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New enzyme therapy aims to reach the brain in hunter syndrome

NCT ID NCT05208281

NEW Disease control Sponsor: AO GENERIUM 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 21, 2026 · Last updated Sep 21, 2026

Summary

Researchers are testing a new enzyme replacement therapy called GNR-055 in people with Hunter syndrome (MPS II), a rare inherited disorder that causes harmful sugar molecules to build up in the body. The trial enrolls about 32 patients, including those who have never had enzyme treatment and those who have used standard idursulfase therapy. Participants receive weekly intravenous infusions of GNR-055 at different doses. The study measures safety, how the drug moves through the body, and whether it reduces GAG buildup in urine and spinal fluid.

What this could mean

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

Active substance
GNR-055, an enzyme replacement therapy given as a weekly intravenous infusion
What this could lead to
If it works, GNR-055 could offer another treatment option for Hunter syndrome, possibly reaching the brain in ways current enzyme therapies do not.
What could go wrong
The trial is small and early, so safety and effectiveness are not yet established. Weekly infusions can cause allergic reactions, and the body may make antibodies that reduce the drug's effect.

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

About 32 people

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

Started

Nov 2021

Expected to finish

Mar 2028

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: * Signed inform consent; * Verified diagnosis of MPS II (Hunter syndrome); * Naïve patients or patients who have received standard ERT whit idursulfase products; * No contraindications for lumbar puncture as judged by the Investigator; * Willingness and ability to follow study procedures. Exclusion Criteria: * Clinically pronounced hypersensitivity to ID2S or any other component of the drug product; * History of hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation; * Implanted or external non-removable metal devices, a cardiac pacemaker, or other objects sensitive to the magnetic field that may pose a danger to both the wearer and the correct operation of magnetic resonance imaging (MRI) equipment; * Concomitant diseases and conditions that, in the Investigator's opinion, can put at risk the patient's safety during his/her participation in the study, or which will influence the safety data analysis in case of the disease/condition exacerbation during the study.

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Conditions

The condition(s) this trial relates to.

autism, susceptibility to, X-linked 2 Cognition Disorders cognitive disorder Cognitive Dysfunction connective tissue disorder hereditary disease inherited neurodegenerative disorder intellectual disability lysosomal storage disease metabolic disease mucopolysaccharidosis type 2 nervous system disorder Neurobehavioral Manifestations Neurologic Manifestations psychiatric disorder Sudden Infant Death X-linked disease

As listed by the trial registrant

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

Metabolic diseases Mucopolysaccharidosis type II

Contacts and locations

Locations

  • Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation

    Saint Petersburg, 194100, Russia

  • Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Academy of Sciences (Research Institute of Pediatrics and Child Health Protection of the Central Clinical Hospital of the Russian Academy of Sciences)

    Moscow, 119333, Russia

  • State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital

    Yekaterinburg, 620149, Russia

  • State Budgetary Healthcare Institution Republican Medical Genetic Center

    Ufa, 450076, Russia

  • V.I. Vernadsky Crimean Federal University

    Simferopol, 295007, Russia

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