Search clinical trials for: Genetic Disease, Inborn

Total 18696 results

Assiut University

Not yet recruiting

Serum Elafin and Vascular Affection in Behcet Disease

Behcet Disease and Vascular Affection
Pole Sante Grace de Dieu

Recruiting

French Validation of the AdT-Physio Scale (Trans-AdT)

Cystic Fibrosis

France
Xinhua Hospital, Shanghai Jiao Tong University...
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Type II Gaucher Disease

China
Nordlandssykehuset HF
University of Oslo; Norwegian University of Science and Technology; The University... and other collaborators

Recruiting

Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment

Porphyria, Acute Intermittent

Norway
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Beta-Thalassemia

United States
Karolinska Institutet

Recruiting

Internet-based Behavior Therapy for Adults With Tourette Syndrome (TICNET)

Tourette Syndrome | Chronic Tic Disorder

Sweden
Sunshine Guojian Pharmaceutical (Shanghai) Co.,...

Recruiting

A Study of SSGJ-613 in Gout Subjects Initiating Urate-Lowering Treatment.

Gout Arthritis

China
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
Abyss Ingredients
Eurofins

Recruiting

Efficacy of Marine By-product Hydrolysate on the Reduction of Atopic Dermatitis Symptoms

Eczema | Atopic Dermatitis

Spain
Investigación en Hemofilia y Fisioterapia

Recruiting

Conditioned Pain Modulation in Patients With Hemophilia

Hemophilia

Spain
S.LAB (SOLOWAYS)
Center of New Medical Technologies

Recruiting

Evaluation of a Genetically Determined Personalized Approach in Prescribing Biologically Active Substances in Patients With Elevated Blood Homocysteine Levels. (PERBIO-HC)

Hyperhomocysteinemia

Russian Federation
Nationwide Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health... and other collaborators

Not yet recruiting

Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness (ADHERE)

Sickle Cell Disease

United States
Investigación en Hemofilia y Fisioterapia

Recruiting

Blood Flow Restriction in Improving Muscle Strength of Patients With Hemophilic Ankle Arthropathy

Hemophilia

Spain
University of California, San Francisco
Johns Hopkins University; Baylor College of Medicine; Children's Hospital of... and other collaborators

Not yet recruiting

Zinc Supplementation in Sickle Cell Disease (ZnSCD)

Sickle Cell Disease

United States
Central Hospital, Nancy, France

Recruiting

Cognitive Disorders in Hereditary Spastic Paraplegia Type 4 (SPG-TEP)

Spastic Paraplegia

France
Université du Québec à Chicoutimi
McGill University; Université de Sherbrooke; University of Alberta; University... and other collaborators

Recruiting

Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias

Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay | Spastic Paraplegia 7

Canada
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
Massachusetts General Hospital
Patient-Centered Outcomes Research Institute

Not yet recruiting

A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

Neurofibromatosis 1

United States
University of Miami
Florida lions eye bank

Not yet recruiting

Plasma Rich in Growth Factors in Corneal Endothelial Transplantation

Corneal Edema | Fuchs' Endothelial 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 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
Chung-Hsing Wang
Sanofi

Not yet recruiting

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Gaucher Disease

Taiwan
Incyte Corporation

Recruiting

Study to Compare Maternal, Fetal, and Infant Outcomes of Women With Mild to Moderate Atopic Dermatitis Exposed to Ruxolitinib Cream During Pregnancy With an Unexposed Control Population (OPERA-1)

Pregnancy Related | Atopic Dermatitis

United States
University Hospital, Antwerp
University Hospital, Ghent

Recruiting

Genome-wide Epistasis for Cardiovascular Severity in Marfan Study (GEMS)

Marfan Syndrome | FBN1 Mutation

Belgium
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
PTC Therapeutics

Recruiting

An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

Huntington Disease

Australia, France, Germany, Netherlands, United Kingdom, Spain, Austria, Canada, Italy, New Zealand
Hospices Civils de Lyon

Recruiting

Muscle Function and Physical Activity in the Modern Era of Cystic Fibrosis (MUCOMUSCLE)

Cystic Fibrosis

France
Hospital Universitari Vall d'Hebron Research Institute
ERN-EuroBloodNet (European Reference Network on Rare Hematological Diseases

Recruiting

European Rare Blood Disorders Platform (ENROL) (ENROL)

Leukemia | Anemia | Iron Metabolism Disorders | Anemia, Sickle Cell | Thalassemia | Bleeding Disorder | Myeloma | Lymphoid Neoplasm | Blood Cancer | Bone Marrow Failure | Red Cell Membrane and Enzyme Abnormalities | Myeloma, Malignant

