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Genetic Diseases, Inborn

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Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,633 public trials 2,161 recruiting studies

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Genetic Diseases, Inborn trial results

Completed

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

NCT02234934

Conditions: Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, +12 more
Locations: University of California, Los Angeles (UCLA) Los Angeles, California, United States

National Institutes of Health Bethesda, Maryland, United States

Children's Hospital Boston Boston, Massachusetts, United States

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Completed

RHD Genotype Matched Red Cells for Anti-D

NCT04156906

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States

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Completed

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

NCT03231306

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +19 more
Locations: University of Alabama at Birmingham Birmingham, Alabama, United States

Children's Hospital of Los Angeles Los Angeles, California, United States

University of California at Los Angeles Los Angeles, California, United States

UCSF Benioff Children's Hospital Oakland Oakland, California, United States

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Completed

Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on Pattern-electroretinogram in Glaucoma

NCT06431113

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +15 more
Locations: Clinica Oculistica Università Federico II Naples, Napoli, Italy

Gemma Caterina Maria Rossi Pavia, PV, Italy

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Completed

A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A

NCT05707351

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations: Beijing Children's Hospital, Capital Medical University Beijing, China

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences Beijing, China

Xiangya Hospital of Central South University Changsha, China

Fujian Medical University Union Hospital Fuzhou, China

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Completed

A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa

NCT00447980

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations: Retina-Vitreous Associates Medical Group Beverly Hills, California, United States

University of Califoria, Davis Sacramento, California, United States

University of California, San Francisco San Francisco, California, United States

Bascom Palmer Eye Insitute Miami, Florida, United States

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Completed

Evaluation of the Psychological Profile of Adult Patients With Prader-Willi Syndrome

NCT06295315

Conditions: Abnormalities, Multiple, Behavior, Chromosome Disorders, +16 more
Locations: Istituto Auxologico Italiano IRCCS, site Piancavallo Oggebbio, Verbania, Italy

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Completed

RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy

NCT05044598

Conditions: Aniridia, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +6 more
Locations: Moorfields Eye Hospital London, United Kingdom

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Completed

Short Stature and Psychological Well-being

NCT06295341

Conditions: Behavior, Bone Diseases, Bone Diseases, Developmental, +9 more
Locations: Istituto Auxologico Italiano Milan, Italy

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Completed

An Exploratory Study to Evaluate a Digital Intervention to Disrupt Scratching in Atopic Dermatitis and Psoriasis

NCT06275659

Conditions: Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +15 more
Locations: Click Therapeutics New York, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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