A Phase 1/2 interventional study of AX 250 in MPS III B and Mucopolysaccharidosis Type IIIB, sponsored by Allievex Corporation. Completed at 7 sites in 7 countries. Open to participants aged 1 Year to 10 Years. Per ClinicalTrials.gov, last updated 2020-08-06.
Sponsored by Allievex Corporation · Phase 1/2, Interventional, and Treatment
The study's primary objectives are to evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of AX 250 on cognitive function in patients with MPS IIIB as assessed by the Development Quotient.
145 studies on the registry are indexed under Mucopolysaccharidoses; 11 are open to participants now.
This study's enrollment of 23 is above the median of 15 across 80 interventional studies indexed under Mucopolysaccharidoses.
Browse Mucopolysaccharidoses studies →Allievex Corporation is the lead sponsor of 4 studies on the registry; 1 is open to participants now.
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Individuals eligible to participate in Part 1 of this study must meet all of the following criteria:
Individuals eligible to participate in Part 2 of this study must meet all of the following criteria:
Exclusion Criteria:
Individuals who meet any of the following exclusion criteria are ineligible to participate in Part 1 of the study:
Individuals who meet any of the following exclusion criteria are ineligible to participate in Part 2 of this study:
In Part 1, patients will receive up to 3 escalating doses of AX 250 (30, 100 and 300 mg) via ICV infusion every week until the maximum tolerated tested dose (MTTD) is established. In Part 2, patients will receive weekly doses of AX 250 via ICV infusion that will continue for 48 weeks at the MTTD established in Part 1.
Drug: AX 250
Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)
Safety Evaluation of weekly infusions of AX 250 (Part 1 & Part 2) - Number of participants with abnormal clinical laboratory values and/or Adverse Events that are related to treatment.
Number of participants with abnormal clinical laboratory values and/or Adverse Events that are related to treatment.
Time frame: Entire study period, up to 124 weeks
Development Quotient (DQ) as efficacy variable with analysis of rate of change of DQ score on treatment vs. rate of change of DQ score prior to treatment.
Time frame: Assessed at study end, up to 124 weeks. Collected at: Part 1 - Baseline; Part 2 - Weeks 12, 24, 36, & 48
Characterize maximum concentration (Cmax) of AX 250 in cerebrospinal fluid (CSF) and plasma as relevant through completion of Part 1 and Part 2
Time frame: Study end, up to 124 weeks. Collected at: Pre-dose, 0, 4, 10, 24, 48, 72, 96, 168 hours post-dose for the first dose during each dose escalation in Part 1. Pre-dose, 0, 4, 10, 24, 48, 72, 96, 168 hours post-dose for Baseline, Weeks 5, 12, 36 in Part 2.
Characterize area under concentration curve (AUC) of AX 250 in cerebrospinal fluid (CSF) and plasma as relevant through completion of Part 1 and Part 2
Time frame: Study end, up to 124 weeks. Collected at: Pre-dose, 0, 4, 10, 24, 48, 72, 96, 168 hours post-dose for the first dose during each dose escalation in Part 1. Pre-dose, 0, 4, 10, 24, 48, 72, 96, 168 hours post-dose for Baseline, Weeks 5, 12, 36 in Part 2.
Characterize immunogenicity of AX 250 total anti-drug anti-body (TAb) in cerebrospinal fluid (CSF) and serum as relevant through completion of Part 1 and Part 2
Time frame: Assessed at study end, up to 124 weeks. Collected at: First dose during each dose escalation in Part 1, and Weeks 0, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 in Part 2
Evaluate GAG levels in cerebrospinal fluid (CSF)
Time frame: Assessed at study end, up to 124 weeks. Collected at: Each weekly visit as relevant through completion of Part 1 and Part 2
Evaluate GAG levels in plasma
Time frame: Assessed at study end, up to 124 weeks. Collected at: Each weekly visit as relevant through completion of Part 1 and Weeks 0, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, as relevant in Part 2
Evaluate GAG levels in urine
Time frame: Assessed at study end, up to 124 weeks. Collected at: Each weekly visit as relevant through completion of Part 1 and Weeks 0, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, as relevant in Part 2
Evaluate the impact of AX 250 treatment on brain structure assessed by magnetic resonance imaging (MRI)
Time frame: Assessed at study end, up to 124 weeks. Part 1 - Screening and Baseline; Part 2 - Screening, Baseline, Week 24, and Week 48
Plan to share: No
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Allievex Corporation