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New hope for kids with rare sanfilippo syndrome? early trial launches

NCT ID NCT06488924

Disease control Sponsor: JCR Pharmaceuticals Co., Ltd. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026

Summary

This early-phase study tests a new drug called JR-446 in 10 children with mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B), a rare genetic disease that affects the brain and body. The drug is given through an IV and aims to be safe and possibly help manage the condition. The main goals are to check safety and how the drug works in the body.

What this could mean

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

Active substance
JR-446 (a drug given by IV infusion)
What this could lead to
If it works, this could point toward a treatment that slows or manages MPS IIIB symptoms in children.
What could go wrong
This is a very early, small trial (only 10 children) focused on safety. It may not show clear benefit, and side effects are unknown.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 10 people

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

Started

Nov 2024

Expected to finish

Apr 2030

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

Up to 17 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: * Chronological age of \<18 years * Confirmed diagnosis of MPS IIIB Exclusion Criteria: * Prior experience to gene therapy or HSCT with successful engraftment * Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF * Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures * Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF * Serious drug allergy or hypersensitivity * Contraindication for lumbar puncture or MRI * History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.

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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.

Mucopolysaccharidosis iii-B

How to take part

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

  1. The places running it

    3 sites. The list below names each one and where it is.

  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

Locations

  • Hiroshima University Hospital

    RECRUITING

    Hiroshima, Japan

  • National Center for Child Health and Development

    RECRUITING

    Tokyo, Japan

  • University of the Ryukyus Hospital

    RECRUITING

    Okinawa, Japan

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