Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01043640
• Date of registration: 05/01/2010
• Prospective Registration: No
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
• Scientific title: Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
• Date of first enrolment: December 2009
• Target sample size: 46
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01043640
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Paul Orchard, MD
• Affiliation: Masonic Cancer Center, University of Minnesota

Key inclusion & exclusion criteria

Inclusion Criteria:

- Must have diagnosis of one of the following: mucopolysaccharidosis disorder,
glycoprotein metabolic disorder, sphingolipidoses or inherited leukodystrophy,
peroxisomal disorder or other inherited diseases of metabolism

- Must have an acceptable graft source as defined by University of Minnesota criteria

- Adequate organ function

Exclusion Criteria:

- Pregnant - menstruating females must have a negative serum pregnancy test within 14
days of treatment start

- Evidence of human immunodeficiency virus (HIV) infection or known HIV positive
serology



Age minimum: N/A
Age maximum: 21 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fucosidosis

Aspartylglucosaminuria

Mucopolysaccharidosis

Hunter Syndrome

Metachromatic Leukodystrophy (MLD)

Peroxisomal Disorders

Sphingolipidoses

Hurler Syndrome

Adrenoleukodystrophy (ALD)

Krabbe Disease

Alpha Mannosidosis

Sly Syndrome

Maroteaux-Lamy Syndrome

Intervention(s)

Drug: Campath-1H

Drug: Mycophenolate Mofetil

Procedure: Allogeneic stem cell transplantation

Drug: Busulfan

Drug: Cyclophosphamide

Drug: Cyclosporine A

Primary Outcome(s)

Number of Patients With Donor Derived Engraftment [Time Frame: Day 100 Post Transplant]

Secondary Outcome(s)

Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) [Time Frame: Day 100 Post Transplant]

Donor Cell Chimerism Following Transplant [Time Frame: Day 28]

Donor Cell Chimerism Following Transplant [Time Frame: One year]

Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) [Time Frame: Day 100 Post Transplant]

Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) [Time Frame: Day 100 Post Transplant]

Donor Cell Chimerism Following Transplant [Time Frame: Day 100]

Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) [Time Frame: Day 100 Post Transplant]

Number of Patients Who Died Peri-Transplant [Time Frame: By Day 100 Post Transplant]

Donor Cell Chimerism Following Transplant [Time Frame: 6 months]

Donor Cell Chimerism Following Transplant [Time Frame: Day 42]

Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) [Time Frame: Day 100 Post Transplant]

Secondary ID(s)

2009LS088

MT2009-19

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/05/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01043640