Search clinical trials for: Genetic Disease

Total 382693 results

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

Not yet recruiting

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

Healthy Volunteers Sickle Cell Disease, Thalassemia

United States
Karolinska University Hospital
University Hospital, Linkoeping

Recruiting

Responders and Non-responders in the Management of Heart Failure - Significance of Genetic Influence and Identification of Novel Informative Biomarkers (Responders)

Heart Failure | Pathophysiology

Sweden
Institut Pasteur

Not yet recruiting

Genetic Determinants of the Antiviral Immune Response in Oceanian Populations (GEDIPOP)

Virus

New Caledonia
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
Firalis SA

Recruiting

A Prospective, Monocentric Clinical Study for the Validation of in Vitro Diagnostic Tests Developed by Firalis (ApoDiag)

Genetic Health Risks

France
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
Hookipa Biotech GmbH

Not yet recruiting

Safety, Reactogenicity and Immunogenicity of HB-502 and HB-501 Versus Placebo in People With HIV on Suppressive ART

Human Immunodeficiency Virus (HIV) Infection

United States
UMC Utrecht
Radboud University Medical Center; University Medical Center Groningen; Amsterdam...

Recruiting

The Electronic Cardiovascular Genetics (eCG) Clinic for Presymptomatic Genetic Counselling (eCG)

Inherited Cardiac Disease

Netherlands
Hospices Civils de Lyon

Not yet recruiting

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

Wilson Disease

France
Novartis Pharmaceuticals

Not yet recruiting

Clinical Study of Asciminib in Previously Treated Indian Patients With Ph+ CML-CP Without T315I Mutation and in Patients With Ph+ CML-CP With T315I Mutation (ASC4INDIA)

Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic Phase
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
University of Southern California
National Cancer Institute (NCI)

Not yet recruiting

Access to Genetic Testing in Underserved Patients With Cancer

Malignant Solid Neoplasm | Breast Carcinoma | Metastatic Prostate Carcinoma | Ovarian Carcinoma | Triple-Negative Breast Carcinoma | Male Breast Carcinoma | Pancreatic Exocrine Neoplasm | Stage IVB Prostate Cancer American Joint Committee on Cancer v8

United States
Rocket Pharmaceuticals Inc.

Not yet recruiting

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency

Spain, United States
Amryt Pharma
Chiesi Farmaceutici S.p.A.

Not yet recruiting

A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez (FOSteR)

Epidermolysis Bullosa, Junctional | Epidermolysis Bullosa, Dystrophic
Takeda

Recruiting

A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)

United Kingdom, Denmark
AbbVie

Not yet recruiting

An Observational Study to Assess Real-World Use of Upadacitinib Tablets in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis in China (REACH-AD)

Atopic Dermatitis
Leiden University Medical Center
The Dutch Brain Foundation (funding)

Not yet recruiting

Stimulating Amyloid Clearance in Cerebral Amyloid Angiopathy (Clear-Brain)

Cerebral Amyloid Angiopathy
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)
Gattaca Genomics

Recruiting

Embryo Assessment Utilizing Timelapse Imaging in Conjunction With Preimplantation Genetic Testing for Aneuploidy With Next Generation Sequencing

Infertility | Aneuploidy | Infertility, Female | Infertility, Male | IVF

United States
Assistance Publique - Hôpitaux de Paris
Institut Pasteur

Recruiting

Physiology and Pathologies Linked to Human Splenic Function : Direct and Ex-vivo Perfusion Explorations (SPLEENVIVO)

Splenectomy

France
University of Haifa
Bnai Zion Medical Center; Clalit Health Services, Haifa and West Galilee

Not yet recruiting

Risk and Clinical Consequences of Low Count Monoclonal B-cell Lymphocytosis (LC MBL) (MBL RiskConseq)

Cardiovascular Diseases | Infections | Autoimmune Diseases | Parkinson Disease | Dementia | Alzheimer Disease | Chronic Lymphocytic Leukemia | Monoclonal B-Cell Lymphocytosis | Cancers
Instituto Nacional de Investigacao em Saude, Angola
FUNDAÇAO CALOUSTE GULBENKIAN; FUNDACIÓN BANCARIA CAIXA D'ESTALVIS I PENSIONS... and other collaborators

