Search clinical trials for: Genetic diseases, Inborn

Total 18709 results

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
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
Power Life Sciences Inc.

Not yet recruiting

Investigating Participation Patterns of Patients in Atopic Dermatitis Clinical Trials

Atopic Dermatitis

United States
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
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
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
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
BioMarin Pharmaceutical
PicnicHealth

Recruiting

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

Achondroplasia

United States
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
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
Verve Therapeutics, Inc.

Not yet recruiting

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

Heterozygous Familial Hypercholesterolemia | Premature Coronary Heart Disease
University of Cincinnati

Recruiting

Hormonal Responses to a Mixed Meal in People With Cystic Fibrosis

Cystic Fibrosis

United States
He Huang
Lingyi Biotech Co., Ltd.

Recruiting

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I (LY-M001)

Gaucher Disease Type I

China
National Institute for Mother and Child Health...
Marie Curie Emergency Children's Hospital, Bucharest; Victor Gomoiu Children...

Recruiting

Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease

Chronic Granulomatous Disease

Romania
Keymed Biosciences Co.Ltd

Not yet recruiting

Study of CM310 in Children Subjects With Moderate-to-severe Atopic Dermatis

Atopic Dermatitis
Royal Marsden NHS Foundation Trust

Recruiting

Inherited Cancer Early Diagnosis (ICED) Study (ICED)

Lynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene Mutation

United Kingdom
David M. Ritter

Recruiting

Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)

Tuberous Sclerosis Complex | Lymphangioleiomyomatosis

United States
Clarametyx Biosciences, Inc.

Recruiting

A Study To Evaluate The Safety Of CMTX-101 In People With Cystic Fibrosis

Cystic Fibrosis | Persistent Infection

United States
University of Bonn

Recruiting

Characteristics of Hypophosphatasia in Adult Patients in Rheumatology (COHIR)

Hypophosphatasia

Germany
Karolinska Institutet

Recruiting

Evaluation of Cascade Screening for Elevated Lipoprotein(a) (LipoaScreen)

Cardiovascular Diseases | Dyslipidemias | Hereditary Diseases

Sweden
Candela Corporation

Recruiting

Clinical Feasibility Study to Evaluate the Safety and Performance of the Profound Matrix System

Skin Condition | Wrinkle | Skin Laxity

United States, Canada, Spain
Guangzhou JOYO Pharma Co., Ltd
Chengdu JOYO pharma Co., Ltd.

Not yet recruiting

A Study to Evaluate JYP0061 Tablets in Patients With Moderate-to-severe Atopic Dermatitis

Moderate-to-severe Atopic Dermatitis
Hospices Civils de Lyon

Recruiting

Study of Surgical Practices in Patients With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®) (CHALE)

Hemophilia A | Hemophilia B

France
Johns Hopkins University
Neurofibromatosis Therapeutic Acceleration Program

Recruiting

Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF). (Mirda)

Cutaneous Neurofibroma | NF1 | Monotherapy

United States
Sohag University

Recruiting

The Role of Endothelin 1 as a Marker of Renal Impairment in Sickle Cell Disease

Sickle Cell Disease

Egypt
Chulalongkorn University

Recruiting

Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A

Joint Bleed | Severe Hemophilia A Without Inhibitor

Thailand
Novartis Pharmaceuticals

Recruiting

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Sickle Cell Disease

United States
Vertex Pharmaceuticals Incorporated

Recruiting

A Phase 1, First-in-human Study of VX-828

Cystic Fibrosis

United States
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Test of Aquatic Mobility of SMA Infants (BAINSMA)

Infantile Spinal Muscular Atrophy

France
Elpida Therapeutics SPC

Not yet recruiting

A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J (CMT4J)

Neuromuscular Diseases | Peripheral Neuropathy | Neuro-Degenerative Disease
Taysha Gene Therapies, Inc.

Recruiting

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Rett Syndrome

United States, United Kingdom
Astellas Gene Therapies

Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Pompe Disease (Late-onset)

United States, Spain, France, Taiwan, Italy
Azidus Brasil
Boya Bio Pharmaceutical Group Co Ltd

Not yet recruiting

Efficacy and Safety and Pharmacokinetics of Boya IVIG (Boya)

Primary Immunodeficiency Disease
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, United Kingdom

Search clinical trials for: Genetic diseases, Inborn

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