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

• Register: ClinicalTrials.gov
• Last refreshed on: 13 November 2023
• Main ID: NCT02171104
• Date of registration: 20/06/2014
• Prospective Registration: Yes
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
• Scientific title: MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
• Date of first enrolment: July 10, 2014
• Target sample size: 100
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02171104
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Lisa Burke

• Name: Paul Orchard, M.D.
• Affiliation: Masonic Cancer Center, University of Minnesota

• Name: Lisa Burke

Key inclusion & exclusion 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)



Age minimum: N/A
Age maximum: 55 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fucosidosis

Hurler Syndrome

Mitochondrial Neurogastrointestingal Encephalopathy

D-Bifunctional Enzyme Deficiency

Niemann-Pick C Subtype 2

Recessive Leukodystrophies

Sly Syndrome

Adrenoleukodystrophy With Cerebral Involvement

Aspartylglucosaminuria

Severe Osteopetrosis

Sphingomyelin Deficiency

Acyl-CoA Oxidase Deficiency

Infantile Refsum Disease

Neonatal Adrenoleukodystrophy

Alpha-Mannosidosis

Maroteaux Lamy Syndrome

Metachromatic Leukodystrophy

Mucopolysaccharidosis Disorders

Multifunctional Enzyme Deficiency

Niemann-Pick B

Peroxisomal Disorders

Alpha-methylacyl-CoA Racmase Deficiency

Glycoprotein Metabolic Disorders

Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)

Hunter Syndrome

Sphingolipidoses

Globoid Cell Leukodystrophy

Zellweger Syndrome

Inherited Metabolic Disorders

Intervention(s)

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

Biological: Stem Cell Transplantation

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

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

Drug: Osteopetrosis Only Preparative Regimen

Drug: IMD Preparative Regimen

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

Drug: Osteopetrosis Haploidentical Only Preparative Regimen

Primary Outcome(s)

Percent of subjects who achieve high-level donor hematopoietic engraftment [Time Frame: Day +42 post-transplant]

Percent of subjects who achieve high-level donor hematopoietic engraftment [Time Frame: Day +100 post-transplant]

Secondary Outcome(s)

Graft-versus-host disease [Time Frame: Day +100 post-transplant]

Regimen-related toxicity [Time Frame: Day +100 post-transplant]

Transplant-related mortality [Time Frame: Day +100 post-transplant]

Post-HSCT changes in disease [Time Frame: 2 years]

Post-HSCT changes in disease [Time Frame: 1 year]

Secondary ID(s)

2013LS104

Source(s) of Monetary Support

Please refer to primary and secondary sponsors