Cerebral Adrenoleukodystrophy
Conditions
Keywords
Cerebral Adrenoleukodystrophy, cALD
Brief summary
The purpose of this study is to determine the maximum tolerated dose (MTD) of mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).
Detailed description
This is a single-institution dose escalation study to determine the maximum tolerated dose (MTD) of intrathecally administered allogeneic, 3rd party mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).
Interventions
BIOLOGICALMesenchymal Stem Cells
Sponsors
Masonic Cancer Center, University of Minnesota
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age ≥ 4 years at time of study enrollment * Diagnosis of ALD - the diagnosis of ALD can be made by either biochemical or molecular/genetic evidence (plasma VLCFA or ABCD1 mutation analysis) supportive of ALD as the cause of cerebral demyelination * Evidence of active cerebral disease - defined as the presence of gadolinium enhancement on a single brain MRI study - MRI used for eligibility determination may be performed at an outside institution; the most recent MRI used to determine eligibility must be within 2 calendar months of the date of enrollment on this study * ALD MRI (Loes) score ≥ 10 * Off of N-acetylcysteine, systemic immunosuppressive drugs or any other therapeutic intervention (except hydrocortisone and/or fludracortisone for the treatment of adrenal insufficiency) for ≥ 10 days prior to the first IT-MSC dose * Life expectancy of \>6 months as determined by the enrolling researcher and documented in the medical record * Voluntary written consent provided by parent(s)/guardian(s)
Exclusion criteria
* A candidate for allogeneic hematopoietic stem cell transplantation as determined by the University of Minnesota Inherited Metabolic and Storage Disease group * Inability to undergo sedation, lumbar puncture or MRI studies for any reason * Inability to stay in Minnesota for therapy through the day 28 evaluation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Maximum Tolerated Dose | Day 28 post intrathecal injection of MSC | Determine the maximum tolerated dose (MTD) of intrathecally-administered allogeneic, third-party mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Radiographic Response | 6 months | Evaluate the incidence of, and time to, radiographic response in patients receiving IT-MSC for active, advanced cerebral adrenoleukodystrophy. Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI, or the absence of an increase in Loes score between the enrollment MRI and 6 month MRI. |
Outcome results
None listed