Hunter Syndrome Clinical Trial
Official title:
A Cerebrospinal Fluid Collection Study in Pediatric and Adult Patients With Hunter Syndrome
The purpose of the study is to collect data on CSF biomarkers in patients with Hunter Syndrome that would serve as reference data for comparison with cognitively impaired patients with Hunter syndrome, patients with other lysosomal storage diseases, or other diseases with CNS involvement.
To determine levels of glycosaminoglycans (GAGs), including dermatan sulfate (DS) and heparan sulfate (HS), GAG-degradation products, and other biomarkers of central nervous system (CNS) and lysosomal function in cerebrospinal fluid (CSF) in pediatric and adult patients with Hunter syndrome. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05422482 -
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ
|
Phase 1 | |
Completed |
NCT00630747 -
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
|
Phase 2/Phase 3 | |
Completed |
NCT03292887 -
Hunter Outcome Survey (HOS)
|
||
Active, not recruiting |
NCT02455622 -
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 |
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 |
NCT01645189 -
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 |
NCT02171104 -
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
|
Phase 2 | |
Recruiting |
NCT02044692 -
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
|
N/A | |
Completed |
NCT03582449 -
Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
|
||
Completed |
NCT01822184 -
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
|
||
Recruiting |
NCT05494593 -
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
|
Phase 4 | |
Completed |
NCT00607386 -
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
|
Phase 4 | |
Completed |
NCT01043640 -
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 |