2026-01-05
notable
Recruiting→Active, Not Recruiting Study Status, Study Design, Contacts/Locations v23
Trial statusRecruiting→Active, Not Recruiting
Primary completion dateestimated 2026-07-14→confirmed 2026-01-05
Enrollment targetestimated 100→confirmed 149
Study sitesdetails revised at 1 of 1 site
Show 17 earlier versions
2021-05-21
minor
Study Status, Contacts/Locations v17
Study sitescontacts updated at 1 of 1 site
2020-09-28
minor
Study Status v16
Primary completion date2020-09→2021-09
Completion date2020-09→2021-12
2019-09-25
minor
Study Status v15
Primary completion date2019-09→2020-09
Completion date2019-09→2020-09
2018-09-25
minor
3 amendments v12-v14
3 re-verifications since 2017-12-03, no change to tracked fields
2017-10-24
minor
Study Status, Eligibility v11
Eligibility criteria+1 characters
[...] ders (IMD): Patients will also be considered who have other lifethreateninglife-threatening, rare lysosomal, peroxisomal or other similar inherited dis [...]
2017-10-04
minor
Study Status v10
Re-verified, no change to tracked fields
2017-06-15
minor
Study Status, Conditions, Arms and Interventions, Outcome Measures, Eligibility v9
Eligibility criteria+42 characters
[...] function
Eligible Diseases:
Mucopolysaccharidosis Disorders ("IMD"):
MPS IH (Hurler syndrome)
MPS II (Hunter syndrome) if the p [...] ome)
MPS VII (Sly syndrome)
Glycoprotein Metabolic Disorders ("IMD"):
Alpha mannosidosis
Fucosidosis
Aspartylglucosaminuria
Sphingolipidoses and Recessive Leukodystrophies ("IMD"):
Globoid cell leukodystrophy
Metachromatic leukodystrophy
N [...] n deficiency)
Niemann-Pick C subtype 2
Peroxisomal Disorders ("IMD"):
Adrenoleukodystrophy with cerebral involvement
Zellweger s [...] ell disease, Tay-Sachs disease, Sandhoff disease or others.
Voluntary written consent
Exclusion Criteria: [...] [...]
Secondary endpoints4 to 5 entries
percentage of subjects who develop graftGraft-versus-host disease
numberTransplant-related of subjects who die from transplant complicationsmortality
incidence of regimenRegimen-related toxicity
Post-HSCT (suchchanges as infection, acute renal failure, respiratory failure, cardiac failure, and veno-occlusivein disease)
incidence of radiographic, physiologic, neuroPost-psychologic, and/or biochemicalHSCT changes in disease
Primary endpoints1 to 2 entries
percentagePercent of subjects who achieve high-level donor hematopoietic engraftment
Percent (definedof assubjects neutrophilwho recoveryachieve by Day +42 posthigh-transplant and ≥ 80%level donor cellshematopoietic on the myeloid fraction of peripheral blood at Day +100 post-transplant)engraftment
2017-03-22
minor
Study Status, Study Design, Arms and Interventions, Contacts/Locations v8
Study arms3→7
InterventionsIMD Preparative Regimen (Drug), Osteopetrosis Haploidentical Only Preparative Regimen (Drug), Osteopetrosis Only Preparative Regimen…→IMD Preparative Regimen (Drug), Osteopetrosis Haploidentical Only Preparative Regimen (Drug), Osteopetrosis Only Preparative Regimen…
Study sites1 entry, revised
Masonic Cancer Center, University of Minnesota Medical Center, Fairview
2017-02-17
minor
Study Status, Oversight, Study Description, Conditions, Study Design, Arms and Interventions, Eligibility, Contacts/Locations v7
Start date2014-07→2014-07-10
Study arms1→3
InterventionsAlpha Lipoic Acid (Drug), Busulfan (Drug), Celecoxib (Drug), Cyclophosphamide (Drug), Cyclosporine A (CSA) (Drug), Fludarabine (Drug)…→IMD Preparative Regimen (Drug), Osteopetrosis Haploidentical Only Preparative Regimen (Drug), Osteopetrosis Only Preparative Regimen…
Eligibility criteria+1,481 characters
Inclusion Criteria:
0 through 55 years of age
Adequate graft available
Adequate organ function
Eligible Diseases:
Mucopolysaccharidosis Disorders ("IMD"):
MPS IH (Hurler syndrome)
MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
MPS VI (Maroteaux-Lamy syndrome)
MPS VII (Sly syndrome)
Glycoprotei [...] Exclusion Criteria:
Any patient who in the judgment of the U [...]
Study description-571 characters
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using a busulfan- and fludarabine-based conditioning regimen with busulfan therapeutic drug monitoring (TDM) for patien [...]
Study sitescontacts updated at 1 of 1 site
2016-08-15
minor
2 amendments v5-v6
2 re-verifications since 2016-01-22, no change to tracked fields
2015-06-19
minor
Study Identification, Study Status, Conditions, Arms and Interventions v4
Study title-18 characters
MT2013-31: Allo BMTHCT for Metabolic Disorders, Osteopetrosis and MalesSevere With Rett SyndromeOsteopetrosis
2015-04-22
minor
Study Status v3
Re-verified, no change to tracked fields
2014-07-18
minor
Study Status, Study Description, Conditions, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v2
InterventionsBusulfan (Drug), Cyclosporine A (Drug), Fludarabine monophosphate (Drug), Granulocyte-Colony Stimulating Factor (Drug), Methylprednisolone…→Alpha Lipoic Acid (Drug), Busulfan (Drug), Celecoxib (Drug), Cyclophosphamide (Drug), Cyclosporine A (CSA) (Drug), Fludarabine (Drug)…
Secondary endpoints3 to 4 entries
incidence of radiographic, physiologic, neuro-psychologic, and/or biochemical changes
Study description+150 characters
[...] ing low rates of transplant-related mortality (TRM) using a single daily dose busulfan- and fludarabine-based conditioning regimen with busulfan therapeutic drug monitoring (TDM) for patients with variouscertain inherited metabolic disorders (IMD), severe osteopetrosis and males with Rett syndrome.
Additionally, this study will explore the efficacy of various anti-oxidant and anti-inflammatory regimens in patients undergoing HCT for cerebral adrenoleukodystrophy.
Study sitescontacts updated at 1 of 1 site
2014-07-10
notable
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Start date2014-06→2014-07
Study sitesdetails revised at 1 of 1 site