Longitudinal Studies of Brain Structure and Function in MPS Disorders

Key facts

Sponsor

University of Minnesota

Principal Investigator

Chester B. Whitley, M.D., Ph.D.

University of Minnesota

Enrollment

100 enrolled

Eligibility

6 years · All sexes

Timeline

2009 – 2019

Study locations (4)

• Oakland Children's Hospital, Oakland, California, United States
• University of Minnesota, Minneapolis, Minnesota, United States
• New York University, New York, New York, United States
• Hospital for Sick Children, Toronto, Ontario, Canada

Collaborators

Rare Diseases Clinical Research Network · National Center for Advancing Translational Sciences (NCATS) · National Institute of Neurological Disorders and Stroke (NINDS) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Lysosomal Disease Network

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Mucopolysaccharidosis type 1

Additional recruiting or active studies for the same condition.

• RECRUITINGPHASE1, PHASE2NCT06488924
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• RECRUITINGPHASE1NCT06519552
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• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06181136
  Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
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• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT06095388
  Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
  JCR Pharmaceuticals Co., Ltd.
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT05665166
  Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
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• ACTIVE NOT RECRUITINGNCT05634512
  Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
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• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT04201405
  Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
  University of Manchester
• ACTIVE NOT RECRUITINGPHASE2, PHASE3NCT03566043
  CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
  REGENXBIO Inc.

See all trials for Mucopolysaccharidosis type 1 →