Search clinical trials for: genetic study

Total 131496 results

Ray Therapeutics, Inc.

Not yet recruiting

Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa Patients

Retinitis Pigmentosa

United States
Istituto Scientifico Romagnolo per lo Studio e...
Charite University, Berlin, Germany; University of Bologna; Hannover Medical... and other collaborators

Not yet recruiting

Master Framework For Relapse or Refractory Acute Myeloid Leukemia (IMPACT STREAM)

Acute Myeloid Leukemia, in Relapse | Acute Myeloid Leukemia Refractory

Italy
Changchun GeneScience Pharmaceutical Co., Ltd.

Recruiting

A Bioequivalence Study of Two Different Dosage Form of Genakumab

Gouty Arthritis (GA)

China
Khondrion BV

Not yet recruiting

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases (KH176-301)

Mitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
Dermal Laboratories Ltd

Not yet recruiting

A Study to Evaluate the Emollient Performance of Doublebase Once in the Treatment of Atopic Eczema

Atopic Eczema
Dana-Farber Cancer Institute

Not yet recruiting

Evaluation of Multi-Cancer Early Detection Testing in a High-Risk Population: The INFORM Study

Cancer Predisposition Syndrome | Predisposition, Genetic

United States
Hoffmann-La Roche

Not yet recruiting

An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (SHIELD DMD)

Duchenne Muscular Dystrophy

United States
Arizona State University
American Speech-Language-Hearing Foundation

Recruiting

Proactive Speech and Language Intervention for Infants With Down Syndrome (BBC-DS)

Down Syndrome

United States
Pfizer

Not yet recruiting

A Study to Learn About the Study Medicine PF-07934040 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Genetic Mutation.

Carcinoma, Non-Small-Cell Lung | Colorectal Neoplasms | Carcinoma, Pancreatic Ductal
University of Central Florida
University of South Florida

Not yet recruiting

Gut Microbiomes in HD

Huntington Disease

United States
GlaxoSmithKline

Not yet recruiting

Long-Term Study (AtDvance) to Evaluate GSK1070806 in Atopic Dermatitis. (AtDvance)

Dermatitis, Atopic

United States, Germany, Spain, France, Italy, Japan, China, Argentina, Greece, Czechia, Korea, Republic of, Panama, Bulgaria, Canada, Poland, Mexico, Thailand
Ascidian Therapeutics, Inc

Recruiting

Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials

Stargardt Disease | Stargardt Disease 1 | Juvenile Macular Degeneration | Cone Rod Dystrophy

United States
Novartis Pharmaceuticals

Not yet recruiting

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (SPARKLE)

Sickle Cell Disease
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
Enloe Health
Robert A. Winn Diversity in Clinical Trials Award Program

Not yet recruiting

A Pilot Study of Genetic Testing Uptake Through Enhanced Oncology Nurse-Led Intervention (GENESIS)

Solid Tumor, Adult
Novo Nordisk A/S

Recruiting

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

Healthy Volunteers Sickle Cell Disease, Thalassemia

United States
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
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
University of Mons

Recruiting

Quantitative Assessment of Orofacial Muscle Function in FSHD

Facioscapulohumeral Muscular Dystrophy

Belgium
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

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

China
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)
Sanofi
Regeneron Pharmaceuticals

Not yet recruiting

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

Atopic Dermatitis
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
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
Spinogenix
Avance Clinical Pty Ltd.

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
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
Avidity Biosciences, Inc.

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

United States
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain, Canada, France, Germany, Sweden, United Kingdom
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
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
Wave Life Sciences Ltd.

Not yet recruiting

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD (RestorAATion-2)

Alpha-1 Antitrypsin Deficiency
Tisento Therapeutics

Not yet recruiting

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

United States, Australia, Italy, Germany, Canada, United Kingdom
Amgen

Not yet recruiting

A Study of AMG 732 in Healthy Participants and Participants With Thyroid Eye Disease

Thyroid Eye Disease
M.D. Anderson Cancer Center
Ascentage Pharma Group Inc.

Not yet recruiting

A Phase I Study of Decitabine, Lisaftoclax, and Olverembatinib in Patients With Advanced Chronic Myeloid Leukemia and Philadelphia Chromosome-Positive Acute Myeloid Leukemia

Advanced Chronic Myeloid Leukemia | Philadelphia Chromosome-Positive Acute Myeloid Leukemia

United States
Children's Oncology Group

Not yet recruiting

A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)

Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor
Essen Biotech

Not yet recruiting

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (BAH243)

Sickle Cell Disease | Sickle-Cell Disease With Crisis

China
Takeda

Not yet recruiting

A Study on Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in Children, Teenagers and Adults in Portugal (DRALEGA-PT)

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)
Apogee Therapeutics, Inc.

Recruiting

A Study Evaluating APG777 in Atopic Dermatitis

Atopic Dermatitis

United States, Canada
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
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
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

Search clinical trials for: genetic study

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