AX 250 for Sanfilippo Syndrome

Not currently recruiting at 9 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests AX 250, a treatment for Sanfilippo Syndrome, specifically the MPS IIIB type, which affects brain function and development. The goal is to determine if AX 250 is safe over a long period and whether it can improve cognitive abilities in those with this condition. Participants who completed a previous related study and can follow the trial rules may be a good fit. This trial may interest those facing the daily challenges of Sanfilippo Syndrome and seeking new treatment options. As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group, offering a chance to contribute to early findings on AX 250's effectiveness.

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.

Is there any evidence suggesting that AX 250 is likely to be safe for humans?

Research has shown that AX 250 is generally well-tolerated by people with Sanfilippo Syndrome. In previous studies, researchers administered AX 250 directly into the brain using a small device, and patients seemed to handle it well. No major safety concerns were reported. However, as this treatment is still in development, new information may emerge. It is crucial to consult a healthcare provider to understand the risks and benefits before deciding to join a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for Sanfilippo Syndrome, which might include supportive therapies to manage symptoms, AX 250 is unique because it is administered through an innovative delivery method: intraventricular (ICV) infusion. This means the treatment is delivered directly into the brain, potentially allowing for more effective targeting of the central nervous system where the disease primarily affects. Researchers are excited about AX 250 because it could offer a more direct and potentially powerful approach to managing the disease, with the hope of enhancing outcomes for patients over a prolonged period of up to 240 weeks.

What evidence suggests that AX 250 might be an effective treatment for Sanfilippo Syndrome?

Research shows that AX 250 is under investigation as a potential treatment for Sanfilippo Syndrome, specifically MPS IIIB. Earlier studies found that administering AX 250 directly into the brain might improve thinking and memory in patients. This is significant because Sanfilippo Syndrome often leads to a decline in these abilities. Although no approved treatment exists for this condition yet, AX 250 has shown promise in early research. The aim is to manage symptoms and enhance the quality of life for patients. ^{2 3 6 7 8}

Who Is on the Research Team?

Medical Monitor, MD

Principal Investigator

Allievex Corporation

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05492799

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in ... A research study involving human volunteers (also called participants) that is intended to add to medical knowledge. There are two types of clinical studies: ...

2. clinicaltrial.be clinicaltrial.be/en/details/6100?per_page=100&recruiting=1&enrolling_by_invitation=1&active_not_recruiting=1&not_yet_recruiting=0&completed=0&only_recruiting=0&only_eligible=0

A Treatment Extension Study of Mucopolysaccharidosis ... The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7391468/

A Cure for Sanfilippo Syndrome? A Summary of Current ... There are currently no approved treatments for MPS III, but a number of therapeutic approaches are under development.

4. sciencedirect.com sciencedirect.com/science/article/pii/S1096719224004190

Community consensus for Heparan sulfate as a biomarker ... There is currently no approved treatment for any of the 4 subtypes of MPS III (Sanfilippo syndrome). Stem cell transplantation has not been as effective in ...

5. clinicaltrialsregister.eu clinicaltrialsregister.eu/ctr-search/search?query=Sanfilippo+Syndrome+(MPS+III)

Clinical trials for Sanfilippo Syndrome (MPS III) ... Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB ... Medical condition: Mucopolysaccharidosis Type IIIB ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05492799

NCT05492799 | Safety, Tolerability and Efficacy of ICV AX ... This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB ...

7. withpower.com withpower.com/trial/phase-4-mucopolysaccharidosis-iii-10-2022-535b3

AX 250 for Sanfilippo Syndrome Type B · Info for Participants Trofinetide was found to be well tolerated and generally safe in a phase 2 study involving 56 adolescent and adult females with Rett syndrome, indicating its ...

8. curesanfilippofoundation.org curesanfilippofoundation.org/2020/03/sanfilippo-type-b-ert-therapy/

TA-ERT Enzyme Replacement | MPS IIIB | Phase I-II Spruce Biosciences announced that integrated long-term clinical data of TA-ERT demonstrated profound and durable efficacy and safety in patients ...