Status:
TERMINATED
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
Lead Sponsor:
Abeona Therapeutics, Inc
Conditions:
Mucopolysaccharidosis Type 3 B
Eligibility:
All Genders
Phase:
PHASE1
PHASE2
Brief 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 per...
Eligibility Criteria
Inclusion
- 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
- 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
- Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing
- Uncontrolled seizure disorder
- Any item (braces, etc.) which would exclude the subject from being able to undergo MRI according to local institutional policy
- Any other situation that precludes the subject from undergoing procedures required in this study
- Subjects with cardiomyopathy or significant congenital heart abnormalities
- The presence of significant non-MPS IlIB related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
- Abnormal laboratory values Grade 2 or higher as defined in CTCAE v4.0 for GGT, total bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT and aPTT
- Female participant who is pregnant or demonstrates a positive urine or serum result at screening assessment (if applicable).
- Any vaccination with viral attenuated vaccines less than 30 days prior to the scheduled date of treatment (and use of prednisolone)
- Previous treatment by Haematopoietic Stem Cell transplantation
- Previous participation in a gene/cell therapy or ERT clinical trial
Key Trial Info
Start Date :
October 16 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 7 2022
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT03315182
Start Date
October 16 2017
End Date
April 7 2022
Last Update
May 5 2022
Active Locations (4)
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1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
2
Armand-Trousseau Hospital
Paris, France
3
University Hospital Hamburg-Eppendorf
Hamburg, Germany
4
Hospital Clinico Universitario de Santiago
Santiago de Compostela, Spain, 15706