Search clinical trials for: genetic condition

Total 439724 results

Kun Sun

Recruiting

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

Spinal Muscular Atrophy 1

China
University of Alabama at Birmingham
Children's Hospital of Philadelphia; Congressionally Directed Medical Research...

Not yet recruiting

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 (NF114)

Neurofibromatosis 1 | Plexiform Neurofibroma
University of California, Los Angeles
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Not yet recruiting

UR+AIMS Gout Wearable Skin Uric Acid Monitor Study

Gout

United States
Wave Life Sciences Ltd.

Recruiting

A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) (RestorAATion-1)

Alpha-1 Antitrypsin Deficiency

United Kingdom
Biocad

Recruiting

A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A (EDELWEISS)

Hemophilia A

Russian Federation
Benitec Biopharma, Inc.

Recruiting

A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

Oculopharyngeal Muscular Dystrophy

United States
Vertex Pharmaceuticals Incorporated

Recruiting

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)

Myotonic Dystrophy Type 1 (DM1)

United Kingdom, Canada, Australia
Rutgers, The State University of New Jersey

Recruiting

Choices About Genetic Testing And Learning Your Risk With Smart Technology (CATALYST)

Breast Cancer | Colorectal Cancer | Ovarian Cancer | Fallopian Tube Cancer | Prostate Cancer | Endometrial Cancer | Pancreas Cancer | Peritoneal Cancer

United States
Chiesi Farmaceutici S.p.A.

Not yet recruiting

Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3 (LAMPO(0-3))

Alpha-Mannosidosis
University of Maryland, Baltimore

Not yet recruiting

Virtual Reality Devices as an Adjunct to Usual Care for Patients With Sickle Cell Disease Experiencing Vaso-Occlusive Crises

Acute Pain | Sickle Cell Disease | Vaso-occlusive Pain Episode in Sickle Cell Disease

United States
St. Justine's Hospital

Not yet recruiting

Virtual Reality for Pain Management During Dupilumab Injection (VR-DERMA)

Pain | Anxiety | Atopic Dermatitis

Canada
Shanghai Mental Health Center

Recruiting

Association of Peripheral Immune Cells With Antidepressant Treatment Response

Depressive Disorder | Genetic Change

China
Sixth Affiliated Hospital, Sun Yat-sen University

Recruiting

Multicenter Analysis of Genomic and Metabolic Data of Neonatal Genetic Diseases (MAOFGAMDNGD)

Hereditary Diseases

China
University Hospital, Strasbourg, France

Recruiting

Interference by Activated Protein C Resistance in the Determination of Protein S Activity (PS)

Resistance to Activated Protein C in the Determination of Protein S Activity

France
University of Nicosia
Cyprus Institute of Neurology and Genetics

Recruiting

Probiotics in Mild Alzheimer's Disease

Neurodegenerative Diseases | Cognition Disorders in Old Age

Cyprus
Denali Therapeutics Inc.

Recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Mucopolysaccharidosis Type IIIA

United States
Sanofi

Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (COAST 2)

Dermatitis Atopic

Argentina, Japan, China, Bulgaria, Czechia, Spain, United States, South Africa, United Kingdom, Portugal, Denmark, Sweden, Turkey, Italy
Viridian Therapeutics, Inc.

Recruiting

An Open-label Study for Participants Who Are Non-responders at the End of Treatment Assessment on the VRDN-001-101 and VRDN-001-301 Pivotal Studies

Thyroid Eye Disease

United States
University of Washington
Breast Cancer Research Foundation

Not yet recruiting

Functional Imaging in Prediction of Response to Abemaciclib for Advanced Hormone Receptor-Positive, HER2-Negative Breast Cancer

Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage IV Breast Cancer AJCC v8 | Metastatic HER2-Negative Breast Carcinoma | Metastatic Hormone Receptor-Positive Breast Carcinoma | Locally Advanced Unresectable HER2-Negative Breast Carcinoma | Locally Advanced Unresectable Hormone Receptor-Positive...

United States
Huashan Hospital

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

Pompe Disease (Late-onset)

China
Assiut University

Not yet recruiting

Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities

Wilson Disease
Azienda Ospedaliero-Universitaria di Modena

Recruiting

Role of Cardiac AngioMR in Diagnosis of Cardiac and Vascular Anomalies in Adult Patients With Turner Syndrome

Turner Syndrome | Anomaly Heart

Italy
ETH Zurich
University of Zurich; Agroscope Liebefeld-Posieux Research Station ALP; Swiss...

Recruiting

Lactobreath: A Study to Diagnose Lactose Intolerance Using Breath Markers (Lactobreath)

Lactose Intolerance

Switzerland
Columbia University

Not yet recruiting

Impact of Exercise on Eyeblink Conditioning in Spinocerebellar Ataxias

Spinocerebellar Ataxias

United States
St. Joseph's Hospital and Medical Center, Phoenix
Texas Tech University

Not yet recruiting

MCLENA-2: A Phase II Clinical Trial for the Assessment of Lenalidomide in Patients With Mild Cognitive Impairment Due to Alzheimer's Disease

Cognitive Dysfunction | Neurodegenerative Disease, Hereditary | Cognitive Impairment, Mild | Amyloid Plaque | Inflammation, Brain
Minoryx Therapeutics, S.L.