Recruiting

LEARNER- Low dosE AspiRiN prEterm tRial (Angola) (LEARNER)

Pregnancy Complications | Pregnancy Related | Sickle Cell Disease | Pre-Eclampsia

Angola
Sanofi
Regeneron Pharmaceuticals

Not yet recruiting

Study in Paediatric Patients With Atopic Dermatitis Treated With Dupilumab in Spain (PROADAP)

Atopic Dermatitis
Ospedale San Raffaele

Recruiting

Genetics in the Progression of Nephropathies

Hypertension | AKI | CKD | Hematologic Malignancy | Ckd-Mbd | ADPKD | Bone Marrow Transplant Complications | CKD (Chronic Kidney Disease) Stage 5D

Italy
AstraZeneca

Recruiting

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia (LOCUS)

Carpal Tunnel Syndrome | Amyloidosis | Polyneuropathy | Hereditary Transthyretin Amyloidosis

Russian Federation
Ultragenyx Pharmaceutical Inc

Not yet recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

United States, Australia, Canada, Germany, United Kingdom, Israel, France, Spain
Poitiers University Hospital

Recruiting

Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington's Disease (MUSIC-HD) (MUSIC-HD)

Huntington Disease

France
Jiangsu HengRui Medicine Co., Ltd.

Not yet recruiting

Clinical Trial of HR091506 Tablets in Treatment of Gout With Hyperuricemia in Adults

Gout With Hyperuricemia in Adults
Spinogenix
Avance Clinical Pty Ltd.

Not yet recruiting

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

United States
Children's Hospital Los Angeles

Not yet recruiting

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Cystic Fibrosis | Constipation Chronic Idiopathic

United States
IRCCS Ospedale San Raffaele

Not yet recruiting

Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies

Rare Diseases | Corneal Neovascularization | Limbal Stem Cell Deficiency | Aniridia | Neurotrophic Keratopathy | Ocular Cicatricial Pemphigoid | Ocular Graft-versus-host Disease | EEC Syndrome
Ospedale San Raffaele
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Recruiting

Identification of New Biological Markers for the Progression of Mycobacterium Abscessus-induced Lung Disease in Cystic Fibrosis (BioMol-MA)

Non-Tuberculous Mycobacterial Pneumonia | Cystic Fibrosis Lung

Italy
All India Institute of Medical Sciences, Bhubaneswar

Not yet recruiting

Study on Efficacy of Add on Selenium in Mild-to-moderate Graves Ophthalmopathy

Thyroid Eye Disease | Graves Ophthalmopathy

India
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
Avidity Biosciences, Inc.

Not yet recruiting

Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Myotonic Dystrophy | Myotonic Disorders | Myotonia | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert Disease | Steinert
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain
Nido Biosciences, Inc.

Recruiting

A Study of NIDO-361 in Patients With SBMA (PIONEER KD)

Spinal and Bulbar Muscular Atrophy | Kennedy's Disease

Korea, Republic of, Denmark, Italy, United Kingdom
Johns Hopkins University
National Center for Complementary and Integrative Health (NCCIH); Bowdoin College

Recruiting

Remote Delivery of a Mindfulness-based Intervention for Tics (MBIT)

Tourette Syndrome | Tic Disorders | Tourette's Disorder | Persistent Tic Disorder | Chronic Motor Tic Disorder | Chronic Vocal Tic Disorder | Persistent Motor Tic Disorder | Persistent Vocal Tic Disorder

United States
Riphah International University

Not yet recruiting

Effects of Lower Limb Fitness Program in Children With DS

Down Syndrome

Pakistan
Sanofi

Recruiting

A Study to Evaluate the Treatment Response and Safety of Two Dose Regimens of Subcutaneous Amlitelimab Monotherapy Compared With Treatment Withdrawal in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (ESTUARY)

Dermatitis Atopic

United States

Search clinical trials for: Genetic Disease

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