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Results for “Mucopolysaccharidosis Type I”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Ended earlyNCT02702115
What this trial is testing

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

Who this might be right for
MPS I
Sangamo Therapeutics 3
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 completedNCT02437253
What this trial is testing

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
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
Testing effectiveness (Phase 2)Study completedNCT03071341
What this trial is testing

Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

Who this might be right for
Mucopolysaccharidosis I
ArmaGen, Inc 19
Not applicableStudy completedNCT01521429
What this trial is testing

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Who this might be right for
Mucopolysaccharidoses
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 55
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
Testing effectiveness (Phase 2)Study completedNCT03053089
What this trial is testing

Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I

Who this might be right for
Mucopolysaccharidosis I
ArmaGen, Inc 21
Testing effectiveness (Phase 2)Study completedNCT03300453
What this trial is testing

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Who this might be right for
Sanfilippo Syndrome B
UniQure Biopharma B.V. 4
Not applicableActive Not RecruitingNCT05634512
What this trial is testing

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Who this might be right for
Hematopoietic Cell TransplantationMucopolysaccharidosis Type I
Masonic Cancer Center, University of Minnesota 13
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)Study completedNCT00741338
What this trial is testing

Immune Tolerance Study With Aldurazyme® (Laronidase)

Who this might be right for
Mucopolysaccharidosis I
Genzyme, a Sanofi Company 7
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 applicableWithdrawnNCT02298647
What this trial is testing

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

Who this might be right for
Hepato-splenomegalyDysostosis MultiplexSeizures+1 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT01474343
What this trial is testing

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
Early research (Phase 1)Study completedNCT01917708
What this trial is testing

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Who this might be right for
Hurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more
Emory University 10
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
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
Not applicableEnrolling By InvitationNCT06103487
What this trial is testing

Long Term Follow-Up for RGX-111

Who this might be right for
Mucopolysaccharidosis I
REGENXBIO Inc. 21
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
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