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Enzyme Replacement Therapy

AX 250 for Sanfilippo Syndrome

Phase 2
Waitlist Available
Research Sponsored by Allievex Corporation
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up entire study period, up to 240 weeks
Awards & highlights

Study Summary

This trial is testing a new treatment for MPS IIIB, which is a disease that affects cognitive function. The goal is to see if the treatment is safe and tolerable long-term, and if it has an impact on cognitive function.

Who is the study for?
This trial is for patients with Mucopolysaccharidosis Type IIIB who completed a previous study and can follow the new study's rules. They must not be pregnant, agree to use contraception or practice abstinence, and cannot have severe seizures, prior gene therapy/stem cell treatments (other than AX 250), or conditions making intraventricular drug delivery risky.Check my eligibility
What is being tested?
The trial tests the long-term safety of AX 250 delivered through an implanted device in the brain and its effects on cognitive function over time in individuals with MPS IIIB. Participants previously treated with AX 250 continue treatment to assess ongoing impacts.See study design
What are the potential side effects?
While specific side effects are not listed here, potential risks may include complications from drug delivery via an implanted brain device, reactions to AX 250 itself, or issues related to long-term treatment affecting cognitive functions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I will not have sex or will use effective birth control during and for 6 months after the study.
Select...
I am not pregnant and agree to regular pregnancy tests during the study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~entire study period, up to 240 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and entire study period, up to 240 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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
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)
Secondary outcome measures
Characterize immunogenicity as measured by IgE levels, formation of anti-AX 250, IGF1 and IGF2 antibodies in Cerebrospinal fluid (CSF) and serum.
Evaluate the impact of long-term AX 250 treatment on Heparan sulfate (HS) levels in cerebrospinal fluid (CSF), serum and urine.
Evaluate the impact of long-term AX 250 treatment on adaptive function derived from the Vineland Adaptive Behavior Scales, 2nd edition (VABS-II)
+2 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: AX 250Experimental Treatment1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AX 250
2016
Completed Phase 2
~30

Find a Location

Who is running the clinical trial?

Allievex CorporationLead Sponsor
4 Previous Clinical Trials
104 Total Patients Enrolled
Medical Monitor, MDStudy DirectorAllievex Corporation
72 Previous Clinical Trials
18,056 Total Patients Enrolled

Media Library

AX 250 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03784287 — Phase 2
Sanfilippo Syndrome Research Study Groups: AX 250
Sanfilippo Syndrome Clinical Trial 2023: AX 250 Highlights & Side Effects. Trial Name: NCT03784287 — Phase 2
AX 250 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03784287 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there an age limitation for participants in this clinical experiment?

"Qualified candidates for this trial must be between 0 and 18 years old. The study encompasses 27 studies geared towards children, as well as 16 trials specifically targeting individuals older than 65."

Answered by AI

Is the enrollment process for this trial open at present?

"As observed on clinicaltrials.gov, this trial is no longer recruiting participants. Despite being initially posted in February 2018 and updated last October 2022, the study has stopped looking for applicants; however, 28 other research projects are actively seeking patients right now."

Answered by AI

May I be eligible to partake in the research?

"Currently, 20 children between 0 and 18 years old with mucopolysaccharidoses are being recruited for this research. The key criteria they must meet include having completed 48 weeks of Study 250-201 within the past 8 weeks, obtaining informed consent from their legal guardians or parents (with assent from the subject), compliance with protocol requirements according to the investigator's judgement, contraceptive precautions if sexually active and not practicing true abstinence during & after study duration, as well as a negative pregnancy test prior to screening."

Answered by AI

What potential risks should be taken into account when administering AX 250 to individuals?

"The safety of AX 250 has been evaluated by the Power team and given a rating of 2, as this is an experimental Phase 2 trial with evidence to support its security but no data proving efficacy."

Answered by AI
~2 spots leftby Jan 2025