Search clinical trials for: Genetic Disease

Total 380103 results

Natural Resources Institute Finland
Tampere University

Recruiting

Sand Play - the Effect of Biodiversity Exposure on Atopic Dermatitis (BIWE)

Microbial Colonization | Atopic Dermatitis | Immune System and Related Disorders | Nature, Human

Finland
Peking University First Hospital

Recruiting

Establishment of a Multidimensional Prediction Model for the Natural Course of VHL Disease-related Renal Cell Carcinoma

Renal Cell Carcinoma | VHL Syndrome

China
Peking Union Medical College Hospital

Not yet recruiting

A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (GEN6050XIIT)

Duchenne Muscular Dystrophy (DMD)
AbbVie

Not yet recruiting

A Study to Assess the Effect of Upadacitinib in Atopic Dermatitis-induced Sleep Disturbance in Adults With Moderate to Severe Atopic Dermatitis (Sleep Up)

Sleep Disturbance | Atopic Dermatitis
Hannover Medical School
Boehringer Ingelheim

Not yet recruiting

Study of Empagliflozin in Patients With Autosomal Dominant Polycystic Kidney Disease (EMPA-PKD) (EMPA-PKD)

Autosomal Dominant Polycystic Kidney
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts (CALNATHIS)

Limb Girdle Muscular Dystrophy
GeneCradle Inc

Recruiting

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Pompe Disease (Late-onset)

China
Gene Solutions
University of Medicine and Pharmacy at Ho Chi Minh City

Recruiting

Clinical Validation of an MCED Test in Symptomatic Populations (K-ACCELERATE) (K-ACCELERATE)

Cancer, Breast | Cancer, Lung | Cancer Colon | Cancer, Gastric | Cancer, Liver

Vietnam
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
Clinuvel Europe Limited

Recruiting

Pharmacokinetics of Afamelanotide in EPP Patients

Erythropoietic Protoporphyria

Netherlands
University College, London
London School of Hygiene and Tropical Medicine; King's College London; University... and other collaborators

Recruiting

Deep Brain Stimulation in Tourette Syndrome (Op-TICS)

Tourette Syndrome | Deep Brain Stimulation

United Kingdom
Laval University

Recruiting

Unravelling the Impact of Diet on Cardiovascular Health in Treated Heterozygous Familial Hypercholesterolemia.

Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy

Canada
Ocugen

Recruiting

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa (liMeliGhT)

Retinitis Pigmentosa

United States
Belite Bio, Inc

Not yet recruiting

A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease (DRAGON II)

Stargardt Disease 1 | STGD1

United States
AbbVie

Not yet recruiting

A Study of Upadacitinib in Adult Participants With Moderate-to-Severe Atopic Dermatitis and Inadequate Response to Dupilumab (Switch-Up)

Atopic Dermatitis
University of Mississippi Medical Center
University of Alabama at Birmingham

Recruiting

Low Systemic/High Local Exercise Load in Peds SCD

Sickle Cell Anemia in Children

United States
Beam Therapeutics Inc.

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

Alpha 1-Antitrypsin Deficiency

United Kingdom
Soligenix

Not yet recruiting

Dusquetide for the Treatment of Behcet's Disease

Behçet Disease

Turkey
RTI International

Recruiting

Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease (RECIPE)

Sickle Cell Disease

United States
Gene Solutions
Medical Genetics Institute (MGI)

Not yet recruiting

Developing & Evaluating Models for Early Predicting Obstetrical Diseases in Pregnant Women by Non-invasive Prenatal Test

Preterm Birth | Preeclampsia | Gestational Diabetes | Pregnant With Complication
University of Utah

Recruiting

Clinical Characteristics and Temporal Properties of Individual Tics in Persistent Tic Disorder (PTD)

Tourette Syndrome | Tic Disorders | Tics | Tourette Syndrome in Children | Tourette Syndrome in Adolescence | Vocal Tic | Motor Tic | Tics/Tremor

United States
Washington University School of Medicine
Alzheimer's Association; Eisai Inc.

Not yet recruiting

DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease (DIAN-TU)

Dementia | Alzheimer's Disease | Alzheimer's Disease, Familial

United States, Australia, United Kingdom
Yonsei University

Recruiting

Intestinal Ultrasound, Magnetic Resonance Enterography, and Conventional Assessment Tools Before and After Treatment of Intestinal Behcet's Disease

Intestinal Behcet Disease

Korea, Republic of
Akeso

Recruiting

A Phase III Study to Evaluate the Efficacy and Safety of AK120 in Patients With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis

China
Viridian Therapeutics, Inc.

Not yet recruiting

A Randomized, Active Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED)

Thyroid Eye Disease

United States
AstraZeneca
Parexel

Recruiting

A Study to Investigate Safety, Tolerability, and Pharmacokinetics of Oral AZD0233 Compared With Placebo in Healthy Adult Participants.

Dilated Cardiomyopathy

United States
BioMarin Pharmaceutical

Not yet recruiting

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Idiopathic Short Stature
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

Search clinical trials for: Genetic Disease

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Pakistan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations