Search clinical trials for: Genetic Disease, Inborn

Total 18726 results

Medibiofarma S.L.

Recruiting

Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients

Huntington Disease

Spain
Ikaria Bioscience Pty Ltd

Not yet recruiting

Study of RN0361 in Adult Subjects

Hypertriglyceridemia | Familial Chylomicronemia

Australia
Concerto Biosciences

Not yet recruiting

Topical ENS-002 for Atopic Dermatitis in Adults (EnSync)

Atopic Dermatitis | Atopic Dermatitis Eczema

United States
Kors van der Ent
European Union

Recruiting

Clinical Trial to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Adults With CF (CHOICES)

Cystic Fibrosis

Netherlands, Spain, France, Germany, Italy, United Kingdom, Portugal, Belgium, Sweden
Ascidian Therapeutics, Inc

Recruiting

Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR) (STELLAR)

Stargardt Disease | Stargardt Disease 1 | Juvenile Macular Degeneration | Cone Rod Dystrophy

United States
Peter Timoney
Inflammasome Therapeutics

Not yet recruiting

Evaluation of K9 in Subjects With Thyroid Eye Disease (TED)

Thyroid Eye Disease

United States
Guangdong Hengrui Pharmaceutical Co., Ltd

Not yet recruiting

Efficacy and Safety Study of SHR-1819 Injection in Adult Patients With Severe Atopic Dermatitis

Moderate to Severe Atopic Dermatitis

China
Ahram Canadian University

Recruiting

Inspiratory Muscle Training in Children With Beta Thalasemia

Beta Thalassemia Major

Egypt
Second Affiliated Hospital, School of Medicine,...

Not yet recruiting

Efficacy and Safety of Dupilumab in Combination With Tofacitinib in Moderate to Severe Adult AD Patients

Atopic Dermatitis
Asociación Española para el Estudio del Hígado

Recruiting

Spanish Wilson Disease Registry

Wilson Disease

Spain
KalVista Pharmaceuticals, Ltd.

Not yet recruiting

Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II (KONFIDENT-KID)

Hereditary Angioedema
Shanghai BDgene Co., Ltd.

Recruiting

A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major

β-thalassemia

China
Stefano Vicari
Italian Institute of Technology (IIT)

Recruiting

Efficacy of Bumetanide to Improve Cognitive Functions in Down Syndrome

Down Syndrome

Italy
Emory University
The Marcus Foundation

Not yet recruiting

A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)

Dilated Cardiomyopathy

United States
Xuanwu Hospital, Beijing

Not yet recruiting

Forel's Field Electrical Stimulation for Lennox-Gastaut Syndrome (FESL)

Lennox Gastaut Syndrome

China
University Hospital, Strasbourg, France

Recruiting

Optimizing the Management of Sickle Cell Patients on Hydroxyurea: The Value of Therapeutic Pharmacological Monitoring (OPTIMDREP)

Sickle Cell Disease

France
University of Pennsylvania

Not yet recruiting

Investigation of Filaggrin Gene Mutations Among Latinx Patients With Atopic Dermatitis

Eczema | Atopic Dermatitis
Biocodex
Exystat

Not yet recruiting

Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3 (CRYSTAL)

Primary Hyperoxaluria Type 3 | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1
Inozyme Pharma

Not yet recruiting

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Pseudoxanthoma Elasticum | Arterial Calcification | Gene Mutations | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | Autosomal Recessive Hypophosphatemic Rickets Type 2
AbbVie

Not yet recruiting

A Study to Assess Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Children From 2 to Less Than 12 Years of Age With Moderate to Severe Atopic Dermatitis (Start Up)

Atopic Dermatitis
Sahlgrenska University Hospital, Sweden

Not yet recruiting

HIBOC = Hepatic Imaging Biomarkers in Obese Children

Fibrosis, Liver | Steatosis | Elastography | Diagnosis | Child Obesity | Metabolic Disease | Shear Wave Elastography | Shear Wave Dispersion | Attenuation Imaging | Metabolic and Genetic Disorder Affecting the Liver | Inflammation; Liver
First Affiliated Hospital Bengbu Medical College

Recruiting

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Familial Hypercholesterolemia

China
Acibadem University
Boston Children's Hospital; Ege University

Recruiting

PTEN Hamartoma Tumor Syndrome Pediatric Patient Registry

PTEN Hamartoma Tumor Syndrome | Macrocephaly Autism Syndrome

Turkey
Riphah International University

Not yet recruiting

Effects of Cuevas Medek Exercises on Trunk Control and Balance in Children With Down Syndrome

Down Syndrome

Pakistan
Takeda

Recruiting

A Survey of Susoctocog Alfa (Genetical Recombination) in Participants With Acquired Haemophilia A

Acquired Hemophilia A

Japan
Shanghai Yueyang Integrated Medicine Hospital

Not yet recruiting

Clinical Study of Trilinolein Cream for Prevention of Relapse in Remission of Moderate-to-severe Atopic Dermatitis.

