Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIB

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 33

Inclusion Criteria

Part 1: Dose Escalation Period:

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 the disease (e.g., siblings of known patients), the determination of eligibility will be at the discretion of the Allievex 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.

Part 2: Stable Dose Period:

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

Are the trial subjects under 18? yes
Number of subjects for this age range: 33
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Part 1: Dose Escalation Phase:

Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.

Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities).

Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain).

Has a history of poorly controlled seizure disorder.

Is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts.

Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study.

Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's well-being or safety, or the interpretability of the subject's clinical data.

Is pregnant at any time during the study

Part 2: Stable Dose Period:
Has received stem cell, gene therapy or ERT for MPS IIIB

Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities)

Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain)

Is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts

Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study

Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject’s ability to comply with protocol requirements, the subject’s well-being or safety, or the interpretability of the subject’s clinical data.

Is pregnant at any time during the study

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIB

Recruitment & Eligibility

Study & Design

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