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Results for “Mucopolysaccharidosis II (MPS II)”

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Enrolling by Invitation

Showing 20 of 0 results

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
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
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 completedNCT01645189
What this trial is testing

Safety and Efficacy of Hunterase

Who this might be right for
Hunter Syndrome
Green Cross Corporation 6
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Ended earlyNCT04088734
What this trial is testing

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 5
Testing effectiveness (Phase 2)Study completedNCT01506141
What this trial is testing

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

Who this might be right for
Hunter Syndrome
Takeda 15
Large-scale testing (Phase 3)Ended earlyNCT00748969
What this trial is testing

Clinical Trial of Growth Hormone 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 2
Large-scale testing (Phase 3)Active Not RecruitingNCT03566043
What this trial is testing

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 48
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 completedNCT04007536
What this trial is testing

Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 18
Early research (Phase 1)WithdrawnNCT05238324
What this trial is testing

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Who this might be right for
Mucopolysaccharidosis II
Homology Medicines, Inc
Not applicableStudy completedNCT03582449
What this trial is testing

Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Who this might be right for
Hunter Syndrome
Shire 5
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 completedNCT03128593
What this trial is testing

JR-141 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 14
Not applicableStudy completedNCT01449240
What this trial is testing

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Who this might be right for
Hunter Syndrome
Shire 10
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableStudy completedNCT01822184
What this trial is testing

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

Who this might be right for
Mucopolysaccharidosis (MPS)Hunter Syndrome
Shire 100
Not applicableStudy completedNCT03161171
What this trial is testing

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more
University Hospital Heidelberg 34
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
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