Back to resultsSafety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
Primary Purpose
MPS III B
Status
Enrolling by invitation
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
AX 250
Sponsored by
About this trial
This is an interventional treatment trial for MPS III B
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
Sites / Locations
- UCSF Benioff Children's Hospital Oakland
- UPMC Children's Hospital Pittburgh
- Fundación Cardio Infantil - Instituto de Cardiología
- University Medical Center Hamburg-Eppendorf
- Gazi University Faculty of Medicine
- Great Ormond Street Hospital For Children, NHS Foundation Trust
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
single arm
Arm Description
AX 250 300 mg - open label
Outcomes
Primary Outcome Measures
Primary - neurocognition
the rate of change in score from neurocognitive assessment using validated scale
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05492799
Brief Title
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
December 2, 2022 (Actual)
Primary Completion Date
October 2027 (Anticipated)
Study Completion Date
December 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Allievex Corporation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
MPS III B
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
single arm
Arm Type
Experimental
Arm Description
AX 250 300 mg - open label
Intervention Type
Drug
Intervention Name(s)
AX 250
Intervention Description
biweekly infusion by intracerebroventricular (ICV) infusion
Primary Outcome Measure Information:
Title
Primary - neurocognition
Description
the rate of change in score from neurocognitive assessment using validated scale
Time Frame
baseline to 144 weeks of treatment
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Allievex Corporation
Official's Role
Study Director
Facility Information:
Facility Name
UCSF Benioff Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
UPMC Children's Hospital Pittburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Fundación Cardio Infantil - Instituto de Cardiología
City
Bogotá,
Country
Colombia
Facility Name
University Medical Center Hamburg-Eppendorf
City
Hamburg
Country
Germany
Facility Name
Gazi University Faculty of Medicine
City
Ankara
Country
Turkey
Facility Name
Great Ormond Street Hospital For Children, NHS Foundation Trust
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
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