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Results for “Mucopolysaccharidosis II”

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Showing 20 of 0 results

Not applicableEnded earlyNCT02095015
What this trial is testing

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 159
Testing effectiveness (Phase 2)Active Not RecruitingNCT05665166
What this trial is testing

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
University of Manchester 5
Testing effectiveness (Phase 2)Study completedNCT03173521
What this trial is testing

Gene Therapy in Patients With Mucopolysaccharidosis Disease

Who this might be right for
Mucopolysaccharidosis Type VI
Fondazione Telethon 9
Early research (Phase 1)Study completedNCT00692926
What this trial is testing

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

Who this might be right for
MDSAnemia, AplasticInborn Errors of Metabolism+2 more
Joanne Kurtzberg, MD 37
Testing effectiveness (Phase 2)Active Not RecruitingNCT03488394
What this trial is testing

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

Who this might be right for
Mucopolysaccharidosis IH
Orchard Therapeutics 8
Testing effectiveness (Phase 2)Active Not RecruitingNCT03153319
What this trial is testing

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 14
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Not applicableStudy completedNCT00882921
What this trial is testing

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Who this might be right for
Hunter Syndrome
Shire 26
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40
Early research (Phase 1)Active Not RecruitingNCT05422482
What this trial is testing

Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

Who this might be right for
Mucopolysaccharidosis IIHunter Syndrome
GC Biopharma Corp 12
Large-scale testing (Phase 3)Study completedNCT02055118
What this trial is testing

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

Who this might be right for
Hunter Syndrome
Shire 58
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Large-scale testing (Phase 3)Study completedNCT00630747
What this trial is testing

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Who this might be right for
Hunter SyndromeMucopolysaccharidosis II (MPS II)
Shire 94
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT03529786
What this trial is testing

Mucopolysaccharidosis Type II Natural History

Who this might be right for
Mucopolysaccharidosis II
REGENXBIO Inc. 36
Testing effectiveness (Phase 2)Active Not RecruitingNCT04251026
What this trial is testing

Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 47
Testing effectiveness (Phase 2)Study completedNCT00146757
What this trial is testing

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

Who this might be right for
Mucopolysaccharidosis IHurler SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 20
Large-scale testing (Phase 3)Study completedNCT03920540
What this trial is testing

GC1111 in Hunter Syndrom Patients

Who this might be right for
Hunter Syndrome
Green Cross Corporation 32
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
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