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Hope for sanfilippo kids: Brain-Infused drug enters final testing

NCT ID NCT07579910

Disease control Sponsor: Spruce Biosciences Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Sep 21, 2026 · Updated 4 times

Summary

This phase 3 trial tests a drug called tralesinidase alfa in 14 children aged 1-5 with Sanfilippo syndrome type B, a rare genetic disease that causes severe brain damage. The drug is given directly into the brain fluid to replace a missing enzyme. The goal is to see if it can slow cognitive decline compared to standard care over 5 years.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
tralesinidase alfa
What this could lead to
If it works, this could slow cognitive decline in children with Sanfilippo syndrome type B, offering a treatment option for this rare, devastating disease.
What could go wrong
This is a small, early-stage trial with only 14 participants. The drug is given directly into the brain, which carries risks like infection or side effects. Success is not guaranteed.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 14 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Dec 2026

An estimate. Start dates often move.

Expected to finish

Aug 2033

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 5 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity during screening. Has nonattenuated severe MPS IIIB by the Genotype and Clinical Assessment Committee. Is ≥1 and ≤5 years of age with BSID-III-C raw score of \<70. Is male or female as identified at birth. Exclusion Criteria: Genotyped for a known MPS IIIB variant associated with an attenuated phenotype or has a sibling with a known attenuated phenotype. Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry. Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.

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Conditions

The condition(s) this trial relates to.

Mucopolysaccharidosis III mucopolysaccharidosis type 3B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MPS iiib

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••