AX 250 for Sanfilippo Syndrome

Recruiting at 7 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Allievex Corporation

Must not be taking: Stem cell, Gene therapy, ERT

Disqualifiers: Cognitive impairment, Neurosurgery contraindications, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 240 weeks

20 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have received any investigational medication other than AX 250 within 30 days before starting the study.

How is the drug AX 250 unique for treating Sanfilippo Syndrome?

AX 250 is unique because there are currently no FDA-approved treatments for Sanfilippo Syndrome, making it a novel option for addressing this rare genetic disorder. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial tests the safety and effectiveness of AX 250, a drug delivered directly to the brain through an implanted device, in improving cognitive function in patients with MPS IIIB.

Research Team

Medical Monitor, MD

Principal Investigator

Allievex Corporation

Eligibility Criteria

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.

Inclusion Criteria

Has the ability to comply with protocol requirements, in the opinion of the investigator

Written informed consent from parent or legal guardian and assent from subject, if required

I will not have sex or will use effective birth control during and for 6 months after the study.

Exclusion Criteria

Is pregnant at any time during the study

You have a condition that may cause problems if the drug is given directly into the ventricles of the brain, such as 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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AX 250 at the MTTD established in 250-201, 300mg administered weekly by ICV infusion

240 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Treatment Details

Interventions

• AX 250

Trial Overview 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.

Participant Groups

Group I: AX 250 Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Allievex Corporation

Lead Sponsor

Trials

5

Recruited

120+

References

1. United States pubmed.ncbi.nlm.nih.gov

Economic Burden of Sanfilippo Syndrome in the United States. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Characterization of a Case of Pigmentary Retinopathy in Sanfilippo Syndrome Type IIIA Associated with Compound Heterozygous Mutations in the SGSH Gene. [2017]

3. France pubmed.ncbi.nlm.nih.gov

[The course of a case of Maffucci's syndrome]. [2016]

4. England pubmed.ncbi.nlm.nih.gov

Expanding the phenotype of Filippi syndrome: a report of three cases. [2004]

5. England pubmed.ncbi.nlm.nih.gov

Filippi syndrome: a new case with skeletal abnormalities. [2019]