City

Milano (1)

Condition

Mucopolysaccharidosis IH (1)

Overall Status

Active, not recruiting (1)

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Phase

phase 1/phase 2 (1)

Sponsor

IRCCS San Raffaele (1)

Collaborator

Fondazione Telethon (1)

Intervention

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. (1)

Study Documents

Intervention Type

genetic (1)

Funder Type

Other (1)

Study Type

Interventional (1)

mucopolysaccharidosis ih

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Mucopolysaccharidosis IH Trial in Milano (Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified

Active, not recruiting

Mucopolysaccharidosis IH

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.

Milano, Italy

Ospedale San Raffaele

Aug 6, 2021