Condition

Mucopolysaccharidosis Type IIIA (3)

Mucopolysaccharidosis Type IIIB (1)

Overall Status

Active, not recruiting (2)

Completed (1)

Phase

phase 1/phase 2 (1)

phase 2/phase 3 (1)

Sponsor

LYSOGENE (1)

Nationwide Children's Hospital (1)

University of Manchester (1)

Collaborator

CTI Clinical Trial and Consulting Services (1)

Great Ormond Street Hospital for Children NHS Foundation Trust (1)

Manchester University NHS Foundation Trust (1)

Orchard Therapeutics (1)

Sanfilippo Children's Research Foundation (1)

The Children's Medical Research Foundation (1)

The Sanfilippo Research Foundation (1)

University College, London (1)

Intervention

autologous cd34+ cells transduced with a lentiviral vector containing the human sgsh gene (1)

lumbar puncture (1)

lys-saf302 (1)

magnetic resonance imaging (mri) of the brain (1)

Study Documents

Intervention Type

drug (2)

device (1)

procedure (1)

Funder Type

Other (2)

Industry (1)

Study Type

Interventional (2)

Observational (1)

mucopolysaccharidosis type iiia

Showing 1 - 3 of 3

Trials per page:

STUDY

CONDITIONS

INTERVENTIONS

LOCATIONS

LAST UPDATED

Mucopolysaccharidosis Type IIIA Trial in Worldwide (LYS-SAF302)

Active, not recruiting

Mucopolysaccharidosis Type IIIA

LYS-SAF302

Orange, California
+7 more

Aug 30, 2021

Mucopolysaccharidosis Type IIIA Trial in Manchester (Autologous CD34+ cells transduced with a lentiviral vector containing the

Active, not recruiting

Mucopolysaccharidosis Type IIIA

Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene

Manchester, United Kingdom

Manchester University NHS Foundation Trust

Jul 2, 2021

Natural History Studies of Mucopolysaccharidosis III

Completed

Mucopolysaccharidosis Type IIIA
Mucopolysaccharidosis Type IIIB

Lumbar puncture
Magnetic Resonance Imaging (MRI) of the brain

Columbus, Ohio

Nationwide Children's Hospital

Nov 12, 2015