Search clinical trials for: Genetic Diseases, Inborn

Total 18740 results

IRCCS Fondazione Stella Maris

Recruiting

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

Fragile X Syndrome

Italy
Zhongmou Therapeutics

Not yet recruiting

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients (ZM-02)

Retinitis Pigmentosa

China
Suzhou Municipal Hospital

Not yet recruiting

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

Familial Hypercholesterolemia | Refractory Hypercholesterolemia
ViGeneron GmbH

Recruiting

Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

Retinitis Pigmentosa

Germany
Halic University

Recruiting

Development,Validity and Reliability of a Caregiver Version of the Breathlessness Beliefs Questionnaire

Cystic Fibrosis | Parents

Turkey
CorrectSequence Therapeutics Co., Ltd

Recruiting

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

Beta-Thalassemia Major

China
Steen Hvitfeldt Poulsen
Novo Nordisk A/S

Recruiting

Phenotyping and Characterization of wtATTR-CM (TRACE 1)

Quality of Life | Transthyretin Amyloidosis | Transthyretin Amyloid Cardiomyopathy | Wild-Type Transthyretin-Related (ATTR)Amyloidosis

Denmark
Children's Hospital Medical Center, Cincinnati
Children's Hospital of Philadelphia; Emory University; Nemours Children's Health...

Not yet recruiting

A Socio-ecological Approach for Improving Self-management in Adolescents With SCD (SC-Thrive)

Anemia, Sickle Cell
Rege Nephro Co., Ltd.

Recruiting

Study of Tamibarotene in Patients With ADPKD

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Japan
University Hospital, Rouen

Not yet recruiting

Safety of RotigotiNe in Patients With Autosomal Dominant Polycystic Kidney Disease (ETERNAL-PKD)

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

Recruiting

Vasodilator and Exercise Study for DMD (VASO-REx)

Muscular Dystrophy | Exercise | Duchenne Muscular Dystrophy | Vasodilation | DMD | Muscular Dystrophy in Children | Duchenne Disease

United States
South Egypt Cancer Institute
Assiut University; Shefa Al-orman Children Cancer Hospital

Recruiting

Study of Wilms Tumors Among Patients of Shefa Al-Orman Children Cancer Hospital

Wilms Tumor

Egypt
Ondokuz Mayıs University

Recruiting

The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence

Quality of Life | Urinary Incontinence | Menopause | Wolman Disease | Psychological Well-Being

Turkey
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Recruiting

Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Hemophilia

China
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience. (ANNPHE)

Phenylketonuria

France
UMC Utrecht
Alfasigma S.p.A.; ReumaNederland; Autoimmune Research and Collaboration Hub

Recruiting

Drug Rediscovery for Rare Immune Mediated Inflammatory Diseases (DRIMID)

IgG4-related Disease | Idiopathic Inflammatory Myopathies | Behcet's Disease

Netherlands
Bioray Laboratories
Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of...

Not yet recruiting

Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease

Sickle Cell Disease
Agios Pharmaceuticals, Inc.

Not yet recruiting

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

Sickle Cell Disease | Nephropathy
Oslo University Hospital
Norwegian National Advisory Unit on Rare Disorders, Oslo University Hospital

Recruiting

Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis

Cystic Fibrosis in Children

Norway
Bioray Laboratories
Nanfang Hospital, Southern Medical University

Not yet recruiting

Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease

Sickle Cell Disease
Kirsehir Ahi Evran Universitesi
TC Erciyes University

Not yet recruiting

An Intervention-Based Approach to Strengthen the Psychological Health of Children With Cystic Fibrosis and Their Mothers

Children | Cystic Fibrosis | Lung Functions | Mothers | Laughter Yoga | Laughter Therapy | Resilience,Psychological | Psychological Health

Turkey
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
Perha Pharmaceuticals
Göteborg University; Proteas Bioanalytics

Recruiting

Phosphoproteomic Profile of Children With Down Syndrome (PEPS)

Down Syndrome

France
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, Turkey, United Kingdom, Italy
Kanglin Biotechnology (Hangzhou) Co., Ltd.

Not yet recruiting

A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants

Transfusion-dependent Beta-Thalassemia

China
Meyer Children's Hospital IRCCS

Recruiting

Transcranial Magnetic Stimulation (TMS) in Genetic Epilepsies

Epilepsy | Genetic Disease

Italy
Assiut University

Not yet recruiting

Vascular Affection in Behcet Disease

Behcet Disease and Vascular Involvement
Eli Lilly and Company

Recruiting

A Study of Lebrikizumab (LY3650150) With/Without Topical Corticosteroid Treatment in Participants With Moderate-to-Severe Atopic Dermatitis

Atopic Dermatitis

China, Korea, Republic of
Keymed Biosciences Co.Ltd

Not yet recruiting

Study of CM310 Injection in Adolescent Subjects With Atopic Dermatis

Atopic Dermatitis

China
University Hospital, Toulouse

Not yet recruiting

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

Prader-Willi Syndrome

France
Lynk Pharmaceuticals Co., Ltd

Recruiting

A Phase 3 Study of LNK01001 Capsule in Subjects With Moderate to Severe Atopic Dermatitis.

Atopic Dermatitis

China
Pilar Azevedo
Queen's University, Belfast; Medical University Innsbruck; Cystic Fibrosis Foundation and other collaborators

Recruiting

COVID-19 Antibody Responses In Cystic Fibrosis (CAR-CF)

COVID-19 | Cystic Fibrosis

Portugal
Novilla Pharmaceuticals

Not yet recruiting

Treatment of Topical Ketorolac Gel in Acute Gouty Flare

Acute Pain | Gout Flare | Acute Gouty Arthritis

Australia
Walter Reed National Military Medical Center

Recruiting

The Effect of Teprotumumab on Thyroid Eye Disease and Thyroid Dysfunction (Teprotumumab)

Thyroid Eye Disease | Graves Ophthalmopathy | Graves Disease

United States
Radboud University Medical Center
Enzyre B.V.

Recruiting

Version Testing of EnzySystem Version A for Hemophilia A

Hemophilia A

Netherlands
Novartis Pharmaceuticals

Recruiting

Specified Drug-use Survey of Leqvio for s.c. Injection.

Hypercholesterolaemia | Familial Hypercholesterolaemia

Japan
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

Search clinical trials for: Genetic Diseases, Inborn

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