Spain
Jimma University

Recruiting

Bromocriptine in Dilated Cardiomyopathy Among Women of Reproductive Age

Dilated Cardiomyopathy

Ethiopia
King's College London
King's College Hospital NHS Trust; Guy's and St Thomas' NHS Foundation Trust; British Academy and other collaborators

Not yet recruiting

Support Strategies for Parents During the First Year Following Their Child's Diagnosis of Sickle Cell Disorder

Sickle Cell Disease
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
Bristol-Myers Squibb

Recruiting

A Study to Evaluate the Safety, Tolerability, Drug Levels, and Drug Effects of BMS-986326 in Participants With Atopic Dermatitis

Dermatitis, Atopic

France, Czechia, Germany, Poland, Spain
Amgen

Recruiting

A Trial to Investigate Teprotumumab Subcutaneous Administration Compared With Placebo in Male and Female Adult Participants With Moderate-to-severe Active Thyroid Eye Disease

Thyroid Eye Disease

United States
Travere Therapeutics, Inc.

Recruiting

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment (HARMONY)

Homocystinuria

United States
Sarepta Therapeutics, Inc.

Recruiting

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)

Limb-girdle Muscular Dystrophy

United States
UNICANCER
Servier

Not yet recruiting

Safety of Trifluridine/Tipiracil in Patients With Dihydropyrimidine Dehydrogenase Deficiency Diagnosed With Metastatic Colorectal or Gastroesophageal Cancer (TRIFLUOX-DP)

Metastatic Colorectal Cancer | Metastatic Gastroesophageal Adenocarcinoma | DPD Deficiency
Wake Forest University Health Sciences

Not yet recruiting

Topical Treatment for Atopic Dermatitis

Atopic Dermatitis

United States
Sanofi

Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab on Background Topical Corticosteroids Therapy in Participants Aged 12 Years and Older With Moderate-to-severe AD Who Have Had an Inadequate Response to Prior Biologic Therapy or an Oral JAK Inhibitor (AQUA)

Dermatitis Atopic

Chile, United Kingdom, United States, Canada, Turkey, Israel, Korea, Republic of
Mahidol University

Recruiting

Safety and Efficacy of Stem Cell Small Extracellular Vesicles in Patients With Retinitis Pigmentosa

Retinitis Pigmentosa

Thailand
medac GmbH

Not yet recruiting

Efficacy and Safety of Methotrexate Versus Placebo in Adults With Atopic Dermatitis.

Moderate to Severe Atopic Dermatitis
Institute of Hematology & Blood Diseases Hospital...

Not yet recruiting

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Hemophilia A

China
Incyte Corporation

Recruiting

A Study to Evaluate the Efficacy, and Safety Study of Ruxolitinib Cream in Adults With Moderate Atopic Dermatitis (TRuE-AD4)

Atopic Dermatitis

Germany, Poland, United States, Spain, United Kingdom, Italy, Belgium, Canada, Netherlands, Australia, Bulgaria, France, Switzerland, Hungary, Korea, Republic of
Rolfs Consulting und Verwaltungs-GmbH (RCV)
Rhythm Pharmaceuticals, Inc.

Recruiting

Early Genetic Identification of Obesity (WEGIO)

Cognitive Impairment | Hyperphagia | Obesity, Childhood | Bardet-Biedl Syndrome | Polydactyly | Retinopathy | Syndactyly | POMC Deficiency

Germany
ReCode Therapeutics

Recruiting

A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants

Cystic Fibrosis

New Zealand
Tongji Hospital
Wuhan University; Wuhan Central Hospital; Wuhan Fourth Hospital

Recruiting

The Effects of Sirolimus in Patients With Dilated Cardiomyopathy Infected With Kaposi Sarcoma-associated Virus (SDCMK)

Dilated Cardiomyopathy | Kaposi's Sarcoma-Associated Herpesvirus Infection

China
Imagine Institute

Recruiting

Genotype-phenotype Characterization Study on Genetic Diseases With Immune and Neurological Dysfunctions (IFN)

Autoimmune Diseases | Genetic Diseases | Neurological Disease | Immune Dysfunction

France
Eye & ENT Hospital of Fudan University
Shanghai Rehabilitation Institute for the Exceptional Children

Recruiting

A Comparative Analysis of Speech Perception Between Cochlear Implant Patients and DFNB9 Patients Receiving Gene Therapy

Hearing Loss | Genetic Disease | Speech Perception

China
Assiut University

Not yet recruiting

Evaluation of the Corneal Endothelial Cell Layer by Specular Microscopy in Patients With Thyroid Eye Disease

Thyroid Eye Disease
Chinese University of Hong Kong

Recruiting

A Pilot Study of Efficacy and Safety of SIM05 in Adults With Atopic Eczema

Atopic Eczema

Hong Kong

Search clinical trials for: Genetic Disease, Inborn

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