Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01626092
• Date of registration: 20/06/2012
• Prospective Registration: Yes
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
• Scientific title: Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)
• Date of first enrolment: July 11, 2012
• Target sample size: 3
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01626092
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Weston Miller, MD
• Affiliation: Masonic Cancer Center, University of Minnesota

Key inclusion & exclusion criteria

Inclusion Criteria:

- Adrenoleukodystrophy (ALD): Patients from 0-55 years of age diagnosed with ALD as
determined by very long chain fatty acid testing will be eligible for this protocol if
they have evidence of cerebral or cerebellar disease based on MRI testing,

- Metachromatic Leukodystrophy (MLD): Patients from 0-55 years of age diagnosed with MLD
as determined by arylsulfatase A activity.

- Globoid Cell Leukodystrophy (GLD): Patients from 0-55 years of age diagnosed with GLD
as determined by galactocerebrosidase activity will be eligible for this protocol

- Wolman's disease, GM1 gangliosidosis, Tay Sachs disease, Sanfilippo syndrome, Sandhoff
disease or other inherited metabolic diseases including but not limited to I-cell
disease (mucolipidosis II): Patients who are determined to be sufficiently advanced or
high risk based on the following reasons:

- Symptomatic disease, as based on neurologic examination or evidence of
deterioration based on subsequent neuropsychologic evaluations.

- Evidence of an expected poor outcome based on genetic testing or a prior family
history of aggressive disease.

- Other metabolic disorders, including but not limited to I-cell disease, that are
deemed to be high-risk for a poor outcome with a standard transplant regimen due
to anticipated toxicity based on experience gained at the University of Minnesota
or other centers.

- Donor Availability

- Transplantation using sufficiently matched related donors (such as matched
siblings) or unrelated donors will be considered. Both granulocyte-colony
stimulating factor (G-CSF) stimulated peripheral blood grafts and bone marrow
grafts will be considered, although bone marrow will be the priority.

- Cord blood grafts, both related and unrelated, are also eligible. As this
protocol will use a reduced intensity regimen, this protocol will use the current
recommendations of the University of Minnesota for choosing cord blood grafts. If
a single cord blood unit cell dose is insufficient, double cord transplantation
should be considered if sufficiently matched cord blood units are available. The
priority of choosing cord blood donors is based on the current institutional
recommendations.

- Exclusion of Metabolic Disorder Carrier Status from related donor and unrelated
cord blood grafts as appropriate for primary disease.

- Adequate Organ Function - Measured within 30 days of study enrollment

- Signed consent

Exclusion Criteria:

- Inability to receive total body irradiation (TBI) with marrow boosting per protocol
guidelines as determined by the Radiation Oncologist

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

- Advanced Disease Exclusion: Following evaluation, if a consensus of the members of the
Inherited Metabolic and Storage Disease Program is that a patient is too advanced to
benefit in a measurable and meaningful way from transplant, this will be communicated
to the family, and transplant will not be offered.



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

Health Condition(s) or Problem(s) studied

Peroxisomal Disorder

Lysosomal Storage Disease

Intervention(s)

Biological: Hematopoietic stem cell transplantation

Drug: Campath-1H

Drug: Cyclosporine A

Drug: Melphalan

Drug: Clofarabine

Drug: Mycophenolate mofetil

Radiation: Total Body Irradiation with Marrow Boosting

Primary Outcome(s)

Donor (Allogeneic) Hematopoietic Engraftment [Time Frame: Day 100 Following Hematopoietic Cell Transplant (HCT)]

Secondary Outcome(s)

Transplant-Related Mortality [Time Frame: Day 100 following HCT]

Neurologic Outcomes [Time Frame: Changes from Baseline, Days 30, 60, 100, Year 1, Year 2, Year 3 Following HCT]

Secondary ID(s)

2011LS147

MT2011-24

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/10/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01626092