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Trial record 3 of 5 for:    Allievex | mps iii
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A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03784287
Recruitment Status : Active, not recruiting
First Posted : December 21, 2018
Last Update Posted : January 18, 2023
Sponsor:
Information provided by (Responsible Party):
Allievex Corporation

Study Description
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Brief Summary:
The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis Type IIIB MPS III B Drug: AX 250 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational, Extension Study to Evaluate the Long Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Actual Study Start Date : February 19, 2018
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : February 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: AX 250
All subjects will receive AX 250 at the MTTD established in 250-201, 300mg administered weekly by ICV infusion that will continue for up to 240 weeks.
 Drug: AX 250
Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)


Outcome Measures
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Primary Outcome Measures :
  1. Evaluate the Long-term safety and tolerability of AX 250 administered in up to 33 subjects with MPS IIIB by assessing the number of participants with abnormal clinical laboratory values and/or Adverse Events related to the treatment [ Time Frame: Entire study period, up to 240 weeks ]
  2. Evaluating the impact of long-term AX 250 treatment on cognitive function in up to 33 patients with MPS IIIB as assessed by developmental quotient (DQ) [ Time Frame: Entire study period, up to 240 weeks ]

Secondary Outcome Measures :
  1. Evaluate the impact of long-term AX 250 treatment on cognitive function in up to 33 patients with MPS IIIB as assessed by age equivalent score (AEq) [ Time Frame: Entire study period, up to 240 weeks ]
    Age equivalent score is derived from neurocognitive tests (Bayley Scales of Infant Development, 3rd edition OR Kaufman Assessment battery for Children, 2nd edition)

  2. Characterize immunogenicity as measured by IgE levels, formation of anti-AX 250, IGF1 and IGF2 antibodies in Cerebrospinal fluid (CSF) and serum. [ Time Frame: Entire study period, up to 240 weeks ]
  3. Evaluate the impact of long-term AX 250 treatment on Heparan sulfate (HS) levels in cerebrospinal fluid (CSF), serum and urine. [ Time Frame: Entire study period, up to 240 weeks ]
  4. Evaluate the impact of long-term AX 250 treatment on brain structure assessed by magnetic resonance imaging (MRI) [ Time Frame: Entire study period, up to 240 weeks ]
  5. Evaluate the impact of long-term AX 250 treatment on adaptive function derived from the Vineland Adaptive Behavior Scales, 2nd edition (VABS-II) [ Time Frame: Entire study period, up to 240 weeks ]
    VABS-II is a measure of adaptive behavior and can be used to assess the level of subjects' functioning at various ages. Scale ranges from 0-4 (0 meaning never performs the behavior, and 4 meaning almost always performs the behavior independently)


Eligibility Criteria
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Ages Eligible for Study:   0 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must have completed 48 weeks in Part 2 of Study 250-201 and enter 250-202 within 8 weeks of study completion
  • 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
  • Males and females who are of reproductive age should practice true abstinence, defined as no sexual activity, during the study and for 6 months after the study has been completed (or withdrawal from the study). If sexually active and not practicing true abstinence, males and females of reproductive age must use a highly effective method of contraception while participating in the study.
  • 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 both (1) a cognitive AEq score ≤ 18 months, and (2) a DQ score ≤ 20
  • Would not benefit from enrolling in the study in the opinion of the investigator
  • Has received stem cell, gene therapy or ERT (other than AX 250) for MPS IIIB
  • Has contraindications for neurosurgery (eg, congenital heart disease, severe respiratory impairment, or clotting abnormalities)
  • Has contraindications for MRI scans (eg, cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain)
  • Has a history of poorly controlled seizure disorder
  • Is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts
  • Has received any investigational medication other than AX 250 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.
  • Is pregnant at any time during the study
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03784287


Locations
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United States, California
Children's Hospital and Research Center Oakland
Oakland, California, United States, 94609
United States, Pennsylvania
UPMC Children's Hospital Pittburgh
Pittsburgh, Pennsylvania, United States, 15224
Colombia
Fundación Cardio Infantil - Instituto de Cardiología
Bogotá,, Colombia
Germany
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
Spain
Complexo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario
Santiago de Compostela, Spain
Taiwan
MacKay Memorial Children's Hospital
Taipei, Taiwan
Turkey
Gazi University Faculty of Medicine
Ankara, Turkey
United Kingdom
Great Ormond Street Hospital For Children, NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Allievex Corporation
Investigators
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Study Director: Medical Monitor, MD Allievex Corporation
More Information
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Responsible Party: Allievex Corporation
ClinicalTrials.gov Identifier: NCT03784287    
Other Study ID Numbers: AX 250-202
First Posted: December 21, 2018    Key Record Dates
Last Update Posted: January 18, 2023
Last Verified: October 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Allievex Corporation:
Sanfilippo Syndrome Type B
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
 Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases