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Results for “Mucopolysaccharidosis II (MPS II)”

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Showing 18 of 0 results

Testing effectiveness (Phase 2)Ended earlyNCT03315182
What this trial is testing

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Who this might be right for
Mucopolysaccharidosis Type 3 B
Abeona Therapeutics, Inc 11
Testing effectiveness (Phase 2)Ended earlyNCT02618512
What this trial is testing

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Who this might be right for
Mucopolysaccharidosis III, Type B (MPS IIIB)Sanfilippo B
Alexion Pharmaceuticals, Inc. 3
Not applicableStudy completedNCT05073783
What this trial is testing

Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting

Who this might be right for
Pompe DiseaseMucopolysaccharidosis Type I (MPS I)
Sanofi 57
Testing effectiveness (Phase 2)Active Not RecruitingNCT04201405
What this trial is testing

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis Type IIIA
University of Manchester 5
Large-scale testing (Phase 3)Study completedNCT00069641
What this trial is testing

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Who this might be right for
Mucopolysaccharidosis II
Shire 96
Large-scale testing (Phase 3)Study completedNCT07344376
What this trial is testing

An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)

Who this might be right for
MPS IIHunter Syndrome (MPS II)Hunter Syndrome+2 more
GC Biopharma Corp 30
Testing effectiveness (Phase 2)Ended earlyNCT03041324
What this trial is testing

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Who this might be right for
Mucopolysaccharidosis IIMPS II
Sangamo Therapeutics 9
Large-scale testing (Phase 3)Looking for participantsNCT02716246
What this trial is testing

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 36
Not applicableStudy completedNCT01521429
What this trial is testing

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Who this might be right for
Mucopolysaccharidoses
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 55
Testing effectiveness (Phase 2)Study completedNCT00920647
What this trial is testing

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®

Who this might be right for
Hunter Syndrome
Shire 16
Testing effectiveness (Phase 2)Study completedNCT02324049
What this trial is testing

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis IIIB
Alexion Pharmaceuticals, Inc. 11
Large-scale testing (Phase 3)Study completedNCT03568175
What this trial is testing

JR-141 in Patients With Mucopolysaccharidosis II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 28
Not applicableStudy completedNCT06022380
What this trial is testing

Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II

Who this might be right for
Mucopolysaccharidosis Type II
Manchester University NHS Foundation Trust 46
Early research (Phase 1)Study completedNCT02262338
What this trial is testing

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
ArmaGen, Inc 6
Not applicableEnded earlyNCT01675674
What this trial is testing

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

Who this might be right for
MucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis II+2 more
National MPS Society 3,000
Testing effectiveness (Phase 2)Study completedNCT03359213
What this trial is testing

JR-141 in Patients With Mucopolysaccharidosis II (BR21)

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 18
Not applicableEnded earlyNCT04976231
What this trial is testing

MPS II Immunophenotyping

Who this might be right for
MPS IIMucopolysaccharidosis IIHunter Syndrome
Duke University 6
Large-scale testing (Phase 3)Study completedNCT02412787
What this trial is testing

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

Who this might be right for
Hunter Syndrome
Shire 56