MPS III BPhase 4NCT05492799
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
A Phase 4 study testing AX 250 in people with MPS III B, run by Allievex Corporation at 6 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MPS III B
- What is being tested?
- AX 250
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 2 Dec 2022to Dec 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must have completed 240 weeks of Study 250-202 and enter 250-401 within 8 weeks of dosing completion.
- Provides written informed consent from parent or legal guardian and assent from subject, if required
- Has the ability to comply with protocol requirements in the opinion of the investigator
- 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
Who cannot join
- 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
- Would not benefit from enrolling in the study in the opinion of the investigator
- 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 prone to complications from ICV drug administration including patients with hydrocephalus or ventricular shunts
- 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
- 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
Trial locations and contacts
6 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 5 countries
United States2 sites
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
UPMC Children's Hospital Pittburgh
Pittsburgh, Pennsylvania, 15224
United Kingdom1 site
Great Ormond Street Hospital For Children, NHS Foundation Trust
London, WC1N 3JH
About this study
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
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.
- Conditions
- MPS III B
- Being tested
- AX 250
- Medical subject headings
- Mucopolysaccharidosis III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AX 250Drug
biweekly infusion by intracerebroventricular (ICV) infusion
| Study group | Role | What they receive |
|---|---|---|
| single arm | Experimental | AX 250 300 mg - open label Receives: Drug: AX 250 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary - neurocognition
the rate of change in score from neurocognitive assessment using validated scale
Measured: baseline to 144 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2022
- Study start2 Dec 2022
- Primary completionOct 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS III BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Allievex Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Allievex Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05492799
- Sponsor's protocol ID
- 250-401
- Status last verified
- Aug 2023
- First submitted
- 5 Aug 2022
- Last updated
- 9 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05492799
- Acronym / sponsor ID
- 250-401
- Conditions
- MPS III B
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Allievex CorporationIndustry (pharma, biotech, devices)
- Study start
- 2 Dec 2022 (actual)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 9 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm | Experimental | Drug: AX 250AX 250 300 mg - open label |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Must have completed 240 weeks of Study 250-202 and enter 250-401 within 8 weeks of dosing completion.
- Provides written informed consent from parent or legal guardian and assent from subject, if required
- Has the ability to comply with protocol requirements in the opinion of the investigator
- 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
Key exclusion criteria (7 listed)
- 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
- Would not benefit from enrolling in the study in the opinion of the investigator
- 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 prone to complications from ICV drug administration including patients with hydrocephalus or ventricular shunts
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AX 250biweekly infusion by intracerebroventricular (ICV) infusion | Drug | single arm |
Primary outcome
Primary - neurocognition
Time frame: baseline to 144 weeks of treatment
the rate of change in score from neurocognitive assessment using validated scale
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2023.
- Individual participant dataSharing planned: No
Related trials for MPS III B
Active, not recruitingPhase 1/2
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Mucopolysaccharidosis IIIB
Not yet recruitingPhase 3
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
MPS IIIB
Not yet recruitingPhase 1/2
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
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Enrolling by invitationObservational study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Allievex Corporation
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing AX 250 in people with MPS III B, run by Allievex Corporation at 6 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 9 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Colombia, Germany, Turkey (Türkiye), United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary - neurocognition (time frame: baseline to 144 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2023.