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New treatment option for MPS IIIA
Official title Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
ClinicalTrials.gov ID: NCT02716246
What this study is testing
What is UX111?
UX111 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for mps iiia.
Also referred to as scAAV9.U1a.hSGSH, ABO-102.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join if
- 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 (based upon review of documented results from a...
- Age:
- For Cohort 1-3: From birth (participating sites in USA and Australia) OR 6 months (participating sites in Spain) to 2 years of age with no BSITD-III...
You likely can't join if
- Inability to participate in the clinical evaluation as determined by PI
- Cohorts 1 to 3 only: Identification of two nonsense or null variants on genetic testing of the SGSH gene (based upon review of documented results...
- At least one S298P mutation in the SGSH gene (based upon review of documented results from a qualified laboratory, and with confirmation with Medical...
- Has evidence of an attenuated phenotype of MPS IIIA, in the judgement of the PI
- Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
- Active viral infection based on clinical observations
See the full eligibility criteria
- 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 (based upon review of documented results from a qualified laboratory, and with confirmation with Medical Monitor)
- Age:
- For Cohort 1-3: From birth (participating sites in USA and Australia) OR 6 months (participating sites in Spain) to 2 years of age with no BSITD-III Cognitive Development Quotient (DQ) requirement, or older than 2 years...
- For Cohort 4 (participating sites in Spain): 3 months to ≤ 2 years of age with no BSITD-III Cognitive DQ requirement or \> 2 years of age with a BSITD-III Cognitive DQ ≥ 60 (n = up to 6). Up to 2 additional people \> 2...
- Cohort 4 only: Vaccination status based on age according to country-specific guidelines that is up to date 30 days prior to Enrollment as verified by documentation from the subject's primary care physician, and willing...
- Inability to participate in the clinical evaluation as determined by PI
- Cohorts 1 to 3 only: Identification of two nonsense or null variants on genetic testing of the SGSH gene (based upon review of documented results from a qualified laboratory, and with confirmation with Medical Monitor)
- At least one S298P mutation in the SGSH gene (based upon review of documented results from a qualified laboratory, and with confirmation with Medical Monitor)
- Has evidence of an attenuated phenotype of MPS IIIA, in the judgement of the PI
- 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
- Cohorts 1 to 3 only: people with total anti-AAV9 antibody titers ≥ 1:100 equivalent to a positive screen as determined by ELISA binding assay in serum
- Cohorts 1-3 only: people with a positive response for the enzyme-linked immunosorbent spot assay (ELISpot) for T-cell responses to AAV9
- Cohorts 1-3 only: Serology consistent with exposure to human immunodeficiency virus (HIV), or serology consistent with active hepatitis B or C infection, Cohort 4: Current clinically significant infections (including...
- 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, hearing, or other impairment sufficient to preclude cooperation with neurodevelopmental testing
- Uncontrolled seizure disorder
- Any item (braces, etc.) or circumstance that 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
- people 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
- Cohorts 1-3: Abnormal laboratory values Grade 2 or higher as defined in common terminology criteria for adverse events (CTCAE) v4.03 for gamma-glutamyl transferase (GGT), total bilirubin, creatinine, hemoglobin, white...
- Aspartate aminotransferase (AST), alanine aminotransferase (ALT), GGT, and/or alkaline phosphatase ≥ 2 × upper limit of normal (ULN) and/or total bilirubin \> 1.5 × ULN
- Anemia (hemoglobin \< 10 g/dL)
- Leukopenia or leukocytosis (total WBC count \ 15,000/mm3 respectively)
- Abnormal absolute neutrophil count (ANC) of \< 1000/mm3
- Platelet count \< 100,000/mm3
- Coagulopathy (international normalized ratio [INR] \> 1.5) or aPTT \> 40 seconds
- Renal impairment, defined as estimated glomerular filtration rate (eGFR) below the lower limit of normal (age and sex appropriate) based on Bedside Schwartz equation
- Female of childbearing potential who is pregnant or demonstrates a positive urine or bhCG result at screening assessment (if applicable)
- Cohorts 1-3: Any vaccination with viral attenuated vaccines less than 30 days prior to the scheduled date of treatment (and use of prednisolone)
- Previous treatment by hematopoietic stem cell transplantation
- Previous participation in a gene/cell therapy or enzyme replacement therapy (ERT) clinical trial Cohort 4 only:
- Known hypersensitivity, that in the judgment of the PI, places the subject at increased risk for adverse effects.
- Unwilling to avoid consumption of grapefruit juice and the use of strong inhibitors of CYP3A4 and/or P-gp (eg, ketoconazole, voriconazole, itraconazole, erythromycin, telithromycin, or clarithromycin), strong inducers...
The study team makes the final eligibility decision.
Where it's taking place
- Columbus, Ohio, United States
- Pittsburgh, Pennsylvania, United States
- North Adelaide, South Australia, Australia
- Barcelona, Barcelona, Spain
- Santiago de Compostela, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Columbus, Ohio, United States; Pittsburgh, Pennsylvania, United States; North Adelaide, South Australia, Australia; Barcelona, Barcelona, Spain; Santiago de Compostela, Spain. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.