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

Sponsor

Allievex Corporation (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05492799

Collaborator

(none)

Enrollment

Locations

Arm

Anticipated Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
MPS III B	Drug: AX 250	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

15 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Nov 1, 2022

Anticipated Primary Completion Date :

Oct 1, 2027

Anticipated Study Completion Date :

Dec 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: single arm AX 250 300 mg - open label	Drug: AX 250 biweekly infusion by intracerebroventricular (ICV) infusion

Arm

Intervention/Treatment

Experimental: single arm

AX 250 300 mg - open label

Drug: AX 250

biweekly infusion by intracerebroventricular (ICV) infusion

Outcome Measures

Primary Outcome Measures

Primary - neurocognition [baseline to 144 weeks of treatment]
the rate of change in score from neurocognitive assessment using validated scale

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCSF Benioff Children's Hospital Oakland	Oakland	California	United States	94609
2	Fundación Cardio Infantil - Instituto de Cardiología	Bogotá,		Colombia
3	University Medical Center Hamburg-Eppendorf	Hamburg		Germany
4	Gazi University Faculty of Medicine	Ankara		Turkey
5	Great Ormond Street Hospital For Children, NHS Foundation Trust	London		United Kingdom	WC1N 3JH

Sponsors and Collaborators

Allievex Corporation

Investigators

Study Director: Medical Director, Allievex Corporation

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Allievex Corporation

ClinicalTrials.gov Identifier:

NCT05492799

Other Study ID Numbers:

250-401

First Posted:

Aug 9, 2022

Last Update Posted:

Aug 9, 2022

Last Verified:

Aug 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Mucopolysaccharidosis III

Study Results

No Results Posted as of Aug 9, 2022