Mucopolysaccharidosis Type IH Clinical Trial
Official title:
Sleeping Beauty Transposon-Engineered Plasmablasts for Expression and Delivery of Alpha-L-iduronidase in Patients With Hurler Syndrome That Have Previously Undergone Allogeneic Transplantation
| Verified date | October 2022 |
| Source | Masonic Cancer Center, University of Minnesota |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Withdrawn |
| Enrollment | 0 |
| Est. completion date | June 2023 |
| Est. primary completion date | June 2023 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 3 Years to 8 Years |
| Eligibility | Inclusion Criteria: - Diagnosis of Mucopolysaccharidosis type IH (MPS IH, Hurler syndrome) - Underwent a previous hematopoietic stem cell transplant >1 year prior to study enrollment - Age =3 years and =8 years at time of study registration - = 10 kilograms body weight - Creatinine <1.5 normal for gender and age. - Ejection fraction = 40% by echocardiogram - Must commit to traveling to the University of Minnesota for the necessary followup evaluations - Must agree to stay in the Twin Cities area (<45-minute drive from the Masonic Children's Hospital) for a minimum of 5 days after each cell infusion - Voluntary written parental consent prior to the performance of any study related procedures Exclusion Criteria: - Prior enzyme replacement therapy within 4 months prior to enrolling on study - History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders - Evidence of active graft vs. host disease - Requirement for systemic immune suppression - Requirement for continuous supplemental oxygen - Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment. - In the investigator's judgement, the subject is unlikely to complete all protocol required study visits or procedures, including follow up visits, or comply with the study requirements for participation. |
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| Masonic Cancer Center, University of Minnesota |
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Maximum Tolerated Dose (MTD) | Maximum tolerated dose (MTD) of autologous plasmablasts engineered to express large amounts of a-L-iduronidase (IDUA) using a Sleeping Beauty transposon approach | 1 Year | |
| Primary | 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 | |
| Primary | Safety and Tolerability after Infusion: Incidence of Adverse Events | Incidence of Adverse Events | 1 Year | |
| Secondary | Z-score Growth Rate | Estimate the 1-year Z-score growth rate standardized for age and gender | 1 Year | |
| Secondary | Donor Engraftment | Estimate percent myeloid donor chimerism (CD33/66b) at baseline and at 6 and 12 months. | Baseline, 6 months and 1 Year | |
| Secondary | 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 |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT01572636 -
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
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