NCT03488394 - Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

May 11, 2018

Primary Completion Date

March 31, 2028

Study Completion Date

March 31, 2028

Conditions

Mucopolysaccharidosis IH

Interventions

GENETIC

Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.

"The drug product target dose is more or equal to 8x10\^6 CD34+ cells/Kg, with a minimum dose of 4x10\^6 CD34+ cells/Kg and a maximum dose of 35x10\^6 CD34+ cells/Kg.~The product will be injected intravenously."

Trial Locations (1)

20132

Ospedale San Raffaele, Milan