MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
1 other identifier
interventional
149
1 country
1
Brief Summary
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 busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Jul 2014
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 20, 2014
CompletedFirst Posted
Study publicly available on registry
June 23, 2014
CompletedStudy Start
First participant enrolled
July 10, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 5, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
July 14, 2029
ExpectedJanuary 7, 2026
January 1, 2026
11.5 years
June 20, 2014
January 5, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Percent of subjects who achieve high-level donor hematopoietic engraftment
Defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant
Day +42 post-transplant
Percent of subjects who achieve high-level donor hematopoietic engraftment
Defined as ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant
Day +100 post-transplant
Secondary Outcomes (5)
Graft-versus-host disease
Day +100 post-transplant
Transplant-related mortality
Day +100 post-transplant
Regimen-related toxicity
Day +100 post-transplant
Post-HSCT changes in disease
1 year
Post-HSCT changes in disease
2 years
Study Arms (7)
IMD - Except Haplo-identical
EXPERIMENTALInherited Metabolic Disease (IMD) - Except Haplo-Identical See intervention descriptions.
OP - Except Haplo-Identical
EXPERIMENTALSevere Osteoperosis (OP) - Except Haplo-Identical See intervention descriptions.
OP and IMD -Haplo-Identical Only
EXPERIMENTALSevere Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD) -Haplo-Identical Only See intervention descriptions.
cALD SR-A (Standard-Risk, Regimen A)
EXPERIMENTALSee intervention descriptions.
cALD SR-B (Standard-Risk, Regimen B)
EXPERIMENTALSee intervention descriptions.
cALD HR-C (High-Risk, Regimen C)
EXPERIMENTALSee intervention descriptions.
cALD HR-D (High-Risk, Regimen D)
EXPERIMENTALSee intervention descriptions.
Interventions
Infusion given on Day 0
* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan
* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Thiotepa
* Rituximab * Alemtuzumab * Busulfan * Fludarabine
N-acetylcysteine start day +1 through day +28
N-acetylcysteine start day +1through day +56
N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100
N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100
Eligibility Criteria
You may qualify if:
- through 55 years of age
- Adequate graft available
- Adequate organ function
- Eligible Diseases:
- Mucopolysaccharidosis Disorders:
- 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)
- Glycoprotein Metabolic Disorders:
- Alpha mannosidosis
- Fucosidosis
- Aspartylglucosaminuria
- Sphingolipidoses and Recessive Leukodystrophies:
- Globoid cell leukodystrophy
- +17 more criteria
You may not qualify if:
- Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
- Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)
- Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Paul Orchard, M.D.
Masonic Cancer Center, University of Minnesota
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 20, 2014
First Posted
June 23, 2014
Study Start
July 10, 2014
Primary Completion
January 5, 2026
Study Completion (Estimated)
July 14, 2029
Last Updated
January 7, 2026
Record last verified: 2026-01