Mucopolysaccharidosis Type Ii
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CLINICAL_TRIALS trial_result · 2026-06-04Clinical Trial Update: RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) [Phase 3]
Phase 3 clinical trial update for RGX-121-3102 gene therapy in MPS II (Hunter syndrome) is active but not recruiting.
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CLINICAL_TRIALS trial_result · 2026-04-21Clinical Trial Update: An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 [Phase 2, Phase 3]
An extension study is evaluating the long-term safety, tolerability, and efficacy of Tividenofusp alfa (DNL310) in participants with mucopol…
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CLINICAL_TRIALS trial_result · 2026-04-02Clinical Trial Update: Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age [Phase 4]
Phase 4 long-term study assessing height and weight outcomes in children with Mucopolysaccharidosis type II (Hunter syndrome) who began idur…
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) [Phase 3]
Phase 3 trial RGX-121-3102 gene therapy for Mucopolysaccharidosis type II (Hunter syndrome) is currently suspended.
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CLINICAL_TRIALS trial_result · 2026-01-15Clinical Trial Update: An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) [Phase 3]
An extension Phase 3 trial update to assess the long-term safety and efficacy of Hunterase (Idursulfase Beta) in Mucopolysaccharidosis type …
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CLINICAL_TRIALS trial_result · 2025-08-06Clinical Trial Update: 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]
An extension Phase 1/2 study evaluating the long-term safety and clinical outcomes of combining Idursulfase-IT with Elaprase in pediatric pa…
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CLINICAL_TRIALS trial_result · 2025-07-30Clinical Trial Update: A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II [Phase 2, Phase 3]
A multi-cohort Phase 2/3 trial is evaluating safety, efficacy, pharmacokinetics and pharmacodynamics of GNR-055 in patients with Mucopolysac…
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CLINICAL_TRIALS trial_result · 2025-07-24Clinical Trial Update: An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II [Phase 2]
Phase 2 extension study update for JR-141-BR21 in patients with Mucopolysaccharidosis II; the study is currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2025-01-28Clinical Trial Update: RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) [Phase 1, Phase 2]
A Phase 1/2 clinical trial update for RGX-121 gene therapy in children aged 5 and older with Mucopolysaccharidosis Type II (Hunter syndrome)…
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CLINICAL_TRIALS trial_result · 2025-01-23Clinical Trial Update: A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India [Phase 4]
This Phase 4 trial update reports completion of a study evaluating Elaprase (idursulfase) in children and adults with Hunter syndrome in Ind…
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CLINICAL_TRIALS trial_result · 2024-10-28Clinical Trial Update: Hunter Outcome Survey (HOS)
Clinical trial update for the Hunter Outcome Survey (HOS) sponsored by Shire, tracking outcomes in Mucopolysaccharidosis type II (Hunter syn…
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CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
This clinical trial update reports on a longitudinal study of bone disease in children with mucopolysaccharidoses types I, II, and VI. The s…
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CLINICAL_TRIALS trial_result · 2024-10-15Clinical Trial Update: A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ [Phase 1]
A Phase 1 clinical trial is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in …
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: MPS II Immunophenotyping
A Duke University-sponsored trial investigating immunophenotyping in mucopolysaccharidosis type II (Hunter syndrome) has been terminated.
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CLINICAL_TRIALS trial_result · 2024-07-03Clinical Trial Update: A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers [Phase 1]
Phase 1 trial evaluating tolerance, safety, and pharmacokinetics of GNR-055 in healthy volunteers has completed; the study is associated wit…
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CLINICAL_TRIALS trial_result · 2024-06-10Clinical Trial Update: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
Denali Therapeutics completed a clinical trial in Mucopolysaccharidosis type II investigating potential treatment-responsive biomarkers and …
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CLINICAL_TRIALS trial_result · 2024-05-03Clinical Trial Update: Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Long-term follow-up study update for subjects treated with Sangamo's SB-318, SB-913, or SB-FIX, evaluating ongoing outcomes in hemophilia B …
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CLINICAL_TRIALS trial_result · 2022-11-14Clinical Trial Update: A Study of JR-141 in Patients With Mucopolysaccharidosis Type II [Phase 1, Phase 2]
Phase 1/2 trial of JR-141 in Mucopolysaccharidosis II has completed.
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CLINICAL_TRIALS trial_result · 2017-12-13Clinical Trial Update: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
A clinical trial examining parental coping with challenging behavior in children with mucopolysaccharidosis types I–III has completed.
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CLINICAL_TRIALS trial_result · 2017-09-15Clinical Trial Update: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI [Phase 1, Phase 2]
Clinical trial evaluating adalimumab in mucopolysaccharidosis types I, II, and VI has completed phases 1 and 2 with results posted in 2017.
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CLINICAL_TRIALS trial_result · 2014-07-08Clinical Trial Update: Safety and Efficacy of Hunterase [Phase 3]
Phase 3 clinical trial of Hunterase for mucopolysaccharidosis type II has completed; results are not disclosed in the summary.
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CLINICAL_TRIALS trial_result · 2012-02-14Clinical Trial Update: To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients [Phase 1, Phase 2]
Clinical trial update announces completion of Phase 1/2 evaluating GC1111 for Hunter syndrome (Mucopolysaccharidosis type II) focusing on sa…