Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AX 250.
- Who it may be relevant to
- Registry conditions: MPS III B. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Colombia, Germany, Turkey (Türkiye), United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Open-Label Multicenter Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients
Overview
This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144 weeks) of treatment with AX 250 administered by ICV infusion every other week. Subjects will be evaluated for neurocognitive function, communication, adaptive behavior, quality of life, imaging characteristics and biochemical markers of disease burden. Safety will be assessed by adverse events, clinical labs, and physical exams.
Interventions
- Drug AX 250
biweekly infusion by intracerebroventricular (ICV) infusion
Primary outcome measures
- Primary - neurocognition [Time frame: baseline to 144 weeks of treatment]
Eligibility criteria
Inclusion criteria
- Must have completed 240 weeks of Study 250-202 and enter 250-401 within 8 weeks of dosing completion.
- Provides 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
- 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 (1) a cognitive age equivalent (AEq ) score ≤ 18 months, (2) a development quotient (DQ) score ≤ 20, and (3) no evidence of improvement during the 250-202 study in secondary or exploratory efficacy endpoints
- Would not benefit from enrolling in the study in the opinion of the investigator
- Has received stem cell, gene therapy or enzyme replacement therapy (other than AX 250) for MPS IIIB
- Has a history of poorly controlled seizure disorder
- Is prone to complications from ICV 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- UCSF Benioff Children's Hospital Oakland — Oakland
- UPMC Children's Hospital Pittburgh — Pittsburgh
Colombia · 1 center
- Fundación Cardio Infantil - Instituto de Cardiología — Bogotá
Germany · 1 center
- University Medical Center Hamburg-Eppendorf — Hamburg
Turkey (Türkiye) · 1 center
- Gazi University Faculty of Medicine — Ankara
United Kingdom · 1 center
- Great Ormond Street Hospital For Children, NHS Foundation Trust — London
Identifiers
NCT: NCT05492799 · 250-401