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Clinical trial

A Phase I-II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced Ex Vivo with CD11b Lentiviral Vector Encoding for Human SGSH in Patients with Mucopolysaccharidosis Type IIIA (MPS IIIa, Sanfilippo Syndrome Type A)

NCT04201405 is registered in ClinicalTrials.gov with University of Manchester as lead sponsor.

NCT NCT04201405
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase I-II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced Ex Vivo with CD11b Lentiviral Vector Encoding for Human SGSH in Patients with Mucopolysaccharidosis Type IIIA (MPS IIIa, Sanfilippo Syndrome Type A)
Lead sponsor
University of Manchester
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
January 7, 2020
Completion date
October 30, 2026
Enrollment
5 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

mucopolysaccharidosis type iiia