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Results for “Mucopolysaccharidosis II”

110 trials

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Showing 20 of 110 results

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
Large-scale testing (Phase 3)Looking for participantsNCT05371613
What this trial is testing

Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 63
Not applicableEnrolling By InvitationNCT03655223
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
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
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
Early research (Phase 1)Study completedNCT06475404
What this trial is testing

The Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 18
Large-scale testing (Phase 3)Active Not RecruitingNCT06031259
What this trial is testing

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Who this might be right for
Hunter Syndrome
Takeda 6
Testing effectiveness (Phase 2)Study completedNCT00176891
What this trial is testing

Stem Cell Transplant w/Laronidase for Hurler

Who this might be right for
Mucopolysaccharidosis IHurler Syndrome
Masonic Cancer Center, University of Minnesota 25
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Testing effectiveness (Phase 2)Study completedNCT00176917
What this trial is testing

Stem Cell Transplantation for Hurler

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more
Masonic Cancer Center, University of Minnesota 41
Testing effectiveness (Phase 2)Study completedNCT04227600
What this trial is testing

JR-171 in Patients With Mucopolysaccharidosis I

Who this might be right for
Mucopolysaccharidosis I
JCR Pharmaceuticals Co., Ltd. 18
Large-scale testing (Phase 3)Enrolling By InvitationNCT05594992
What this trial is testing

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 80
Testing effectiveness (Phase 2)Active Not RecruitingNCT03708965
What this trial is testing

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 19
Testing effectiveness (Phase 2)Active Not RecruitingNCT06095388
What this trial is testing

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis III-A
JCR Pharmaceuticals Co., Ltd. 15
Not applicableLooking for participantsNCT06036693
What this trial is testing

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more
Institut National de la Santé Et de la Recherche Médicale, France 1,000
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
Not applicableStudy completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
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
Not applicableLooking for participantsNCT05408715
What this trial is testing

A Natural History Study in Children With a Type II Collagen Disorder With Short Stature

Who this might be right for
SEDCHypochondrogenesisSemd, Strudwick Type+1 more
Innoskel 60
Large-scale testing (Phase 3)Active Not RecruitingNCT07236606
What this trial is testing

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Who this might be right for
MPS IIHunter Syndrome (MPS II)
REGENXBIO Inc. 2
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