Search clinical trials for: genetic investigation

Total 31458 results

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
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
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
Apogee Therapeutics, Inc.

Recruiting

A Study Evaluating APG777 in Atopic Dermatitis

Atopic Dermatitis

United States, Canada
LEO Pharma

Not yet recruiting

A Trial to Evaluate the Efficacy and Safety of Tralokinumab in Combination With Topical Corticosteroids in Children and Infants With Moderate-to-severe Atopic Dermatitis (TRAPEDS 2)

Atopic Dermatitis

United States
Eli Lilly and Company

Recruiting

A Study to Investigate the Effect of Lepodisiran on the Reduction of Major Adverse Cardiovascular Events in Adults With Elevated Lipoprotein(a) - ACCLAIM-Lp(a)

Elevated Lp(a) | Atherosclerotic Cardiovascular Disease (ASCVD)

United States, Australia, Spain, Canada, Argentina, China, Italy, France, Japan, Taiwan, Korea, Republic of, Denmark, Germany, Hungary, Israel, Austria, Belgium, Netherlands, Brazil, Mexico, Romania, United Kingdom, Greece, Czechia, Poland and more
Travere Therapeutics, Inc.

Recruiting

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment (HARMONY)

Homocystinuria

United States
Sanofi

Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab on Background Topical Corticosteroids Therapy in Participants Aged 12 Years and Older With Moderate-to-severe AD Who Have Had an Inadequate Response to Prior Biologic Therapy or an Oral JAK Inhibitor (AQUA)

Dermatitis Atopic

Chile, United Kingdom, United States, Canada, Turkey, Israel, Korea, Republic of
Sanofi

Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis on Background Topical Corticosteroids (SHORE)

Dermatitis Atopic

Japan, Chile, Canada, Argentina, United States, Turkey, China
Sanofi

Recruiting

A Study to Observe How Adolescent Patients With Severe Atopic Dermatitis Despite Less Extensive Skin Lesions (Eczema Area and Severity Index Score < 16) Respond to Dupilumab Treatment (AD-BEASCUITS)

Dermatitis Atopic

Italy
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, Czechia, Spain, United States, South Africa, United Kingdom, Denmark, Sweden, Turkey
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
Sanofi

Recruiting

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Hemophilia A

Taiwan
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 1)

Dermatitis Atopic

Israel, Chile, Australia, United States, France, Canada, Germany, Korea, Republic of, Taiwan, China
Verve Therapeutics, Inc.

Not yet recruiting

Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial Hypercholesterolemia

New Zealand, United Kingdom
Novo Nordisk A/S

Recruiting

Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed

Growth Hormone Deficiency in Children

Japan
Sanofi
Regeneron Pharmaceuticals

Recruiting

Observational Study of Canadian Patients (6 Years or Older) Receiving Dupixent® for Moderate to Severe Atopic Dermatitis (CANDID)

Moderate to Severe Atopic Dermatitis

Canada
Gazi University

Recruiting

Investigation of the Effects of Pulmonary Rehabilitation in Children With Primary Immunodeficiency

Primary Immunodeficiency

Turkey
Ascendis Pharma Growth Disorders A/S

Recruiting

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Achondroplasia

United States, Australia
Sanofi

Recruiting

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia (upreACH-2)

Osteochondrodysplasia

Australia, Spain, Korea, Republic of, China, Italy
Sanofi
Kymera Therapeutics, Inc.

Recruiting

Phase 2 Study of SAR444656 in Adult Participants With Moderate to Severe Atopic Dermatitis (ADVANTA)

Atopic Dermatitis

Greece, Poland, United States, Germany
Sanofi

Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

Fabry Disease

Taiwan
Sanofi

Recruiting

Real-world Effectiveness Study of Long-term Treatment With Dupilumab in Participants ≥6 Years With Atopic Dermatitis (PROTYPE2)

Atopic Dermatitis

Germany
Sanofi

Recruiting

A Study to Investigate Vaccine Responses in Subcutaneous Amlitelimab Treated Atopic Dermatitis Participants Aged 18 Years and Older Compared With Placebo (HYDRO)

Dermatitis Atopic

Canada, United States
GlaxoSmithKline

Recruiting

A Dose Finding Study to Investigate the Safety and Effectiveness of GSK1070806 in Adult Participants With Moderate to Severe Atopic Dermatitis

Dermatitis, Atopic

Spain, Bulgaria, Germany, Korea, Republic of, Canada, Argentina, China, United States, Poland, Italy, Czechia, Japan, Panama, Greece, Thailand, France, Mexico
Sanofi
Regeneron Pharmaceuticals

Recruiting

A Study of Long-term Effect of Dupilumab on Skin Barrier Function in Pediatric Participants With Atopic Dermatitis (PELISTAD-EX2)

Dermatitis Atopic

United States, United Kingdom
Sanofi

Recruiting

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

Phenylketonuria

Turkey, United States
LEO Pharma

Recruiting

A Trial to Evaluate the Efficacy and Safety of Different Doses of LEO 138559 in Adults With Moderate-to-severe Atopic Dermatitis

Atopic Dermatitis

United States, Canada, France, Germany, Japan, Poland, United Kingdom, Czechia, Spain, Romania, Hungary
Sanofi

Recruiting

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

Gaucher Disease

United States
Novo Nordisk A/S

Recruiting

A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

Idiopathic Short Stature

United States
Pfizer

Recruiting

A Study to Learn About the Study Medicine (PF-06823859) in Adults With Active CLE or SLE With Skin Symptoms.

