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Active clinical trials for "Mucopolysaccharidosis II"

Results 61-68 of 68

A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723...

Mucopolysaccharidosis II

Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion

Completed5 enrollment criteria

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Mucopolysaccharidosis (MPS)

The main aims of the study are to learn about the percentage of mucopolysaccharidosis type II (MPS II) in adults in Brazil as well as about the diagnosis process. No study medicines will be provided to participants in this study. The data available for participants diagnosed with MPS II in DATASUS (a database of the Informatics Department of Brazilian Health System) will be reviewed. No clinic visits will be required as part of participation in this study.

Completed2 enrollment criteria

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of...

Hunter Syndrome

This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in a clinical study with an investigational agent.

Completed7 enrollment criteria

Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis II

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in characteristic storage lesions and diverse disease sequelae, and in patients with the more severe form of the disease, irreversible neurocognitive decline and higher morbidity and mortality than in patients with the attenuated form of the disease. There is currently limited information on the natural history of MPS II, especially with respect to neurocognitive decline in patients with the more severe form of the disease. This study is planned to be an observational medical records review study (data collected retrospectively and no investigational product treatment or procedures) in subjects with the severe form of MPS II. Collectively, the data may inform the design of future MPS II gene therapy treatment studies and may be utilized as historical comparative control data.

Completed14 enrollment criteria

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Mucopolysaccharidosis Type IMucopolysaccharidosis Type II3 more

The study quantitates behavioral challenges in mucopolysaccharidosis type I-III and parental coping strategies

Completed4 enrollment criteria

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

Hunter Syndrome

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

Unknown status6 enrollment criteria

Mucopolysaccharidosis Type II Observational

Mucopolysaccharidosis II

This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic ("severe") MPS II undergoing current standard of care and/or intrathecal Elaprase® for their condition. Some patients may be offered the opportunity to screen for a gene therapy study conducted by the same sponsor.

Withdrawn8 enrollment criteria

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome...

Hunter Syndrome

This post-trial access program is to allow continued access for people who participated and are benefitting from treatment on the HGT-HIT-046 (NCT01506141) or SHP609-302 (NCT02412787) studies, which will be closed down. Idursulfase-IT also known as TAK-609, is a medicine to help treat Hunter Syndrome and issues with remembering, learning new things, and concentrating, also called cognitive impairment.

Available4 enrollment criteria
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