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Results for “Mucolipidoses”

16 trials

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Showing 16 of 16 results

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
Early research (Phase 1)Enrolling By InvitationNCT07398872
What this trial is testing

Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV

Who this might be right for
Mucolipidosis Type IV
The Children's Hospital of Zhejiang University School of Medicine 1
Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableWithdrawnNCT02385162
What this trial is testing

Biomarker for Glycogen Storage Diseases (BioGlycogen)

Who this might be right for
Fructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more
CENTOGENE GmbH Rostock
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
Not applicableStudy completedNCT00015782
What this trial is testing

The Natural History and Pathogenesis of Mucolipidosis Type IV

Who this might be right for
Mucolipidosis Type IV
National Institute of Neurological Disorders and Stroke (NINDS) 30
Not applicableWithdrawnNCT02298673
What this trial is testing

Biomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML)

Who this might be right for
Skeletal AbnormalitiesPsychomotor Retardation
CENTOGENE GmbH Rostock
Not applicableLooking for participantsNCT05782387
What this trial is testing

Mucolipidosis Type IV Natural History Study

Who this might be right for
Mucolipidosis Type IV
Massachusetts General Hospital 50
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Not applicableStudy completedNCT01891422
What this trial is testing

Longitudinal Studies of the Glycoproteinoses

Who this might be right for
AspartylglucosaminuriaFucosidosisGalactosialidosis+6 more
Greenwood Genetic Center 100
Not applicableEnded earlyNCT01067742
What this trial is testing

The Natural History of Mucolipidosis Type IV

Who this might be right for
Mucolipidosis Type IV
Baylor Research Institute 7
Testing effectiveness (Phase 2)Study completedNCT00176917
What this trial is testing

Stem Cell Transplantation for Hurler

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more
Masonic Cancer Center, University of Minnesota 41
Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Not applicableLooking for participantsNCT06316752
What this trial is testing

The Natural History of Sialidosis Type I

Who this might be right for
Observational Study
National Taiwan University Hospital 30
Not applicableLooking for participantsNCT00029965
What this trial is testing

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Who this might be right for
Neurological RegressionMyoclonusCherry Red Spot+1 more
National Human Genome Research Institute (NHGRI) 200