NCT05619900
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +36 more
- Locations
-
- University of California San Francisco San Francisco, California, United States
Clinical trial condition
Mucopolysaccharidosis II
Explore clinical trials studying Mucopolysaccharidosis II. Study availability and eligibility vary by location and protocol.
28 public trials
4 recruiting studies
Mucopolysaccharidosis II trial results
Recruiting
MPS (RaDiCo Cohort) (RaDiCo-MPS)
NCT06036693
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +33 more
- Locations
-
- Centre Hospitalier Universitaire d'Angers Angers, France
- Hôpital des Enfants - Groupe Hospitalier Pellegrin Bordeaux, France
- Hôpital Morvan Brest, France
- Hôpital d'Estaing Clermont-Ferrand, France
NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Hackensack University Medical Center Hackensack, New Jersey, United States
- UNC Children's Research Institute Chapel Hill, North Carolina, United States
Enrolling by invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05368038
- Conditions
- Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
- Locations
-
- Maimonides Medical Center Brooklyn, New York, United States
- NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
- North Shore University Hospital Manhasset, New York, United States
- NYU Langone Health - Tisch Hospital New York, United States
Enrolling by invitation
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
NCT06075537
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Hackensack University Medical Center Hackensack, New Jersey, United States
- UNC Children's Research Institute Chapel Hill, North Carolina, United States
Enrolling by invitation
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects
NCT05594992
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
- Hospital Universitario Austral Buenos Aires, Argentina
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
Active, not recruiting
Long-term Follow-Up for RGX-121
NCT04597385
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
- Locations
-
- University of California, San Francisco (UCSF)- Benioff Children's Hospital San Francisco, California, United States
- Saint Peter's Healthcare System New Brunswich, New Jersey, United States
- The Children's Hospital of Philadelphia (CHOP) Philadelphia, Pennsylvania, United States
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
Active, not recruiting
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
NCT04573023
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
- Locations
-
- Phoenix Children's Hospital Phoenix, Arizona, United States
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- University of Minnesota Minneapolis, Minnesota, United States
Active, not recruiting
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
NCT05665166
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
- Locations
-
- Manchester University Foundation Trust Manchester, United Kingdom
Related conditions
- Carbohydrate Metabolism, Inborn Errors (28)
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities (28)
- Connective Tissue Diseases (28)
- Genetic Diseases, Inborn (28)
- Genetic Diseases, X-Linked (28)
- Heredodegenerative Disorders, Nervous System (28)
- Intellectual Disability (28)
- Metabolic Diseases (28)