AX 250 for Sanfilippo Syndrome Type B

Enrolling by invitation at 7 trial locations

Overseen By Study Director

Age: Any Age

Sex: Any

Trial Phase: Phase 4

Sponsor: Allievex Corporation

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

Disqualifiers: Seizure disorder, Hydrocephalus, 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: 144 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the potential benefits of AX 250 for individuals with Sanfilippo Syndrome Type B, a rare genetic condition affecting brain and body function. Researchers aim to assess the treatment's impact on mental abilities, communication, daily life skills, and overall quality of life. The trial administers an infusion of AX 250, a new potential drug, directly into the brain every other week. Ideal participants are those who have completed 240 weeks in a related study and can adhere to the study guidelines. As a Phase 4 trial, AX 250 has already received FDA approval and proven effective, and this research seeks to understand its benefits for more patients.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, you cannot have received any investigational medication within 30 days before the study starts or during the study.

What is the safety track record for AX 250?

Research has shown that AX 250 may help treat Sanfilippo Syndrome Type B. Studies have found that patients with this condition tolerate the treatment well. Participants who took AX 250 experienced manageable side effects, indicating the treatment is generally safe. Researchers closely monitored any negative effects, and earlier research found no serious problems. This information provides some confidence in the safety of AX 250, especially since no FDA-approved treatments exist for this rare syndrome. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Unlike the standard of care for Sanfilippo Syndrome Type B, which primarily focuses on managing symptoms, AX 250 acts directly on the disease by delivering a potentially corrective enzyme. Researchers are excited about AX 250 because it offers a new mechanism of action that targets the root cause of the condition rather than just alleviating symptoms. This treatment is administered at a high dose of 300 mg in an open-label format, providing a more direct and potentially effective approach to addressing the enzyme deficiency characteristic of the syndrome.

What is the effectiveness track record for AX 250 in treating Sanfilippo Syndrome Type B?

Research shows that AX 250, also known as tralesinidase alfa, holds promise for treating Sanfilippo Syndrome Type B (MPS IIIB). Earlier studies found tralesinidase alfa effective and well-tolerated, suggesting it could be a helpful treatment for MPS IIIB. Long-term data indicate that this enzyme replacement therapy can offer significant and lasting benefits. Although no treatments for MPS III have been approved, AX 250 stands out as a strong candidate due to these positive results. Overall, the evidence supports its potential to improve symptoms related to this condition. ^{1 3 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Allievex Corporation

Are You a Good Fit for This Trial?

This trial is for individuals with Mucopolysaccharidosis (MPS) IIIB, also known as Sanfilippo Syndrome, who have completed the Study 250-202. Participants must be able to follow the study plan and females of childbearing age should test negative for pregnancy. Those with very low cognitive scores or no improvement in previous studies, recent other treatments or investigational drugs, severe medical conditions, or risk from brain infusions cannot join.

Inclusion Criteria

My parent or guardian and I have both agreed to participate.

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

I am not pregnant and agree to regular pregnancy tests during the study.

Exclusion Criteria

You have a condition that may cause problems with receiving the drug through a particular method of administration.

Would not benefit from enrolling in the study in the opinion of the investigator

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AX 250 treatment via intracerebroventricular infusion every other week

144 weeks

Bi-weekly visits for infusion

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Continuation of AX 250 treatment to further evaluate safety, tolerability, and efficacy

Up to 3 years

What Are the Treatments Tested in This Trial?

Interventions

• AX 250

Trial Overview The trial tests AX 250 delivered directly into the brain fluid every two weeks for up to three years. It aims to see how well it works on neurocognitive function and quality of life in MPS IIIB patients while monitoring safety through adverse events and regular health checks.

How Is the Trial Designed?

Group I: single arm Experimental Treatment 1 Intervention

AX 250 300 mg - open label

Who Is Running the Clinical Trial?

Allievex Corporation

Lead Sponsor

Trials

5

Recruited

120+

Published Research Related to This Trial

Mavoglurant, a drug targeting metabotropic glutamate receptor subtype-5, was found to be well tolerated in a long-term study with 267 participants (119 adolescents and 148 adults) over up to 34 months, showing no new safety concerns.

While the drug did not show significant behavioral benefits in initial studies, gradual improvements were noted in the open-label extensions, suggesting that further research is needed to explore its efficacy in treating Fragile X syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavoglurant in Fragile X Syndrome: Results of two open-label, extension trials in adults and adolescents. Hagerman, R., Jacquemont, S., Berry-Kravis, E., et al.[2019]

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 potential as a therapeutic option for this condition.

At the higher dose of 70 mg/kg, trofinetide showed promising efficacy compared to placebo, suggesting it may lead to clinically meaningful improvements in key symptoms of Rett syndrome, despite the study's short duration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome. Glaze, DG., Neul, JL., Percy, A., et al.[2018]

NNZ-2566, a synthetic analog of IGF-1, showed promise in correcting various symptoms of Fragile X syndrome in fmr1 knockout mice, including learning and memory deficits, hyperactivity, and social interaction issues.

The study suggests that targeting the IGF-1 molecular pathway may be a novel and effective approach for treating Fragile X syndrome, with a clinical trial currently underway to evaluate its effects in human patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

NNZ-2566, a novel analog of (1-3) IGF-1, as a potential therapeutic agent for fragile X syndrome. Deacon, RM., Glass, L., Snape, M., et al.[2018]

Citations

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

NCT05492799 | Safety, Tolerability and Efficacy of ICV AX ... Has received stem cell, gene therapy or enzyme replacement therapy (other than AX 250) for MPS IIIB; Has a history of poorly controlled seizure disorder; Is ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9426762/

Tralesinidase Alfa Enzyme Replacement Therapy Prevents ... This work illustrates the efficacy and tolerability of tralesinidase alfa as a potential therapeutic for patients with mucopolysaccharidosis type IIIB (MPS IIIB) ...

3. 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 ...

4. 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 ...

5. 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 ...

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

AX 250 for Sanfilippo Syndrome Type B · Info for Participants AX 250 is unique because there are currently no FDA-approved treatments for Sanfilippo Syndrome Type B, making it a novel option for addressing this rare ...

7. orpha.net orpha.net/en/research-trials/clinical-trial-network/630165?orphaCode=630165

Knowledge on rare diseases and orphan drugs Open-Label Multicenter Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in ...

8. clinicaltrialsregister.eu clinicaltrialsregister.eu/ctr-search/trial/2022-002762-33/DE

Clinical Trials Register - To evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted intracerebroventricular (ICV) ...