Search clinical trials for: genetic

Total 18467 results

Shanghai Ninth People's Hospital Affiliated to...

Recruiting

A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)

Neurofibromatosis 1 | Plexiform Neurofibroma

China
The First Affiliated Hospital of Zhejiang Chinese...

Recruiting

Peripheral Mechanisms of Humoral Immune Effector-mediated Tourette's Syndrome and the Role of Tuina Intervention

Inflammatory Response | Tourette Syndrome in Children | Food Intolerance

China
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
Imagine Institute

Not yet recruiting

Developmental and Epileptic Encephalopathy of Genetic Etiology: Natural History Through Reuse of Clinical Data (DEE-RETRO)

Developmental and Epileptic Encephalopathy
IRCCS Eugenio Medea

Recruiting

Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe] (MDCJ-NeuBeRe)

Myotonic Dystrophy 1

Italy
University of Pennsylvania

Not yet recruiting

Using the EHR to Advance Genomic Medicine Across a Diverse Health System

Parkinson Disease | Alzheimer Disease | ALS | Polyneuropathies | Frontotemporal Dementia | Pheochromocytoma | Thoracic Aortic Aneurysm | Paraganglioma | Genetic Predisposition | Cardiomyopathy Non-ischemic
Assiut University

Not yet recruiting

Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia

B Thalassemia
IRCCS Eugenio Medea

Recruiting

Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI] (RIMUDI)

Muscular Dystrophies | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy | Facio-Scapulo-Humeral Dystrophy

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

Not yet recruiting

Efficacy and Safety of Telitacicept in the Treatment of Systemic Sclerosis

Diffuse Cutaneous Systemic Sclerosis

China
Ray Therapeutics, Inc.
The Vision Research and Assessment Institute (VRAI)

Recruiting

Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Retinitis Pigmentosa Patients

Retinitis Pigmentosa

United States
Clinic for Special Children

Recruiting

WiTNNess - TNNT1 Myopathy Natural History Study (WiTNNess)

Myopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANM

United States
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain (PRESENCE)

Pain | Sickle Cell Disease
Apollo Therapeutics Ltd

Not yet recruiting

A Study to Investigate APL-4098 Alone and/or in Combination With Azacitidine in R/R AML and High-Risk MDS

Myelodysplastic Syndrome With Excess Blasts | Acute Myeloid Leukemia, in Relapse | Acute Myeloid Leukemia Refractory | Myelodysplastic Syndrome Acute Myeloid Leukemia
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Next-Generation alzheImer'S Therapeutics (ENERGISE)

Alzheimer Disease

Italy
Enzyre B.V.
Indiana Hemophilia & Thrombosis Center

Not yet recruiting

Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia A Monitoring

Hemophilia A

United States
Chugai Pharmaceutical

Not yet recruiting

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS) | Immune Thrombocytopenia (ITP) | Dermatomyositis (DM) | Bullous Pemphigoid (BP) | Behçet's Syndrome (BS) | Immune-mediated Necrotizing Myopathy (IMNM)

United States, Japan
Sensorion

Not yet recruiting

A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (AUDIOGENE)

Otorhinolaryngologic Diseases | Ear Diseases | Hearing Disorders | Deafness | Hearing Loss, Sensorineural | Congenital Deafness | DFNB9 | OTOF Gene Mutation

France
Hospices Civils de Lyon

Not yet recruiting

Circadian Rhythm Disorders in Children With Cystic Fibrosis Under CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators (CHRONO-MUCO)

Cystic Fibrosis

France
Massachusetts General Hospital
n-Lorem Foundation

Not yet recruiting

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Genetic Disease
Centre Hospitalier Universitaire de la Réunion

Recruiting

Pathogenicity of Species of the Achromobacter Genus in Patients From Reunion Island With Cystic Fibrosis (ACHROMO-MUCO)

Cystic Fibrosis

Réunion
Eilean Therapeutics

Not yet recruiting

Dose Escalation and Expansion Study to Evaluate the Safety, PK, PD and Efficacy of ZE46-0134 in Adults With FLT3 Mutated Relapsed or Refractory Acute Myeloid Leukemia

AML With Gene Mutations

Australia
Medical University of Warsaw

Recruiting

Tocilizumab in Corticosteroid-Resistant Graves' Orbitopathy (Thyroid Eye Disease)

Thyroid Eye Disease | Ophthalmopathy | Orbitopathy, Graves

Poland
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

phenotypeS in Non Ambulant Duchenne Muscular Dystrophy (GUP21003)

Duchenne Muscular Dystrophy | Natural History | Motor Function; Retardation

Italy
Harmony Biosciences, LLC

Recruiting

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

School Readiness Intervention for Preschool Children With Sickle Cell Disease

Sickle Cell Disease

United States
Instituto de Investigación Hospital Universitario...

