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Results for “Mucopolysaccharidosis Type I (MPS I)”

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Showing 20 of 0 results

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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04201405
What this trial is testing

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis Type IIIA
University of Manchester 5
Large-scale testing (Phase 3)Study completedNCT00146770
What this trial is testing

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 45
Testing effectiveness (Phase 2)Active Not RecruitingNCT03488394
What this trial is testing

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

Who this might be right for
Mucopolysaccharidosis IH
Orchard Therapeutics 8
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
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
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)Study completedNCT01155778
What this trial is testing

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 12
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
Not applicableActive Not RecruitingNCT03655223
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
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
Not applicableStudy completedNCT00144794
What this trial is testing

Mucopolysaccharidosis I (MPS I) Registry

Who this might be right for
Mucopolysaccharidosis I (MPS I)
Genzyme, a Sanofi Company 1,406
Not applicableStudy completedNCT00852358
What this trial is testing

Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Who this might be right for
Cognitive DeclineMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 9
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
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableEnded earlyNCT01675674
What this trial is testing

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

Who this might be right for
MucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis II+2 more
National MPS Society 3,000
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
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
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
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