Search clinical trials for: Genetic Diseases, Inborn

Total 18528 results

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
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
Apyx Medical

Recruiting

Histological Evaluation of Human Skin Bx to Assess the Effects of APR Tx as an Adjunct Procedure in Facelift Surgery

Skin Laxity | Tissue Breakdown | Collagen Degeneration | Collagen Shrinkage | Tissue Degeneration

United States
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

United States, Chile, Canada, United Kingdom
Mahidol University

Not yet 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

Not yet 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
Riphah International University

Recruiting

Effects of Maze Activities in Down's Syndrome

Down Syndrome

Pakistan
Chinese University of Hong Kong

Recruiting

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

Atopic Eczema

Hong Kong
Mahidol University

Recruiting

Efficacy and Safety of Fractional 1064-nm Picosecond Laser for Facial Skin Tightening

Laxity; Skin

Thailand
Centre Hospitalier Universitaire de Liege
Centre Hospitalier Régional de la Citadelle; SYSNAV

Recruiting

Natural History Study for Patients With Angelman Syndrome (NatHisAngelman)

Angelman Syndrome

Belgium
Children's Hospital Medical Center, Cincinnati
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders (ENTRAIN)

Autism Spectrum Disorder | Autistic Disorder | Fragile X Syndrome | Asperger Syndrome

United States
Amgen

Recruiting

A Trial to Investigate the Non-inferiority of Pegloticase Administered Every 4 Weeks (Q4W) With MTX Compared With Every 2 Weeks (Q2W) With MTX in Participants With Uncontrolled Refractory Gout

Gout

United States
Swedish Orphan Biovitrum

Recruiting

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Hereditary Tyrosinemia, Type I

China
Hospices Civils de Lyon

Recruiting

Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France. (DAILY-LUMA)

Patients With PH1 Treated With Lumasiran in France

France
University of Cambridge
Sanofi

Recruiting

Molecular Imaging in Fabry Disease of the Heart

Fabry Disease, Cardiac Variant

United Kingdom
Radboud University Medical Center
Solve FSHD

Recruiting

Magnetic Resonance Imaging and Ultrasound Comparison With Load Evaluation (MUSCLE+)

Muscular Dystrophy, Facioscapulohumeral

Netherlands
Lassen Therapeutics Inc.

Recruiting

A Study to Evaluate the Efficacy and Safety of LASN01 in Patients With Thyroid Eye Disease

Eye Diseases | Endocrine System Diseases | Thyroid Eye Disease | Graves Ophthalmopathy | Orbital Diseases | Thyroid Associated Ophthalmopathy | Graves Orbitopathy | Proptosis

United States, United Kingdom, Spain
Papworth Hospital NHS Foundation Trust
Innovate UK; Cystic Fibrosis Trust; US Cystic Fibrosis Foundation

Recruiting

Analysis of Remote Monitoring/Virtual Clinic Data in Adult Patients With Cystic Fibrosis (Project Breathe)

Cystic Fibrosis

United Kingdom
Sardocor Corp.

Not yet recruiting

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (MUSIC-DMD)

DMD-Associated Dilated Cardiomyopathy
BioMarin Pharmaceutical

Recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

Cerebral Adrenoleukodystrophy (CALD)

United States
Sanofi

Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis on Background Topical Corticosteroids (SHORE)

Dermatitis Atopic

Japan, Chile, Canada, United States
Bayer

Recruiting

A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A

Hemophilia A | Prophylaxis of Bleeding | Treatment of Bleeding

Korea, Republic of
Amgen

Recruiting

A Study With Self-administered Rocatinlimab in Adolescent and Adult Participants With Moderate-to-severe AD (ROCKET-Outpost)

Atopic Dermatitis

United States
University College, London

Not yet recruiting

Coloured-light in Retinitis Pigmentosa. (CLIRP)

Retinitis Pigmentosa

United Kingdom
Centre Hospitalier Universitaire de Nice

Recruiting

Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1 (REINFORCE)

Facioscapulohumeral Muscular Dystrophy 1

France, Canada
National Cancer Institute (NCI)

Not yet recruiting

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

Neurofibromatosis 1 | Nerve Sheath Neoplasms

United States
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

Glutaric Acidemia Type I | Glutaric Aciduria Type I

China
Samsung Medical Center

Not yet recruiting

Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E

Charcot-Marie-Tooth Disease, Type 1
Institute of Hematology & Blood Diseases Hospital...
Kanglin Biotech

Recruiting

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Transfusion-dependent Beta-Thalassemia

China
Boston University Charles River Campus
Boston Children's Hospital

Not yet recruiting

Harness-based Mobility Intervention for Infants With Down Syndrome

Down Syndrome

United States
Tampere University Hospital
Tampere University

Recruiting

Urothelial Cancer Screening in Individuals With Lynch Syndrome Using a Urine Tumor DNA Panel (LS-URO Study)

Lynch Syndrome | Urothelial Carcinoma

Canada, Finland
Genrix (Shanghai) Biopharmaceutical Co., Ltd.

Not yet recruiting

Phase III Study of GR1802 Injection in Patients With Atopic Dermatitis

Atopic Dermatitis

China
Boston Children's Hospital
Cystic Fibrosis Foundation

Recruiting

Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis

Cystic Fibrosis

United States
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Early-goal Directed Automated Red Blood Cell Exchange for Acute Chest Syndrome in Sickle Cell Disease (ARCAD)

Acute Chest Syndrome

France
Luye Pharma Group Ltd.

Recruiting

Safety, Tolerability and Pharmacokinetic of Multiple-ascending Doses of LPM3770164 in Healthy Subjects

Tardive Dyskinesia | Huntington Disease

China
University of Pennsylvania

Recruiting

LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study (LCAT NHS)

LCAT Deficiency

United States
Assistance Publique Hopitaux De Marseille
Aix Marseille Université; ALPHENYX

Not yet recruiting

Involvement of Pollutants in Atopic Dermatitis and Psoriasis (DIAhR)

Psoriasis | Atopic Dermatitis
Hospices Civils de Lyon

Recruiting

EVALUATION OF THE OVERALL HAEMOSTATIC CAPACITY OF MIM8 WITH GLOBAL HAEMOSTASIS ASSAYS AND FIBRIN CLOT ULTRASTRUCTURE (Mim8-TGT)

Hemophilia A

France
Rocket Pharmaceuticals Inc.

Recruiting

Danon Disease Natural History Study

Danon Disease

United States, Germany
BioMarin Pharmaceutical

Recruiting

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia

United States, Canada, France, Australia, Japan, Argentina, Brazil, Germany, Italy, Spain

Search clinical trials for: Genetic Diseases, Inborn

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