UnknownPhase 2

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

United States, Colombia, Germany20 participantsStarted 2018-02-19

Plain-language summary

The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).

Who can participate

Age range

0 Years – 18 Years

Sex

ALL

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: * Must have completed 48 weeks in Part 2 of Study 250-201 and enter 250-202 within 8 weeks of study completion * 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 * Males and females who are of reproductive age should practice true abstinence, defined as no sexual activity, during the study and for 6 months after the study has been completed (or withdrawal from the study). If sexually active and not practicing true abstinence, males and females of reproductive age must use a highly effective method of contraception while participating in the study. * If female with childbearing potential, must have a negative pregnancy test at the Screening visit and be willing to have additional pregnancy tests during the study. Exclusion Criteria: * Has both (1) a cognitive AEq score ≤ 18 months, and (2) a DQ score ≤ 20 * Would not benefit from enrolling in the study in the opinion of the investigator * Has received stem cell, gene therapy or ERT (other than AX 250) for MPS IIIB * Has contraindications for neurosurgery (eg, congenital heart disease, severe respiratory impairment, or clotting abnormalities) * Has contraindications for MRI scans (eg, 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 dru…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Evaluate the Long-term safety and tolerability of AX 250 administered in up to 33 subjects with MPS IIIB by assessing the number of participants with abnormal clinical laboratory values and/or Adverse Events related to the treatment

Timeframe: Entire study period, up to 240 weeks

Evaluating the impact of long-term AX 250 treatment on cognitive function in up to 33 patients with MPS IIIB as assessed by developmental quotient (DQ)

Timeframe: Entire study period, up to 240 weeks

Trial details

NCT IDNCT03784287

SponsorAllievex Corporation

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-01

View on ClinicalTrials.gov More Mucopolysaccharidosis Type IIIB trials

Plain-language summary

Who can participate

Age range

0 Years – 18 Years

Sex

ALL

See this in plain English?

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

What they're measuring

Timeframe: Entire study period, up to 240 weeks

Evaluating the impact of long-term AX 250 treatment on cognitive function in up to 33 patients with MPS IIIB as assessed by developmental quotient (DQ)

Timeframe: Entire study period, up to 240 weeks