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

NCT05492799 | Enrolling By Invitation Phase 4 FDA Drug

• 1 other identifier: 250-401
• Study Type: interventional
• Target: 15
• Locations: 5 countries
• Sites: 6

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

Outcome Measures

Primary Outcomes (1)

• Primary - neurocognition

  the rate of change in score from neurocognitive assessment using validated scale

  baseline to 144 weeks of treatment

Study Arms (1)

single arm

EXPERIMENTAL

AX 250 300 mg - open label

Drug: AX 250

Interventions

AX 250 DRUG

biweekly infusion by intracerebroventricular (ICV) infusion

single arm

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Allievex Corporation: lead

Study Sites (6)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

UPMC Children's Hospital Pittburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Fundación Cardio Infantil - Instituto de Cardiología

Bogotá, Colombia

Location

University Medical Center Hamburg-Eppendorf

Hamburg, Germany

Location

Gazi University Faculty of Medicine

Ankara, Turkey (Türkiye)

Location

Great Ormond Street Hospital For Children, NHS Foundation Trust

London, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Mucopolysaccharidosis III

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Allievex Corporation

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2022
• First Posted: August 9, 2022
• Study Start: December 2, 2022
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: August 9, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1); GB (1); CO (1); US (2); DE (1)