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)

Trial Information
Who is this study for? Patients with mucopolysaccharidosis type IIIA
What treatments are being studied? Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
Status: Active, not recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2

Patients with MPS IIIA have a clinical disorder marked by severe and progressive brain disease and neurological symptoms due to the accumulation of undigested glycosaminoglycans in all cells of the body. This study will be the first in human clinical trial to explore the safety, tolerability and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients. Following treatment with the gene therapy patients will be followed up for a minimum of 3 years.

Am I eligible for this trial?
Participation Requirements
Minimum Age:
3 months
Maximum Age:
Healthy Volunteers:

• Written informed consent of a legally authorized guardian(s)

• Age at baseline ≥3 months and ≤24 months

• Normal cognitive function or mild cognitive deterioration (subject has a Development Quotient (DQ) score ≥80) at baseline as determined by the Bayley Scale of Infant Development-third edition (BSID-III), cognitive domain)

• Sibling or relative of known MPS IIIA patients with rapidly progressing phenotype, or genotype associated with rapidly progressing phenotype, or presence of somatic features predictive of rapid progression

• SGSH activity ≤10% of the Lower Limit of Normal as measured in leukocytes, plus either (1) a normal enzyme activity level of at least one other sulfatase (to rule out multiple sulfatase deficiency) as measured in leukocytes or (2) two documented mutations in the SGSH gene.

• Medically stable and able to accommodate the protocol requirements, including travel without placing an undue burden on the patient/patient's family, as determined by the CI.

Where is this trial taking place?
Other Locations
United Kingdom
Manchester University NHS Foundation Trust
When is this trial taking place?
Start Date: January 7, 2020
Estimated Completion Date: October 30, 2024
How many participants will be in this trial?
Target number of participants: 5
What treatment is being studied in this trial?
Experimental: Haematopoietic stem cell gene therapy for MPS IIIA
Open label
Who are the authors of this trial?

This content was sourced from clinicaltrials.gov

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