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Can a weekly infusion slow the toll of a rare genetic disease?

NCT ID NCT04348136

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 03, 2026 · Last updated Sep 04, 2026 · Updated 1 time

Summary

This trial tests whether JR-141, an enzyme replacement therapy given as a weekly IV infusion, can safely manage mucopolysaccharidosis type II (MPS II) over the long term. Researchers will follow about 27 people who completed an earlier JR-141 study, tracking side effects, developmental progress, and markers like blood and urine sugar levels. The goal is to see if continued treatment keeps helping without new safety concerns.

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, an enzyme replacement therapy given by IV infusion weekly
What this could lead to
If it works, JR-141 could offer a long-term treatment option to slow or manage the effects of MPS II, potentially improving quality of life.
What could go wrong
This is an extension study with a small group, so results may not apply broadly. Long-term safety and effectiveness are still unproven, and side effects or lack of benefit remain possible.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

27 people

The number who actually took part.

Started

Sep 2019

Expected to finish

Mar 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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: 1. A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator. 2. A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.) 3. For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.) 4. Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception. Exclusion Criteria: 1. A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator. 2. A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator. 3. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.

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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

  • Fukui Clinical site

    Fukui, 910-1193, Japan

  • Fukuoka Clinical site

    Fukuoka, 830-0011, Japan

  • Fukuoka Clinical site 2

    Fukuoka, 813-0017, Japan

  • Gifu Clinical site

    Gifu, 501-1194, Japan

  • Hiroshima Prefectural Hospital

    Hiroshima, 734-8530, Japan

  • Hokkaido Clinical site

    Hokkaido, 063-0005, Japan

  • Kananagawa Ckinical site

    Kanagawa, 232-8555, Japan

  • Kumamoto Clinical site

    Kumamoto, 860-8556, Japan

  • Okayama Clinical site

    Okayama, 701-1192, Japan

  • Okayama Clinical site 2

    Okayama, 710-8602, Japan

  • Okinawa Clinical site

    Okinawa, 903-0215, Japan

  • Osaka Clinical site

    Osaka, 565-0871, Japan

  • Osaka Clinical site 2

    Osaka, 545-8586, Japan

  • Osaka Clinical site 3

    Osaka, 534-0021, Japan

  • Saitama Clinical site

    Saitama, 330-8777, Japan

  • Shizuoka Clinical site

    Shizuoka, 420-8660, Japan

  • Shizuoka Clinical site 2

    Shizuoka, 426-8677, Japan

  • Tochigi Clinical site

    Tochigi, 329-0498, Japan

  • Tokyo Clinical site

    Tokyo, 157-8535, Japan

  • Tottori Clinical site

    Tottori, 683-8504, Japan

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