Search clinical trials for: Genetic diseases, inborn

Total 18695 results

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
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
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
Anhui Medical University

Not yet recruiting

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

Mitochondrial Diseases
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
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

Not yet 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
The University of Hong Kong

Not yet recruiting

Evaluation of Efficacy and Safety of Enhanced External Counter-pulsation on Patients With Atopic Dermatitis and Inflammatory Skin Disease Requiring Wet Wrap Therapy

Atopic Dermatitis | Inflammatory Skin Disease
Lancashire Teaching Hospitals NHS Foundation Trust
Duchenne UK

Recruiting

Hydrotherapy in Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United Kingdom
Dacima Consulting
Société Tunisienne D'endocrinologie De Diabétologie Et Des Maladies Métaboliques

Not yet recruiting

Tunisian Clinical Registry on Hypoparathyroidism and Pseudo-hypoparathyroidism. (THYPARS)

Hypoparathyroidism | Pseudo Hypoparathyroidism

Tunisia
Ascidian Therapeutics, Inc

Recruiting

Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials

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

United States
Eli Lilly and Company

Not yet recruiting

Lebrikizumab Pen Ease of Use in Participants With Atopic Dermatitis

Atopic Dermatitis

United States
University Hospital, Angers

Not yet recruiting

Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD (FibroTG-HD)

Huntington Disease

France
Rofaida Hassan Ahmed

Not yet recruiting

EKLF Gene Expression in β-thalassemia

β-thalassemia
St. Jude Children's Research Hospital
BioLineRx, Ltd.

Not yet recruiting

SCD Stem Cell Mobilization and Apheresis Using Motixafortide

Sickle Cell Disease

United States
Arthrosi Therapeutics

Recruiting

Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients

Hyperuricemia | Gout | Arthritis, Gouty | Gout Chronic

United States, Australia, New Zealand
Novartis Pharmaceuticals

Not yet recruiting

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (SPARKLE)

Sickle Cell Disease
Atlantic Health System
Regeneron Pharmaceuticals

Not yet recruiting

Atlantic Lipid Lowering Treatment Optimization Program (ALLTOP)

Familial Hypercholesterolemia | High Density Lipoprotein Deficiency | Lipoprotein Types--Lp System Lp(A) Hyperlipoproteinemia | Apolipoprotein B 100, Familial Defective | Low-Density-Lipoid-Type Hyperlipoproteinemia

United States
argenx

Not yet recruiting

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

Congenital Myasthenic Syndrome
Splice Bio

Recruiting

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene (POLARIS)

Stargardt Disease | Stargardt's Disease | STGD1 | Stargardt
Apollo Therapeutics Ltd

Recruiting

A Study of the Effects of Camoteskimab in Adults With Moderate to Severe Atopic Dermatitis

Dermatitis | Eczema | Atopic Dermatitis | Atopic | Eczema, Atopic | Dermatologic Disease | Eczema Atopic Dermatitis

United States, Canada
Universitätsklinikum Hamburg-Eppendorf

Recruiting

Cold Atmospheric Plasma for the Endoscopic Treatment of Duodenal Polyps in Patients With Familial Adenomatous Polyposis (coldAPC)

Familial Adenomatous Polyposis | Adenomatous Polyposis Coli | Duodenal Adenoma

Germany
Cantonal Hospital Graubuenden
University of Zurich

Not yet recruiting

Short-term Effects of an SGLT2 Inhibitor on Divalent Ions in Autosomal Dominant Polycystic Kidney Disease (SIDIA)

Autosomal Dominant Polycystic Kidney Disease

Switzerland
Hospices Civils de Lyon

Not yet recruiting

Biocollection of Rare Pediatric-onset of Autoimmune and Autoinflammatory Diseases (GENIALII)

Autoimmune Diseases | Genetic Disease | Autoinflammatory Disease | Systemic Lupus

France
Novo Nordisk A/S

Recruiting

A Research Study of the Effect of Food on Etavopivat in Healthy Participants

Healthy Volunteers Sickle Cell Disease, Thalassemia

United States
Assiut University

Not yet recruiting

Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients

EKLF Mutations in Beta Thalassemia Patients
Ascendis Pharma Growth Disorders A/S

Not yet recruiting

A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (COACH)

Achondroplasia

Denmark, Ireland, United Kingdom
Ionis Pharmaceuticals, Inc.

Not yet recruiting

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Hospices Civils de Lyon

Not yet recruiting

Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients (VARCUWIC)

Wilson Disease

France
SpliSense Ltd.

Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis

Cystic Fibrosis

United States
University of North Carolina, Chapel Hill
North Carolina Translational and Clinical Sciences Institute

Not yet recruiting

PeLear CCC: Proyecto Latino Contra Cancer Colorectal (PeLear CCC)

Colorectal Cancer | Rectal Cancer | Rectal Neoplasms | Colon Cancer | Colon Adenocarcinoma | Colon Rectal Cancer | Hereditary Nonpolyposis Colon Cancer

United States
University of Cologne
The Clinical Trials Centre Cologne; ESCRS (European Society of Cataract and...

Not yet recruiting

Trial Comparing Cataract Surgery With Triple-DMEK in Patients With Cataract and Fuchs Endothelial Corneal Dystrophy

Cataract Surgery | Cataract and Fuchs Endothelial Corneal Dystrophy

Denmark, Germany, Netherlands, Spain
University of Mons

Recruiting

Quantitative Assessment of Orofacial Muscle Function in FSHD

Facioscapulohumeral Muscular Dystrophy

Belgium

Search clinical trials for: Genetic diseases, inborn

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