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Results for “Mucopolysaccharidosis II”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Testing effectiveness (Phase 2)Looking for participantsNCT07640984
What this trial is testing

A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis IIIB
JCR Pharmaceuticals Co., Ltd. 12
Testing effectiveness (Phase 2)Study completedNCT04571970
What this trial is testing

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 6
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
Large-scale testing (Phase 3)Active Not RecruitingNCT04348136
What this trial is testing

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

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 27
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 completedNCT00744692
What this trial is testing

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Who this might be right for
Non Malignant DisordersImmunodeficienciesCongenital Marrow Failures+5 more
Duke University 22
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
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
Not applicableStudy completedNCT01870375
What this trial is testing

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more
University of Minnesota 100
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Not applicableStudy completedNCT05155488
What this trial is testing

Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Who this might be right for
Mucopolysaccharidosis (MPS)
Takeda 250
Testing effectiveness (Phase 2)Temporarily pausedNCT03580083
What this trial is testing

RGX-111 Gene Therapy in Patients With MPS I

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Hurler SyndromeHurler-Scheie Syndrome
REGENXBIO Inc. 21
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
Large-scale testing (Phase 3)Study completedNCT04759885
What this trial is testing

Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep®

Who this might be right for
Elective Colonoscopy
NTC srl 886
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
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
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
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
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
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