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Results for “Hurler Disease”

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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
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 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
Early research (Phase 1)Looking for participantsNCT06519552
What this trial is testing

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I

Who this might be right for
Mucopolysaccharidosis Type I
West China Hospital 6
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
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
Not applicableUnknownNCT04958070
What this trial is testing

The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan

Who this might be right for
MPS I
Mackay Memorial Hospital 20
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
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
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 completedNCT02067650
What this trial is testing

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

Who this might be right for
MPS IMPS IV
Children's Hospital of Eastern Ontario 18
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
Testing effectiveness (Phase 2)WithdrawnNCT05665036
What this trial is testing

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

Who this might be right for
MPS IMPS1-SMPS1-HS
Sigilon Therapeutics, Inc.
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
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
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 applicableStudy completedNCT03576729
What this trial is testing

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

Who this might be right for
Mucopolysaccharidosis Type I
University of Minnesota 30
Early research (Phase 1)Study completedNCT01173016
What this trial is testing

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Who this might be right for
Hurler Syndrome
Masonic Cancer Center, University of Minnesota 11
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
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