Lupus Erythematosus, Systemic | Lupus Erythematosus, Cutaneous

United States, Spain, Taiwan, Canada, Greece
University of Milano Bicocca
Genetic s.p.a.

Recruiting

Vancomycin in Primary Sclerosing Cholangitis in Italy (VanC-IT)

Primary Sclerosing Cholangitis | IBD | Liver and Intrahepatic Bile Duct Disorder

Italy
Sanofi

Recruiting

Open Label, Long-term Study Evaluating Safety and Efficacy of Subcutaneous Amlitelimab in Participants Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis (ATLANTIS)

Dermatitis Atopic

Italy, United Kingdom, Argentina, France, Chile, Czechia, Germany, Japan, Spain, China, Netherlands, Brazil, Canada, Denmark, Korea, Republic of, South Africa, Taiwan, Poland, India, Mexico
Sanofi

Recruiting

Avalglucosidase Alfa Pregnancy Study

Pregnancy | Pompe Disease

United States
Number 2 Children's Hospital, Ho Chi Minh City
University Hospital, Geneva; Swiss National Science Foundation; University of... and other collaborators

Recruiting

Genetic Investigations in Children With Developmental and Epileptic Encephalopathies in Ho Chi Minh City, Vietnam

Epileptic Encephalopathy | Whole Exome Sequencing

Vietnam
Lisa Gundestrup

Not yet recruiting

Aortic Stenosis Associated Left Ventricular Remodeling - An Investigation of Genetic and Gender Specific Differences in 170 Patients Undergoing Aortic Valve Replacement Surgery (SALVAGE)

Aortic Valve Stenosis
Pharmacosmos A/S
ICON plc

Recruiting

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia

Beta Thalassemia Major Anemia

Denmark
Sanofi

Recruiting

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia (ATLAS-NEO)

Hemophilia

Greece, Korea, Republic of, France, Taiwan, Italy, Spain, United States, Canada, China, Germany, India, Japan, Mexico, South Africa, Turkey, Poland, Saudi Arabia
GlaxoSmithKline

Recruiting

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

Hematologic Diseases | Anaemia, Sickle Cell

United States
Sage Therapeutics

Recruiting

A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease

Huntington Disease

United States, Canada
Novo Nordisk A/S

Recruiting

An Observational Research Study of the Health of Joints in People With Haemophilia Taking the Medicine Esperoct

Haemophilia A

Canada
Marmara University

Recruiting

Investigation of Autonomic Dysfunction in Patients With Familial Mediterranean Fever

Familial Mediterranean Fever | Autonomic Dysfunction

Turkey
LG Chem

Recruiting

Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients (EURELIA2)

Hyperuricemia | Gout | Gout Flare | Tophi

United States
LG Chem

Recruiting

Tigulixostat, Phase 3 Study, Placebo Controlled in Gout Patients (EURELIA1)

Hyperuricemia | Gout | Gout Flare | Tophi

United States
National Taiwan University Hospital
Mackay Memorial Hospital; National Cheng-Kung University Hospital; Tri-Service... and other collaborators

Recruiting

Investigation of Prognostic Biomarkers, Host Factors and Viral Factors for COVID-19 in Children

Multisystem Inflammatory Syndrome in Children | COVID-19-Associated Encephalitis

Taiwan
Sanofi

Recruiting

A Study to Evaluate the Quality of Life of Male and Female Adult Patients With Severe Refractory Atopic Dermatitis Treated With Dupilumab in Czech Republic

Atopic Dermatitis

Czechia
Regeneron Pharmaceuticals

Recruiting

A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Non-Alcoholic Steatohepatitis (NASH) in Adult Participants at Increased Genetic Risk for This Condition (NASHGEN-2)

Non-alcoholic Steatohepatitis (NASH) | Metabolic Dysfunction Associated Steatohepatitis (MASH)

United States, Korea, Republic of, Puerto Rico, Japan
Sanofi

Recruiting

Long-Term Safety and Efficacy Evaluation of Amlitelimab in Participants of Previous Amlitelimab Moderate to Severe Atopic Dermatitis Clinical Trials (RIVER-AD)

Dermatitis Atopic

Canada, United States, Australia, Bulgaria, Czechia, Germany, Hungary, Japan, Poland, Spain, Taiwan, United Kingdom
Pfizer
Pfizer

Recruiting

A Phase 2/3 Study in Adult and Pediatric Participants With SCD

Sickle Cell Disease

United States, Nigeria, Kenya, Lebanon
Clinuvel Europe Limited

Recruiting

A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V

Xeroderma Pigmentosum

Belgium, Spain

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