NCT07469657
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Monogenic Diseases
- Locations
-
- Hôpital Necker Enfants Malades Paris, Île-de-France Region, France
Clinical trial condition
Genetic Diseases, Inborn
Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.
35 public trials
4 recruiting studies
Genetic Diseases, Inborn trial results
NCT05477563
- Conditions
- Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +11 more
- Locations
-
- New York Presbyterian Hospital - Morgan Stanley Children's Hospital New York, United States
- Levine Children's Hospital - Hematology Charlotte, North Carolina, United States
- TriStar Medical Group Children's Specialists - Pediatric Oncology Nashville, Tennessee, United States
- University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology Düsseldorf, Germany
NCT06917690
- Conditions
- Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
- Locations
-
- Fukuoka Children's Hospital - Dermatology Fukuoka, Japan
- Kurume University Hospital Fukuoka, Japan
- Kobe University Hospital Kobe, Japan
- Niigata University Medical and Dental Hospital Niigata, Japan
NCT06337864
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
- Locations
-
- Center for Inherited Metabolic Diseases Copenhagen, Denmark
- Copenhagen University Hospital, Rigshospitalet Copenhagen, Denmark
Not yet recruiting
Acceptability and Tolerance Market Research of a Slow Release, Phenylalanine-free Protein Substitute for the Dietary Management of Phenylketonuria
NCT07802366
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
- Locations
-
- Birmingham Children´s Hospital Birmingham, England, United Kingdom
Not yet recruiting
Prenatal Transplantation for Fetuses With Fanconi Anemia
NCT07408583
- Conditions
- Anemia, Anemia, Aplastic, Anemia, Hypoplastic, Congenital, +13 more
- Locations
-
- University of California, San Francisco San Francisco, California, United States
- Stanford University Stanford, California, United States
Enrolling by invitation
A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529
- Conditions
- Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +11 more
- Locations
-
- Lucile Packard Children's Hospital Palo Alto, California, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology Chicago, Illinois, United States
- Herbert Irving Pavilion - Hematology New York, United States
- New York Presbyterian Hospital - Morgan Stanley Children's Hospital New York, United States
Active, not recruiting
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
NCT05126758
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
- Locations
-
- Phoenix Children's Hospital Phoenix, Arizona, United States
- Arkansas Children's Hospital Little Rock, Arkansas, United States
- UCSD Altman Clinical and Translational Research Institute La Jolla, California, United States
- Children's Hospital of Los Angeles, Division of Neurology Los Angeles, California, United States
Active, not recruiting
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
NCT05356195
- Conditions
- Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
- Locations
-
- TriStar Medical Group Children's Specialists - Pediatric Oncology Nashville, Tennessee, United States
- Hospital for Sick Children - Hematology Toronto, Canada
- University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology Düsseldorf, Germany
- IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Rome, Italy
Completed
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
NCT05479981
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +11 more
- Locations
-
- University of California Los Angeles Los Angeles, California, United States
- Stanford University Palo Alto, California, United States
- University of Colorado Denver, Colorado, United States
- University of Florida Gainesville, Florida, United States