Thalassemia Intermedia | Endocrinopathy

Italy
CHU de Reims

Recruiting

Cystic Fibrosis and Periodontal Diseases (MucoParo)

Periodontitis | Gingivitis | Cystic Fibrosis

France
Radboud University Medical Center

Recruiting

A 5-year Natural History Study in LAMA2-related Muscular Dystrophy and SELENON-related Myopathy.

SELENON-related Myopathy | LAMA2-related Muscular Dystrophy

Netherlands
Ad scientiam

Not yet recruiting

A Usability Validation of the MuCopilot Mobile Application, a Digital Tool for the Unsupervised Objective Assessment of Cystic Fibrosis (MuControl-Sum)

Cystic Fibrosis

France
Massachusetts General Hospital
Johns Hopkins University

Recruiting

Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas

Neurofibromatosis 1

United States
The University of Hong Kong

Recruiting

Root Caries Prevention Using Fluoride Varnish Fortified With SBGC (SBGC)

Root Caries | Dental Caries of Root Surface

Hong Kong
Arrowhead Pharmaceuticals

Recruiting

Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Facio-Scapulo-Humeral Dystrophy

New Zealand
McMaster University

Not yet recruiting

Nutritional Therapy in Late-onset Pompe Disease (PDT-MIS)

Obesity | Lysosomal Storage Diseases | Muscle Loss | Glycogen Storage Disease Type II | Pompe Disease | Nutrition Poor | Glycogen Storage Disease Type II, Adult | Glycogen Storage Disease Type II Late Onset
Sanofi

Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (COAST 1)

Dermatitis Atopic

Israel, Chile, Australia, United States, France, Canada, Germany, Korea, Republic of, Taiwan, China, Brazil, Poland
Seagen Inc.
Sanofi

Recruiting

A Study of SGN-CEACAM5C in Adults With Advanced Solid Tumors

Stomach Neoplasms | Carcinoma, Non-Small-Cell Lung | Small Cell Lung Carcinoma | Colorectal Neoplasms | Pancreatic Ductal Adenocarcinoma | Gastroesophageal Junction Adenocarcinoma

United States, Canada
Intellia Therapeutics
Regeneron Pharmaceuticals

Recruiting

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, United Kingdom, New Zealand, Canada
NYU Langone Health

Recruiting

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
Hoffmann-La Roche
Sarepta Therapeutics, Inc.

Recruiting

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Duchenne Muscular Dystrophy

Spain, Germany, Italy
Nadir Hastalıkları Araştırma Derneği

Recruiting

A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofuxinosis Type 2 (CLN2) (ENIGMA)

Neuronal Ceroid Lipofuxinosis Type2 (CLN2)

Turkey
Martin Dichgans

Recruiting

High Frequency Imaging in Cerebral Amyloid Angiopathy (HIFI-CAA)

Cerebral Amyloid Angiopathy

Germany
Hacettepe University

Not yet recruiting

Development a Core Set for Adults With Cystic Fibrosis Based on the ICF

Cystic Fibrosis

Turkey
Innovent Biologics (Suzhou) Co. Ltd.

Recruiting

A Multicenter, Open-label Extension Study Evaluating the Efficacy and Safety of IBI311 in Subjects With Thyroid Eye Disease

Thyroid Eye Disease, TED

China
First Affiliated Hospital Xi'an Jiaotong University

Recruiting

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

China
National Cancer Institute (NCI)

Not yet recruiting

A Study Comparing the Combination of Dasatinib and Chemotherapy Treatment With or Without Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or Philadelphia Chromosome-Like (Ph-Like) ABL-Class B-Cell Acute Lymphoblastic Leukemia (B-ALL)

B Acute Lymphoblastic Leukemia

Search clinical trials for: Genetic causes

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