Recruiting

Optimization of Atopic Dermatitis Treatment That Requires Second-line Systemic Therapy Through Predictive Models

Atopic Dermatitis

Spain
University of Kansas Medical Center

Not yet recruiting

Targeting Inflammation With Losartan to Improve Response to Modulator Therapy in Cystic Fibrosis.

Cystic Fibrosis

United States
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Dan Bing

Not yet recruiting

Genetic Feature of Congenital Hearing Loss in Chinese Population

Congenital Hearing Loss | Congenital Deafness
AstraZeneca

Recruiting

hATTR Polyneuropathy in Russia (PRIMER)

Hereditary Transthyretin Amyloidosis

Russian Federation
National Cancer Institute (NCI)

Not yet recruiting

Anti-CD19 Chimeric Antigen Receptor T-Cell Immunotherapy for Chronic Lymphocytic Leukemia (CLL)

Leukemia, Lymphocytic, Chronic, B-Cell | B-Cell Chronic Lymphocytic Leukemia | B-Lymphocytic Leukemia, Chronic

United States
Editas Medicine, Inc.

Not yet recruiting

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Sickle Cell Disease | Hemoglobinopathies | Transfusion-dependent Beta-Thalassemia
Beijing Tiantan Hospital
West China Hospital; The First Affiliated Hospital of Zhengzhou University; Capital... and other collaborators

Recruiting

Evaluate the Effectiveness and Safety of Raman IVD Analyzer in the Molecular Diagnosis of Gliomas During Surgery

Glioma

China
Octapharma

Not yet recruiting

Phase 3 Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema
University Hospital, Toulouse

Not yet recruiting

Efficacy of Injectable Gentamicin in Hereditary Ichthyosis (GENTIC)

Ichthyosis

France
West China Hospital

Not yet recruiting

AI App for Management of Atopic Dermatitis

Artificial Intelligence | Atopic Dermatitis
Chang Gung University

Recruiting

Electrical Stimulation Cycling Training Effects on SCA

Spinocerebellar Ataxia (SCA)

Taiwan
University of Malaga

Not yet recruiting

Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. (DMDrespy2024)

Muscular Dystrophy | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Spain
Fondazione SISA (Societa Italiana per lo Studio...

Recruiting

Lipid Transport Disorder Italian Genetic Record (LIPIGEN) (LIPIGEN)

Familial Hypercholesterolemia | Genetic Disorder

Italy
Seoul National University Hospital

Not yet recruiting

The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne | Upper Extremity | Orthotic Devices

Korea, Republic of
Technoderma Medicines Inc.
Therapeutics, Inc.

Not yet recruiting

Study of TDM-180935 in Atopic Dermatitis Patients

Atopic Dermatitis

United States
AstraZeneca

Not yet recruiting

Real-World Treatment Study of Koselugo (Selumetinib)

Neurofibromatosis 1 | Neurofibroma, Plexiform
Cliniques universitaires Saint-Luc- Université...

Recruiting

Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases (BUSARD)

Rare Diseases | Inborn Errors of Metabolism

Belgium
Xuanwu Hospital, Beijing

Not yet recruiting

Exploring DBS Efficacy in Treatment-refractory Tourette's Syndrome

Tourette's Syndrome

China
Alnylam Pharmaceuticals

Not yet recruiting

Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

Hereditary Amyloidosis, Transthyretin-Related | Asymptomatic Carrier State

France
Peking University Third Hospital

Recruiting

Exploring the Value of 18F-AlF-FAPI PET/CT in Assessing the Activity of Thyroid Eye Disease

Thyroid Eye Disease

China
Children's National Research Institute
The Hospital for Sick Children; Ann & Robert H Lurie Children's Hospital of... and other collaborators

Recruiting

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell AntibodY (SUN-RAY)

Sickle Cell Disease

United States
Rigshospitalet, Denmark

Not yet recruiting

Preimplantation Genetic Testing for Aneuploidy (PGT-A) in Women Aged 37-41 Years

Infertility

Denmark
Azienda Ospedaliera Universitaria Integrata Verona
Abbott Medical Devices

Recruiting

Microvascular Dysfunction Assessment to Predict Left Ventricular Reverse Remodeling (MICROREV)

Heart Failure | Left Ventricular Dysfunction | Idiopathic Dilated Cardiomyopathy

Italy
Assistance Publique - Hôpitaux de Paris
Vaincre la Mucoviscidose

Not yet recruiting

Oral Health Status of Cystic Fibrosis Patients. An Online Survey in Collaboration With the Vaincre la Mucoviscidose Patient Association. (E-MUCODENT)

Cystic Fibrosis

France

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