Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT ID: NCT05492799

Last Updated: 2023-08-09

Basic Information

• Recruitment Status: ENROLLING_BY_INVITATION
• Clinical Phase: PHASE4
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-12-02
• Study Completion Date: 2027-12-31

Brief Summary

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.

Conditions

MPS III B

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

single arm

AX 250 300 mg - open label

Group Type: EXPERIMENTAL

AX 250

Intervention Type: DRUG

biweekly infusion by intracerebroventricular (ICV) infusion

Interventions

AX 250

biweekly infusion by intracerebroventricular (ICV) infusion

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Must have completed 240 weeks of Study 250-202 and enter 250-401 within 8 weeks of dosing completion.

2. Provides written informed consent from parent or legal guardian and assent from subject, if required

3. Has the ability to comply with protocol requirements in the opinion of the investigator

4. 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

1. 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

2. Would not benefit from enrolling in the study in the opinion of the investigator

3. Has received stem cell, gene therapy or enzyme replacement therapy (other than AX 250) for MPS IIIB

4. Has a history of poorly controlled seizure disorder

5. Is prone to complications from ICV drug administration including patients with hydrocephalus or ventricular shunts

6. 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

7. 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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Allievex Corporation

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Allievex Corporation

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

UPMC Children's Hospital Pittburgh

Pittsburgh, Pennsylvania, United States

Fundación Cardio Infantil - Instituto de Cardiología

Bogotá, , Colombia

University Medical Center Hamburg-Eppendorf

Hamburg, , Germany

Gazi University Faculty of Medicine

Ankara, , Turkey (Türkiye)

Great Ormond Street Hospital For Children, NHS Foundation Trust

London, , United Kingdom

Countries

United States; Colombia; Germany; Turkey (Türkiye); United Kingdom

Other Identifiers

250-401

Identifier Type: -

Identifier Source: org_study_id