Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| MT2018-18 | Other Identifier | University of Minnesota Masonic Cancer Center |
Not provided
Not provided
Not provided
Study went on hold because more pre-clinical work needs to be done per FDA feedback. However, the study never went off hold and the study team has decided to close this study without opening to enrollment.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a single center, Phase 1/2 study in which patients with Hurler syndrome who have previously undergone allogeneic hematopoietic stem cell transplantation are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Phase 1: Dose Escalation | Experimental |
| |
| Phase 2 - Expansion at MTD | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous Plasmablasts | Drug | Autologous Plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system. Phase 1:
Phase 2: - Maximum Tolerated Dose (MTD) established in Phase I |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Tolerated Dose (MTD) | Maximum tolerated dose (MTD) of autologous plasmablasts engineered to express large amounts of α-L-iduronidase (IDUA) using a Sleeping Beauty transposon approach | 1 Year |
| Growth Velocity (cm/year) | Growth velocity in centimeters/year over a one-year period through determinations of sitting and standing height at baseline and post infusion | 1 Year |
| Safety and Tolerability after Infusion: Incidence of Adverse Events | Incidence of Adverse Events | 1 Year |
| Measure | Description | Time Frame |
|---|---|---|
| Z-score Growth Rate | Estimate the 1-year Z-score growth rate standardized for age and gender | 1 Year |
| Donor Engraftment | Estimate percent myeloid donor chimerism (CD33/66b) at baseline and at 6 and 12 months. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Paul Orchard, MD | University of Minnesota, Department of Pediatrics | Principal Investigator |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
|
| Baseline, 6 months and 1 Year |
| Levels of circulating antibodies (IgG, IgM, IgA and IgE) | Determine levels of circulating antibodies (IgG, IgM, IgA and IgE) at baseline and at scheduled time points post infusion. | 1 Year |
| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C494471 | sleeping beauty transposase, human |
Not provided
Not provided
Not provided