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Results for “Mucopolysaccharidosis Type I”

97 trials

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Showing 20 of 97 results

Post-approval studies (Phase 4)Study completedNCT00144781
What this trial is testing

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 34
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)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
Post-approval studies (Phase 4)Ended earlyNCT00418821
What this trial is testing

The Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 2
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
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 applicableEnded earlyNCT01572636
What this trial is testing

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Who this might be right for
Mucopolysaccharidosis Type IHMPS IHurler Syndrome
Masonic Cancer Center, University of Minnesota 20
Not applicableStudy completedNCT01873911
What this trial is testing

Neurobehavioral Phenotypes in MPS III

Who this might be right for
Sanfilippo Syndrome Type ASanfilippo Syndrome Type BHurler Syndrome
University of Minnesota 30
Testing effectiveness (Phase 2)Study completedNCT03411161
What this trial is testing

S 81694 Plus Paclitaxel in Metastatic Breast Cancer

Who this might be right for
Metastatic Breast CancerMetastatic Triple Negative Breast Cancer
Institut de Recherches Internationales Servier 22
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 applicableNo Longer AvailableNCT03639844
What this trial is testing

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Who this might be right for
Hurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more
Bellicum Pharmaceuticals
Not applicableWithdrawnNCT02298712
What this trial is testing

Biomarker for Hurler Disease (BioHurler)

Who this might be right for
Mucopolysaccharidosis Type IGargoylismMetabolism, Inborn Errors
CENTOGENE GmbH Rostock
Not applicableStudy completedNCT00695279
What this trial is testing

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Who this might be right for
Severe Combined ImmunodeficiencyMalignancy, HematologicNeuroblastoma+2 more
St. Jude Children's Research Hospital 41
Large-scale testing (Phase 3)Active Not RecruitingNCT06149403
What this trial is testing

Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Who this might be right for
MPS-IH (Hurler Syndrome)
Orchard Therapeutics 41
Not applicableEnded earlyNCT02232477
What this trial is testing

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Who this might be right for
Mucopolysaccharidosis ICognitive Decline
agnes chen 6
Early research (Phase 1)Ended earlyNCT00215527
What this trial is testing

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Who this might be right for
Mucopolysaccharidosis ILysosomal Storage DiseasesSpinal Cord Compression
Patricia I. Dickson, M.D. 4
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
Early research (Phase 1)Ended earlyNCT00786968
What this trial is testing

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Who this might be right for
Spinal Cord CompressionMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 3
Post-approval studies (Phase 4)Study completedNCT00144768
What this trial is testing

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 25
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