CompletedPhase 1/2

A Treatment Study of Mucopolysaccharidosis Type IIIB

United States, Colombia, Germany23 participantsStarted 2016-04

Plain-language summary

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.

Who can participate

Age range1 Year – 10 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: Individuals eligible to participate in Part 1 of this study must meet all of the following criteria: * Has deficient NAGLU enzyme activity at Screening. Blood for NAGLU enzyme activity will be collected and analyzed centrally. * Is ≥ 1 and \< 11 years of age (at least 1 of the 3 subjects in Part 1 must be ≥ 1 and \< 6 years of age) * Has presented with signs/symptoms consistent with MPS IIIB; for individuals who have not presented with signs/symptoms of disease (eg, siblings of known patients), the determination of eligibility will be at the discretion of the BioMarin medical monitor in conjunction with the site investigator. * Written informed consent from parent or legal guardian and assent from subject, if required * Has the ability to comply with protocol requirements, in the opinion of the investigator Individuals eligible to participate in Part 2 of this study must meet all of the following criteria: * Participated in and met protocol requirements for transitioning from Study 250-901 or participated in Part 1 of Study 250-201 * Written informed consent from parent or legal guardian and assent from subject, if required Exclusion Criteria: Individuals who meet any of the following exclusion criteria are ineligible to participate in Part 1 of the study: * Has received stem cell, gene therapy or ERT for MPS IIIB * Has contraindications for neurosurgery (eg, congenital heart disease, severe respiratory impairment, or clotting abnormalities) * Has c…

What they're measuring

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.

Timeframe: 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.

Timeframe: Assessed at study end, up to 124 weeks. Collected at: Part 1 - Baseline; Part 2 - Weeks 12, 24, 36, & 48

Trial details

NCT IDNCT02754076

SponsorAllievex Corporation

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-06-24

View on ClinicalTrials.gov More MPS III B trials