Brief Title
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Official Title
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
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).
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Percent of subjects who achieve high-level donor hematopoietic engraftment
Secondary Outcome
Graft-versus-host disease
Condition
Mucopolysaccharidosis Disorders
Intervention
Stem Cell Transplantation
Study Arms / Comparison Groups
IMD - Except Haplo-identical
Description: Inherited Metabolic Disease (IMD) - Except Haplo-Identical See intervention descriptions.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
100
Start Date
July 10, 2014
Completion Date
December 2022
Primary Completion Date
September 2022
Eligibility Criteria
Inclusion Criteria:
- 0 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
- Metachromatic leukodystrophy
- Niemann-Pick B patients (sphingomyelin deficiency)
- Niemann-Pick C subtype 2
- Peroxisomal Disorders:
- Adrenoleukodystrophy with cerebral involvement
- Zellweger syndrome
- Neonatal Adrenoleukodystrophy
- Infantile Refsum disease
- Acyl-CoA-Oxidase Deficiency
- D-Bifunctional enzyme deficiency
- Multifunctional enzyme deficiency
- Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)
- Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
- Severe Osteopetrosis (OP)
- Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
- Other Inherited Metabolic Disorders (IMD): Patients will also be considered who
have other life-threatening, rare lysosomal, peroxisomal or other similar
inherited disorders characterized by white matter disease or other neurologic
manifestations for which there is rationale that transplantation would be of
benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis,
I-cell disease, Tay-Sachs disease, Sandhoff disease or others.
- Voluntary written consent
Exclusion Criteria:
- 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)
Gender
All
Ages
N/A - 55 Years
Accepts Healthy Volunteers
No
Contacts
Paul Orchard, M.D., 612-273-8482, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02171104
Organization ID
2013LS104
Responsible Party
Sponsor
Study Sponsor
Masonic Cancer Center, University of Minnesota
Study Sponsor
Paul Orchard, M.D., Principal Investigator, Masonic Cancer Center, University of Minnesota
Verification Date
May 2021