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Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB | Clinical Trials | Clareo Health

TERMINATEDPHASE1/PHASE2

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB

NCT03315182•Abeona Therapeutics, Inc

View on ClinicalTrials.gov

Summary

Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein

Detailed Description

Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Confirmed diagnosis of MPSIIIB by both of the following two methods:
•* No detectable or significantly reduced NAGLU enzyme activity by plasma.
•* Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the NAGLU gene
•Age: From Birth to 2 years or children older than 2 years with a minimum cognitive Development Quotient (DQ) of 60 or above (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)

Exclusion Criteria

•Inability to participate in the clinical evaluation as determined by Principal Investigator
•Identification of two nonsense or null variants on genetic testing of the NAGLU gene
•Has evidence of an attenuated phenotype of MPS IIIB
•Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
•Active viral infection based on clinical observations as infections by Adenoviruses, Epstein-Barr Virus, Cytomegalovirus, Respiratory Syncytial Virus
•Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer , or precludes the child from participating in the protocol assessments and follow up as autoimmune diseases requiring immunosuppression, such as juvenile rheumatoid arthritis or idiopathic thrombocytopenia purpura
•Subjects with total anti-AAV9 antibody titers ≥ 1:100 as determined by ELISA binding immunoassay
•Subjects with a positive response for the ELISPOT for T-cell responses to AAV9
•Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection
•Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy
+11 more criteria

Locations (4)

Nationwide Children's Hospital

Columbus, Ohio, United States

Armand-Trousseau Hospital

Paris, France

University Hospital Hamburg-Eppendorf

Hamburg, Germany

Hospital Clinico Universitario de Santiago

Santiago de Compostela, Spain

Key Dates

Start Date

October 16, 2017

Primary Completion Date

April 7, 2022

Completion Date

April 7, 2022

Last Updated

May 5, 2022

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis Type 3 B

Interventions

rAAV9.CMV.hNAGLU

BIOLOGICAL