Atopic Dermatitis
Riphah International University

Not yet recruiting

Effect of Group-Task-Oriented Training on GMFM and ADL in Children With DS

Downs Syndrome

Pakistan
Ray Therapeutics, Inc.

Not yet recruiting

Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa Patients

Retinitis Pigmentosa

United States
Vertex Pharmaceuticals Incorporated

Not yet recruiting

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

Cystic Fibrosis
PYC Therapeutics

Not yet recruiting

SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

Autosomal Dominant Optic Atrophy | Kjer Optic Atrophy | OPA1 Gene Mutation | Hereditary Optic Atrophies
University Hospital, Tours
Fondation Ildys

Not yet recruiting

Impact of a Coordinated Dietetic-adapted Physical Activity Program on the Percentage of Lean Body Mass in Adults With Cystic Fibrosis Treated With Elexacaftor-Tezacaftor-Ivacaftor: Multicentre Randomised Controlled Trial (DIAPASOM)

Cystic Fibrosis

France
RenJi Hospital

Recruiting

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Familial Hypercholesterolemia

China
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

MYCobiome Analysis in Atopic Dermatitis With Head and Neck Involvement (MYCAH)

Atopic Dermatitis

France
PYC Therapeutics

Not yet recruiting

MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)

Eye Diseases | Retinal Degeneration | Retinal Dystrophies | Retinal Disease | Retinitis Pigmentosa 11

United States
National University of Singapore
National University Hospital, Singapore; Ministry of Education, Singapore

Not yet recruiting

Impact of Regular Consumption of Eggs and Nutrients Fortified Eggs on Eczema Condition in Singapore Individuals

Diet Modification | Aging | Atopic Dermatitis Eczema
Ataturk University

Recruiting

Thrombophilia and Thrombosis in Behçet's Disease

Behçet Disease

Turkey
Changchun GeneScience Pharmaceutical Co., Ltd.

Recruiting

A Bioequivalence Study of Two Different Dosage Form of Genakumab

Gouty Arthritis (GA)

China
Khondrion BV

Not yet recruiting

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases (KH176-301)

Mitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
Dermal Laboratories Ltd

Not yet recruiting

A Study to Evaluate the Emollient Performance of Doublebase Once in the Treatment of Atopic Eczema

Atopic Eczema
Jaeb Center for Health Research
Massachusetts General Hospital; Cystic Fibrosis Foundation; Beta Bionics, Inc.

Not yet recruiting

Bionic Pancreas in CFRD

Cystic Fibrosis-related Diabetes
Anhui Medical University

Not yet recruiting

Establishment of Reproductive Cohort and Prediction Model of Genetic Counseling for Mitochondrial Genetic Diseases

Mitochondrial Diseases
Hoffmann-La Roche

Not yet recruiting

An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (SHIELD DMD)

Duchenne Muscular Dystrophy

United States
Arizona State University
American Speech-Language-Hearing Foundation

Recruiting

Proactive Speech and Language Intervention for Infants With Down Syndrome (BBC-DS)

Down Syndrome

United States
Hacettepe University

Recruiting

Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAH

Quality of Life | Congenital Adrenal Hyperplasia | Physical Activity | Exercise Capacity

Turkey
London North West Healthcare NHS Trust

Recruiting

PSYLIVED: the Psychological Impacts of Living With an Inherited Colorectal Cancer Predisposition Syndrome (PSYLIVED)

Lynch Syndrome | Polyposis Syndrome, Hereditary Mixed, 1 | Polyposis Syndrome, Hereditary Mixed, 2

United Kingdom
University of Central Florida
University of South Florida

Not yet recruiting

Gut Microbiomes in HD

Huntington Disease

United States
ActiGraph L.L.C

Recruiting

Analytical Validation of Wrist-Based Assessment of Nocturnal Scratch in Atopic Dermatitis and Psoriasis (DECODE-Scratch)

Psoriasis | Atopic Dermatitis

France
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Primary Immunodeficiencies and Obstetrical Neuraxial Anaesthesia (ANEU-DIP)

Infectious Complications (Epidural Abscess or Meningitis) | Obstetrical Neuraxial Anaesthesia

France
GlaxoSmithKline

Not yet recruiting

Long-Term Study (AtDvance) to Evaluate GSK1070806 in Atopic Dermatitis. (AtDvance)

Dermatitis, Atopic

United States, Germany, Spain, France, Italy, Japan, China, Argentina, Greece, Czechia, Korea, Republic of, Panama, Bulgaria, Canada, Poland, Mexico, Thailand
Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical...

Recruiting

Ultrasound to Assess Sarcopenia in Prader Willi Syndrome (PWS)

Metabolic Syndrome | Sarcopenia | Prader-Willi Syndrome

Taiwan

Search clinical trials for: Genetic Disease, Inborn

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