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

Launched by MASONIC CANCER CENTER, UNIVERSITY OF MINNESOTA · Jun 20, 2014

Keywords

ClinConnect Summary

The clinical trial named MT2013-31 is investigating a treatment method called donor hematopoietic cell transplantation (HCT) for patients with certain inherited metabolic disorders and severe osteopetrosis. The study aims to see if this approach can successfully help patients while keeping the risk of serious complications from the transplant low. Participants in this trial may include individuals aged 0 to 55 years who have specific conditions like Hurler syndrome, Hunter syndrome, or other rare metabolic disorders, and who have a suitable organ function and donor match.

If you or a loved one are eligible and choose to participate, you can expect to receive a specific type of chemotherapy before the transplant to prepare your body. This trial is currently open for recruitment, meaning they are looking for volunteers. It's important to note that certain factors, like being pregnant or having severe infections, may exclude someone from participating. The study will provide careful monitoring and support throughout the process to ensure the best possible care.

Gender

ALL

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)