Cerebral Adrenoleukodystrophy
Conditions
Brief summary
This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.
Interventions
BIOLOGICALMesenchymal stem cells (IV-MSC)
Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.
Sponsors
Masonic Cancer Center, University of Minnesota
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* age ≥ 3 years * diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation * evidence of active cerebral disease as determined by the presence of gadolinium enhancement * ALD MRI (Loes) score ≥ 1 * Patients who have not received prior gene therapy or transplant * Life expectancy of \> 6 months as determined by the enrolling researcher * Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment
Exclusion criteria
* Inability to undergo sedation or MRI studies for any reason * Other concurrent life-threatening disease (life expectancy \<6 months) or eligible for hospice care * Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). | 6 months | Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). | 6 months | Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI. |
Countries
United States
Outcome results
None listed