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Can a weekly infusion reach the brain to fight a rare childhood disease?

NCT ID NCT06095388

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 04, 2026 · Last updated Sep 04, 2026

Summary

This trial tests an experimental drug called JR-441 in children with mucopolysaccharidosis type IIIA (MPS IIIA), a rare genetic condition that harms the brain and body. Researchers give the drug as a weekly intravenous infusion to see if it is safe and tolerable. They also measure whether it changes markers of the disease and affects thinking and development. The study includes children aged 1 to 18 years who weigh at least 10 kilograms.

What this could mean

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

Active substance
JR-441 (posnafusp alfa), an experimental enzyme replacement therapy given by intravenous infusion
What this could lead to
If it works, this could point toward a treatment that slows or eases the severe brain and body effects of MPS IIIA in children.
What could go wrong
This is an early, small trial focused on safety, so the drug may not prove effective. Infusion reactions, including allergic-type responses, are a known risk.

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

15 people

The number who actually took part.

Started

Oct 2023

Expected to finish

Oct 2029

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 18 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 ≥1 year and ≤18 years. * Confirmed diagnosis of MPS IIIA. * Body weight ≥ 10 kg. 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 3A

As listed by the trial registrant

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

Mucopolysaccharidosis iii-A

Contacts and locations

Locations

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, Germany