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Hunter Syndrome clinical trials

View clinical trials related to Hunter Syndrome.

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NCT ID: NCT02412787 Completed - Hunter Syndrome Clinical Trials

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Start date: April 14, 2015
Phase: Phase 2/Phase 3
Study type: Interventional

This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.

NCT ID: NCT02171104 Recruiting - Hunter Syndrome Clinical Trials

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Start date: July 10, 2014
Phase: Phase 2
Study type: Interventional

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

NCT ID: NCT02055118 Completed - Hunter Syndrome Clinical Trials

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

AIM-IT
Start date: March 24, 2014
Phase: Phase 2/Phase 3
Study type: Interventional

Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.

NCT ID: NCT02044692 Recruiting - Hunter Syndrome Clinical Trials

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Start date: January 2014
Phase: N/A
Study type: Observational

The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II) Patients

NCT ID: NCT01822184 Completed - Hunter Syndrome Clinical Trials

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Start date: January 18, 2013
Phase:
Study type: Observational

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.

NCT ID: NCT01645189 Completed - Hunter Syndrome Clinical Trials

Safety and Efficacy of Hunterase

GC1111
Start date: July 2012
Phase: Phase 3
Study type: Interventional

The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.

NCT ID: NCT01506141 Completed - Hunter Syndrome Clinical Trials

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

Start date: August 1, 2010
Phase: Phase 1/Phase 2
Study type: Interventional

This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV) Elaprase enzyme replacement therapy.

NCT ID: NCT01449240 Completed - Hunter Syndrome Clinical Trials

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Start date: November 12, 2012
Phase:
Study type: Observational

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.

NCT ID: NCT01330277 Terminated - Clinical trials for Mucopolysaccharidosis II

Biomarkers for Hunter Syndrome

BioHunter
Start date: August 20, 2018
Phase:
Study type: Observational

International, multicenter, observational, longitudinal study to establish Hunter Syndrom biomarker/s and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s

NCT ID: NCT01043640 Completed - Hunter Syndrome Clinical Trials

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Start date: December 2009
Phase: Phase 2
Study type: Interventional

Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells. When healthy stem cells from a donor are infused into the patient, the donor white blood cells can provide the missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving a monoclonal antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening. This may be an effective treatment for inherited metabolic disorders. Purpose: The design of this study is to achieve donor cell engraftment in patients with standard-risk inherited metabolic diseases with limited peri-transplant morbidity and mortality. This will be achieved through the administration of the chemotherapy regimen described. The intention is to follow transplanted patient for years after transplant monitoring them for complications of their disease and assisting families with a multifaceted interdisciplinary approach.