Hunter syndrome drug JR-141 tested for Long-Term safety
NCT ID NCT05594992
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study is an extension of a previous trial testing JR-141, a drug given weekly by IV, for people with Hunter syndrome (a rare genetic disorder). It aims to see if the drug remains safe and effective over a longer period. About 80 participants who completed the earlier study will continue receiving JR-141, and researchers will track changes in spinal fluid markers, thinking skills, and physical abilities.
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 (a drug given by IV infusion weekly)
What this could lead to
If successful, this could confirm that JR-141 safely controls Hunter syndrome symptoms long-term, offering a continued treatment option for patients.
What could go wrong
This is an extension study, so it builds on earlier results but is still open-label (no placebo). Long-term benefits are not guaranteed, and side effects may emerge over time.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Hospital Sant Joan de Déu
Barcelona, Spain
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Hospital Universitario Austral
Buenos Aires, Argentina
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Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
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Hôpital Femme Mère Enfant
Bron, France
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Instituto de Genética e Erros Inatos do Metabolismo
São Paulo, Brazil
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Osp. Pediatrico Bambino Gesù, IRCCS
Rome, Italy
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
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Uniwersytecki Szpital Dziecięcy
Krakow, Poland