Status:
TERMINATED
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Collaborating Sponsors:
Abeona Therapeutics, Inc
Conditions:
MPS IIIA
Sanfilippo Syndrome
Eligibility:
All Genders
2-18 years
Phase:
PHASE1
PHASE2
Brief Summary
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
Detailed Description
This is an open-label, single dose clinical trial. All participants will receive 3 X 10\^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The targ...
Eligibility Criteria
Inclusion
- Diagnosis of MPS IIIA confirmed by the following methods:
- No detectable or significantly reduced SGSH enzyme activity by leukocyte assay and
- Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene
- Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)
- Must be ambulatory, though may receive assistance with ambulation
- Age range of 2 years up to 18 years (excluded)
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 SGSH gene
- At least one S298P mutation in the SGSH gene
- Has evidence of an attenuated phenotype of MPS IIIA
- Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
- Active viral infection based on clinical observations
- 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
- Participants with total anti-AAV9 antibody titers greater than or equal to 1:100 as determined by ELISA binding immunoassay
- Participants 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
- Any item (braces, etc.) which would exclude the participant from being able to undergo MRI according to local institutional policy
- Any other situation that precludes the participant from undergoing procedures required in this study
- Participants with cardiomyopathy or significant congenital heart abnormalities
- The presence of significant non-MPS IlIA 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 v4.03 for GGT, total bilirubin (except in subjects diagnosed with Gilbert's syndrome), creatinine, hemoglobin, WBC count, platelet count, PT and aPTT
- Female participant who is pregnant or demonstrates a positive urine or beta-hCG 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
- Participants who are anticipated to undergo a procedure involving anesthesia within 6 months post- drug administration
- Dysphagia present at Grade 3 or higher, as defined in CTCAE v4.03
Key Trial Info
Start Date :
September 18 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 10 2022
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT04088734
Start Date
September 18 2019
End Date
March 10 2022
Last Update
July 25 2023
Active Locations (3)
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1
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
2
Adelaide Women's and Children's Hospital
North Adelaide, South Australia, Australia, 5006
3
Hospital Clínico Universitario de Santiago
Santiago de Compostela, Spain, 15706