Recruiting

Disease Progression in Women With X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

France
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Study of the Blood and Skin Immunological Profile of Patients With Recessive Dystrophic Epidermolysis Bullosa: in Vivo Analysis and the Impact of Placental Stem Cells in Vitro (ISTRADEB)

Epidermolysis Bullosa Dystrophica
Tavotek Biotherapeutics

Recruiting

A Study of TAVO101 in Atopic Dermatitis Patients

Atopic Dermatitis

New Zealand
AstraZeneca

Recruiting

Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs (PEDIA)

Neurofibroma, Plexiform

China
University Hospital, Ghent

Not yet recruiting

PPOS (Progestin Primed Ovarian Stimulation) and Corifollitropin Alfa (CFA) Cross-over Study

Quality of Life | Ovarian Stimulation | Preimplantation Genetic Testing | Fertility Preservation
Power Life Sciences Inc.

Not yet recruiting

Investigating Participation Patterns of Patients in Atopic Dermatitis Clinical Trials

Atopic Dermatitis

United States
Hamilton Health Sciences Corporation
Canadian Institutes of Health Research (CIHR); Population Health Research Institute and other collaborators

Not yet recruiting

Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy (TaRGET)

Arrhythmogenic Right Ventricular Cardiomyopathy | Arrhythmogenic Cardiomyopathy

Canada
Genrix (Shanghai) Biopharmaceutical Co., Ltd.

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of GR2002 Injection in Patients With Atopic Dermatitis.

Atopic Dermatitis

China
Sun Yat-sen University

Not yet recruiting

The Effect of Sodium Fluoride Application on the Prevention of Radiation Caries in NPC Patients

Sodium Fluoride
Hacettepe University

Recruiting

Assessment of Cognitive Function and Exercise Capacity in Cystic Fibrosis

Cystic Fibrosis

Turkey
ReCode Therapeutics
Sano Genetics; Reverba

Recruiting

A Study Providing Genetic Testing to Find Those Who May Have Primary Ciliary Dyskinesia for Potential Clinical Trials

Primary Ciliary Dyskinesia

United States
ACADIA Pharmaceuticals Inc.

Recruiting

Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

Hyperphagia in Prader-Willi Syndrome

United States, Canada, United Kingdom
Sunshine Guojian Pharmaceutical (Shanghai) Co.,...

Recruiting

Evaluation of 611(Recombinant Humanized Anti-interleukin-4 Receptor Alpha IgG4 Monoclonal Antibody) in Chinese Adults With Moderate to Severe Atopic Dermatitis

Dermatitis, Atopic

China
Dow University of Health Sciences
University of Karachi

Recruiting

A Randomized Controlled Trial of Vitamin D Supplementation on Salivary Biomarkers and Cariogenic Oral Microbiome (Incipient)

Dental Caries | White Spot Lesion | Initial Caries | Incipient Enamel Caries

Pakistan
Children's Hospital Medical Center, Cincinnati
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Realizing Effectiveness Across Continents With Hydroxyurea (REACH)

Children | Sickle Cell Disease

Congo, The Democratic Republic of the, Angola, Kenya, Uganda
Sunshine Guojian Pharmaceutical (Shanghai) Co.,...

Not yet recruiting

A Study of Recombinant Anti-IL-1β Humanized Monoclonal Antibody Injection in Chinese Participants With Acute Gout

Acute Gout
BioMarin Pharmaceutical
PicnicHealth

Recruiting

A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States (VISTA)

Achondroplasia

United States
Sanofi
Regeneron Pharmaceuticals

Recruiting

A Long-term Data Collection Study of Participants in France Aged 6 Years Old or More With Atopic Dermatitis Receiving Dupilumab (DUPILAB)

Atopic Dermatitis

France
Qilu Hospital of Shandong University

Recruiting

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy (SEARCH)

Fabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular Hypertrophy

China
Gianni Soraru
Mario Negri Institute for Pharmacological Research

Recruiting

A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy (BetaSBMA)

Spinal and Bulbar Muscular Atrophy

Italy
Central Hospital, Nancy, France

Not yet recruiting

Study of N-homocysteinylation of Key Proteins in Alzheimer's Disease (HO-MA) (HO-MA)

Alzheimer Disease

France
Gene Solutions

Recruiting

Analysis of Circulating Tumor DNA Dynamics to Predict and Monitor Response to TKI in Patients With Advanced NSCLC.

Non Small Cell Lung Cancer | Stage IIIB Non-small Cell Lung Cancer | Stage IV Non-small Cell Lung Cancer | Stage IIIC Non-Small Cell Lung Cancer | Tyrosine Kinase Inhibitors

Vietnam
Hematology department of the 920th hospital

Recruiting

Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia

Effect of Drug

China
Assiut University

Not yet recruiting

Celiac Disease Among Egyptian Children With Unexplained Short Stature

Celiac Disease
Takeda

Recruiting

Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

Alpha1-Antitrypsin Deficiency

Italy, United States, Poland, Portugal

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