Hunter Syndrome Clinical Trial
Official title:
A Prospective, Longitudinal, Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Hunter syndrome (Mucopolysaccharidosis II, [MPS II]) is a rare, genetically linked lysosomal storage disease (LSD) caused by deficiency of the enzyme, iduronate-2-sulfatase (I2S). Most MPS II patients will present with some degree of neurodevelopmental involvement, ranging from severe cognitive impairment and behavioral problems to mildly impaired cognition. This is an observational study; no investigational treatment will be administered. The primary objective of this study is to evaluate the neurodevelopmental status of pediatric patients with MPS II over time and to gain information to guide future treatment studies in this patient population.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
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|
Phase 1 | |
| Completed |
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|
Phase 2/Phase 3 | |
| Completed |
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|
||
| Active, not recruiting |
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Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
|
Phase 4 | |
| Completed |
NCT00882921 -
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
|
||
| Completed |
NCT00920647 -
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
|
Phase 1/Phase 2 | |
| Completed |
NCT01449240 -
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
|
||
| Completed |
NCT03920540 -
A Study of GC1111 in Hunter Syndrom Patients
|
Phase 3 | |
| Recruiting |
NCT06031259 -
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
|
Phase 2/Phase 3 | |
| Completed |
NCT02055118 -
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
|
Phase 2/Phase 3 | |
| Completed |
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Safety and Efficacy of Hunterase
|
Phase 3 | |
| Completed |
NCT00937794 -
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
|
||
| Recruiting |
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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
|
Phase 2 | |
| Recruiting |
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The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
|
N/A | |
| Completed |
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Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
|
||
| Recruiting |
NCT05494593 -
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
|
Phase 4 | |
| Completed |
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Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
|
Phase 4 | |
| Completed |
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Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
|
Phase 2 | |
| Terminated |
NCT01330277 -
Biomarkers for Hunter Syndrome
|
||
| Completed |
NCT01506141 -
An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
|
Phase 1/Phase 2 |