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Can a new enzyme therapy tame MPS II over time?

NCT ID NCT03708965

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 08, 2026 · Last updated Sep 09, 2026 · Updated 1 time

Summary

This study tests the long-term safety and effects of JR-141, an enzyme replacement therapy, in people with mucopolysaccharidosis II (MPS II), a rare genetic disorder. Participants who completed an earlier JR-141 trial receive the drug again by IV infusion. Researchers track side effects and measure markers like urinary and blood sugar levels, liver and spleen size, and heart function to see if the treatment keeps working.

What this could mean

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

Active substance
JR-141 (also known as pabinafusp alfa), an enzyme replacement therapy given by IV infusion
What this could lead to
If JR-141 proves safe and effective over the long term, it could offer a lasting treatment option for MPS II, potentially slowing or easing the disease's effects.
What could go wrong
This is a small, open-label extension study, so it may not detect rare side effects or prove long-term benefit. The drug may also not work equally well in all patients.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

19 people

The number who actually took part.

Started

Jan 2019

Expected to finish

Dec 2027

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

0 years and older

Sex

Male participants only

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: * Patients who will have completed clinical trial JR-141-BR21. * Capable of providing written consent by himself, unless the patient is under the age of 18 years at the time of informed consent process, or it is not possible to obtain consent from the patient himself due to his intellectual disabilities associated with MPS II. * In the case of a patient who is under the age of 18 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MSP II, he may be included if written consent can be provided by legal representative; however written consent should be obtained from the patient himself too, wherever possible. Exclusion Criteria: * Refusal to sign the informed consent form. * Unable to perform the study procedures, except for neurocognitive testing. * Previous engrafted BMT/HSCT. * Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumber puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process. * Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or sensitivity. * Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study out of consideration for the subject safety.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 2

As listed by the trial registrant

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

Mucopolysaccharidosis ii

Contacts and locations

Locations

  • Grupo de Pesquisa Clínica em Genética Médica - HCPA

    Porto Alegre, Brazil

  • Igeim - Unifesp

    São Paulo, Brazil

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