Safety, Tolerability and Efficacy of ICV AX 250 Treatment... | NCT05492799

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

NCT05492799

Sponsor

Allievex Corporation

Status

Enrolling by invitation

Last Update Posted

Aug 9, 2023Actual

Enrollment

15Estimated

Phase

Phase 4

Conditions

MPS III B

Interventions

AX 250

Countries

United States
Colombia
Germany
Turkey (Türkiye)
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2023

Overall Recruitment Status or Expanded Access Status

Enrolling by invitation

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 2, 2022Actual

Primary Completion Date

Oct 2027Estimated

Completion Date

Dec 2027Estimated

First Submitted Date

Aug 5, 2022

First Submission Date that Met QC Criteria

Aug 8, 2022

First Posted Date

Aug 9, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 7, 2023

Last Update Posted Date

Aug 9, 2023Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 4

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

15Estimated

Label	Type	Description	Intervention Names
single arm	Experimental	AX 250 300 mg - open label	Drug: AX 250

Name	Type	Description	Arm Group Labels	Other Names
AX 250	Drug	biweekly infusion by intracerebroventricular (ICV) infusion	single arm

Measure	Description	Time Frame
Primary - neurocognition	the rate of change in score from neurocognitive assessment using validated scale	baseline to 144 weeks of treatment

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
UCSF Benioff Children's Hospital Oakland		Oakland	California	94609	United States
UPMC Children's Hospital Pittburgh

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn