Search clinical trials for: Genetic disease

Total 384039 results

argenx

Recruiting

A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease. (UplighTED)

Thyroid Eye Disease

United States
Forge Biologics, Inc

Not yet recruiting

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Krabbe Disease

United States
BioMarin Pharmaceutical

Recruiting

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Phenylketonuria (PKU)

United States
Rady Pediatric Genomics & Systems Medicine Institute

Recruiting

An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing (BeginNGS)

Genetic Disease

United States
Halic University

Recruiting

Effect of Parental Attitude on Functional and Physical Level of Children With Cystic Fibrosis

Cystic Fibrosis | Cystic Fibrosis in Children

Turkey
Peking University Cancer Hospital & Institute

Not yet recruiting

Effectiveness of Risk-based Sequential Screening for Esophageal and Gastric Cancer (ERSS-EGC)

Gastric Cancer | Esophageal Cancer | Risk Stratification | Precision Screening | Sequential Screening

China
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Biomarkers in Retinitis Pigmentosa (RPMARKER)

Retinitis Pigmentosa

Italy
Massachusetts General Hospital
Johns Hopkins University

Not yet recruiting

Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)

Neurofibromatosis 1 | Neurofibromas, Cutaneous

United States
Inozyme Pharma
GACI Global

Not yet recruiting

PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

ATP-Binding Cassette Subfamily C Member 6 Deficiency | Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency
Bioray Laboratories
First Affiliated Hospital of Guangxi Medical University

Not yet recruiting

Clinical Study of BRL-101 in Severe SCD

Sickle Cell Disease

China
Children's Hospital Medical Center, Cincinnati
Makerere University; Ministry of Health, Uganda; Mulago Hospital, Uganda

Recruiting

Uganda Sickle Surveillance Study (US-3)

Sickle Cell Disease

Uganda
University of Michigan

Recruiting

Michigan Food and Atopic Dermatitis (M-FAD) Program - Molecular Analytics Project

Healthy | Atopic Dermatitis | Food Allergy

United States
Marco Capogrosso
Roche-Genentech

Not yet recruiting

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

Spinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy 4

United States
Kind Pharmaceuticals LLC

Not yet recruiting

A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia

β -Thalassemia
Hôpital Necker-Enfants Malades

Not yet recruiting

Assessment of Patients Not Concluded After Neonatal Screening of Cystic Fibrosis.

CFTR-RD
Bangabandhu Sheikh Mujib Medical University, Dhaka...

Recruiting

Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial

Transfusion-dependent Thalassemia

Bangladesh
Region Stockholm
Karolinska Institutet

Recruiting

CFTR Modulators in Pregnancy and Postpartum

Cystic Fibrosis

Sweden
PTC Therapeutics

Recruiting

A Study of Sepiapterin in Participants With Phenylketonuria (PKU) (EPIPHENY)

Phenylketonuria

United States
ARTHEx Biotech S.L.

Recruiting

Study of ATX-01 in Participants With DM1 (ArthemiR)

Myotonic Dystrophy 1

United States
Shanghai Institute Of Biological Products
The First Affiliated Hospital of Henan University of Science and Technology; First Affiliated Hospital Bengbu Medical College and other collaborators

Recruiting

A Phase Ib Clinical Trial of Peguricase for Injection With Methotrexate in Patients With Uncontrolled Gout.

Gout

China
metaX Institut fuer Diatetik GmbH
Great Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...

Not yet recruiting

Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies (ZeroMinisMR)

Alkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
Chia Tai Tianqing Pharmaceutical Group Nanjing...

Not yet recruiting

A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

Hemophilia A

China
Bioray Laboratories
Xiangya Hospital of Central South University; Sun Yat-Sen Memorial Hospital... and other collaborators

Not yet recruiting

Long-term Follow-up Study of BRL-101 for TDT

Thalassemia, Beta

China
GENGE

Recruiting

GENETIC VARIABILITY TO GLP1 TREATMENT (GLP1 treatment)

Obesity | Type 2 Diabetes

Israel
Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
John Elfar

Not yet recruiting

4AP to Delay Carpal Tunnel Release (CTR) (CTR)

Carpal Tunnel Syndrome | Compression Neuropathy
Lise Aksglæde

Not yet recruiting

Klinefelter Syndrome and Testosterone Treatment in Puberty (TiPY)

Klinefelter Syndrome
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI); Cystic Fibrosis Foundation

Recruiting

Health Outcomes of Parents With Cystic Fibrosis-Aim 2 (HOPeCF)

Cystic Fibrosis | Parenthood Status

United States
Istituto Auxologico Italiano

Recruiting

Short Stature and Psychological Well-being (PSICOSHORT)

Short Stature | Short; Stature, Psychosocial

Italy
Assiut University

Not yet recruiting

Relation Between Nail Fold Capillaroscopy, Optical Coherence Tomography Angiography, and Femoral Vein Wall Thickness in Behçet's Disease Relation Between Nail Fold Capillaroscopy, Optical Coherence Tomography Angiography, and Femoral Vein Wall Thickness in Behçet's Disease

Behçet's Disease
Istituto Auxologico Italiano

Recruiting

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

Prader-Willi Syndrome

Italy
Istituto Auxologico Italiano

Recruiting

Biological Age in Children With GH Deficiency Undergoing Hormone Replacement Therapy (ETABIOGHD)

Growth Hormone Deficiency | Short Stature

Italy
IRCCS Fondazione Stella Maris

Recruiting

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

Fragile X Syndrome

Italy
Baylor College of Medicine
National Eye Institute (NEI); National Institutes of Health (NIH)

Recruiting

Microphthalmia, Anophthalmia, and Coloboma Genetic Epidemiology in Children (MAGIC)

Coloboma | Microphthalmia | Anophthalmia

United States
Zhongmou Therapeutics

Not yet recruiting

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

Retinitis Pigmentosa

China
University of Évora
Universidade Nova de Lisboa; Comprehensive Health Research Centre; Unidade Local... and other collaborators

Recruiting

Effects of Vitamin D on Health Promotion During Pregnancy and Its Impact on Prematurity-Related Outcome (VitDTracking)

Vitamin D Deficiency | Pregnancy

Portugal
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)

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
Eli Lilly and Company

Recruiting

A Study to Investigate the Effect of Lepodisiran on the Reduction of Major Adverse Cardiovascular Events in Adults With Elevated Lipoprotein(a) - ACCLAIM-Lp(a)

Elevated Lp(a) | Atherosclerotic Cardiovascular Disease (ASCVD)

United States, Australia, Spain, Canada, Argentina, China, Italy, France, Japan, Taiwan, Korea, Republic of, Denmark, Germany, Hungary, Israel, Austria, Belgium, Netherlands, Brazil, Mexico, Romania, United Kingdom, Greece, Czechia, Poland and more
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
Australasian Leukaemia and Lymphoma Group

Not yet recruiting

Epcoritamab (Epcor)-Containing Combination Salvage Therapy Followed by ASCT & Epcor Consolidation in Patients With Relapsed LBCL

High-grade B-cell Lymphoma | DLBCL - Diffuse Large B Cell Lymphoma | High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements | DLBCL, Nos Genetic Subtypes | High Grade B-Cell Lymphoma, Not Otherwise Specified | Follicular Large Cell Lymphoma, Relapsed | Follicular Large Cell Lymphoma
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Recruiting

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

Hemophilia

China

Search clinical trials for: Genetic disease

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