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Results for “Niemann-Pick Disease Type B”

12 trials

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Showing 12 of 12 results

Not applicableUnknownNCT05914727
What this trial is testing

Biomarkers Of Lung InVolvement In ASMD

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 68
Not applicableUnknownNCT05904366
What this trial is testing

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 34
Not applicableEnrolling By InvitationNCT03655223
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
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
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
Early research (Phase 1)Study completedNCT01747135
What this trial is testing

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

Who this might be right for
Niemann-Pick Disease, Type C1
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company 14
Not applicableNot Yet RecruitingNCT06869499
What this trial is testing

Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease

Who this might be right for
SplenomegalySplenectomyThrombopenia+2 more
Wladimir MAUHIN, Dr 200
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 applicableNot Yet RecruitingNCT06985212
What this trial is testing

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)Niemann Pick Disease
Wladimir MAUHIN, Dr 200
Testing effectiveness (Phase 2)Ended earlyNCT03887533
What this trial is testing

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

Who this might be right for
Niemann-Pick Disease, Type C1
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 2
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Large-scale testing (Phase 3)Active Not RecruitingNCT04860960
What this trial is testing

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 94