NCT02171104View on ClinicalTrials.gov

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

PHASE2RecruitingINTERVENTIONAL
Enrollment

100

Participants

Timeline

Start Date

July 10, 2014

Primary Completion Date

July 14, 2026

Study Completion Date

July 14, 2029

Conditions
Mucopolysaccharidosis DisordersHurler SyndromeHunter SyndromeMaroteaux Lamy SyndromeSly SyndromeAlpha-MannosidosisFucosidosisAspartylglucosaminuriaGlycoprotein Metabolic DisordersSphingolipidosesRecessive LeukodystrophiesGloboid Cell LeukodystrophyMetachromatic LeukodystrophyNiemann-Pick BNiemann-Pick C Subtype 2Sphingomyelin DeficiencyPeroxisomal DisordersAdrenoleukodystrophy With Cerebral InvolvementZellweger SyndromeNeonatal AdrenoleukodystrophyInfantile Refsum DiseaseAcyl-CoA Oxidase DeficiencyD-Bifunctional Enzyme DeficiencyMultifunctional Enzyme DeficiencyAlpha-methylacyl-CoA Racmase DeficiencyMitochondrial Neurogastrointestingal EncephalopathySevere OsteopetrosisHereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)Inherited Metabolic Disorders
Interventions
BIOLOGICAL

Stem Cell Transplantation

Infusion given on Day 0

DRUG

IMD Preparative Regimen

"* Anti-thymocyte Globulin (ATG)~* Fludarabine~* Busulfan"

DRUG

Osteopetrosis Only Preparative Regimen

"* Anti-thymocyte Globulin (ATG)~* Fludarabine~* Busulfan~* Thiotepa"

DRUG

Osteopetrosis Haploidentical Only Preparative Regimen

"* Rituximab~* Alemtuzumab~* Busulfan~* Fludarabine"

DRUG

cALD SR-A (Standard-Risk, Regimen A)

N-acetylcysteine start day +1 through day +28

DRUG

cALD SR-B (Standard-Risk, Regimen B)

N-acetylcysteine start day +1through day +56

DRUG

cALD HR-D (High-Risk, Regimen C)

N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100

DRUG

cALD HR-D (High-Risk, Regimen D)

N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100

Trial Locations (1)

55455

RECRUITING

Masonic Cancer Center, University of Minnesota, Minneapolis

All Listed Sponsors
lead

Masonic Cancer Center, University of Minnesota

OTHER