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Trial record 2 of 5 for:    Allievex | mps iii

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

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: NCT05492799
Recruitment Status : Enrolling by invitation
First Posted : August 9, 2022
Last Update Posted : January 18, 2023
Information provided by (Responsible Party):
Allievex Corporation

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

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Study Type : Interventional  (Clinical Trial)
Estimated 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
Actual Study Start Date : December 2, 2022
Estimated Primary Completion Date : October 2027
Estimated Study Completion Date : December 2027

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

Primary Outcome Measures :
  1. Primary - neurocognition [ Time Frame: baseline to 144 weeks of treatment ]
    the rate of change in score from neurocognitive assessment using validated scale

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Must have completed 240 weeks of Study 250-202 and enter 250-401 within 8 weeks of dosing completion.
  2. Provides written informed consent from parent or legal guardian and assent from subject, if required
  3. Has the ability to comply with protocol requirements in the opinion of the investigator
  4. 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:

  1. 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
  2. Would not benefit from enrolling in the study in the opinion of the investigator
  3. Has received stem cell, gene therapy or enzyme replacement therapy (other than AX 250) for MPS IIIB
  4. Has a history of poorly controlled seizure disorder
  5. Is prone to complications from ICV drug administration including patients with hydrocephalus or ventricular shunts
  6. 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
  7. 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

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): NCT05492799

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United States, California
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
United States, Pennsylvania
UPMC Children's Hospital Pittburgh
Pittsburgh, Pennsylvania, United States, 15224
Fundación Cardio Infantil - Instituto de Cardiología
Bogotá,, Colombia
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
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
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Study Director: Medical Director Allievex Corporation
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Responsible Party: Allievex Corporation
ClinicalTrials.gov Identifier: NCT05492799    
Other Study ID Numbers: 250-401
First Posted: August 9, 2022    Key Record Dates
Last Update Posted: January 18, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases