Long-term Safety and Efficacy Study of JR-141 (Pabinafusp Alfa) for Male Patients with Hunter Syndrome (Mucopolysaccharidosis II)

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What is this study about?

This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis Type II, also known as Hunter Syndrome. This condition affects the body’s ability to break down certain complex sugars, leading to various health issues. The study involves a treatment called JR-141, which is a medication in the form of a lyophilized powder that is prepared for injection. The active substance in JR-141 is pabinafusp alfa, a type of protein. The medication is administered through an intravenous injection, which means it is given directly into a vein.

The purpose of this study is to evaluate the long-term effects of JR-141 on symptoms affecting the central nervous system in individuals with Hunter Syndrome. Participants in this study will have previously completed a related study and will continue to receive JR-141 to assess its safety and effectiveness over an extended period. The study will monitor various health aspects, including liver and spleen size, shoulder movement, and walking ability, as well as other health indicators like heart and lung function.

Throughout the study, participants will undergo regular assessments to track their quality of life, sleep patterns, and overall health. These assessments will help researchers understand how JR-141 impacts the symptoms of Hunter Syndrome over time. The study aims to provide valuable insights into the long-term benefits and safety of JR-141 for individuals living with this condition.

1 joining the study

Participation in this study requires completion of a previous study, JR-141-GS31, and the absence of safety concerns as determined by the principal investigator.

Written informed consent must be obtained from the participant or their legally acceptable representative. If the participant is underage or unable to provide consent due to intellectual disability, consent can be provided by a guardian.

2 treatment administration

The treatment involves the administration of JR-141, which is a lyophilized powder prepared for injection.

The medication is administered through an intravenous injection. The specific dosage and frequency are determined by the study protocol and the healthcare team.

3 monitoring and assessments

Regular monitoring includes assessments of cerebrospinal fluid (CSF) concentrations, neuropsychological evaluations, and quality of life assessments.

Additional evaluations may include liver and spleen volume, shoulder range of motion, a six-minute walk test, and pulmonary function tests if applicable.

4 safety evaluations

Safety is continuously assessed through laboratory tests, vital signs monitoring, electrocardiograms (ECG), and physical examinations.

The presence of anti-JR-141 antibodies and any adverse events are also monitored throughout the study.

5 study duration

The study is expected to continue until April 30, 2029.

Participants will be involved in regular assessments and follow-ups as outlined in the study protocol.

Who Can Join the Study?

  • The patient must have participated in the previous study (JR-141-GS31) and completed the necessary assessments at specific weeks, with no safety concerns according to the main doctor in charge.
  • The patient or their legal representative must sign a written consent form approved by an ethics committee. If the patient is under 18 years old (or under 16 in the UK) or cannot confirm their willingness to participate due to intellectual disability from MPS II, a parent or guardian can sign on their behalf. If possible, the patient should also provide written agreement to participate.
  • If the patient is a female who can have children or a male with a female partner who can have children, they must agree to use a highly effective method of birth control from the time they sign the consent form. This applies from the start of menstruation until menopause unless they are permanently unable to have children.
  • If the patient has hearing problems and needs hearing aids, they should agree to use them during the study and on days when cognitive tests are done. The study team will encourage the use of working hearing aids.

Who Cannot Join the Study?

  • Patients who have a different condition than Mucopolysaccharidosis II (also known as Hunter Syndrome) cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who do not meet the specific health requirements set by the study cannot participate.
  • Patients who are unable to follow the study procedures or instructions cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are taking medications that might affect the study results cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have a history of allergic reactions to the study medication cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Uniwersytecki Szpital Dzieciecy W Krakowie Cracow Poland
SphinCS GmbH Hochheim Am Main Germany
Virgen del Rocío University Hospital Sevilla Spain
Centre Hospitalier Universitaire De Montpellier Montpellier France
Assistance Publique Hopitaux De Paris Paris France
Justus-Liebig-Universitaet Giessen Giessen Germany
Centre Hospitalier Lyon Sud Pierre Benite France
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Urjcggtrvh Mvubihd Cgztmz Hblpxdukpgpqanhee Hamburg Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
01.04.2024
Germany Germany
Recruiting
01.04.2024
Italy Italy
Recruiting
01.04.2024
Poland Poland
Recruiting
01.04.2024
Spain Spain
Recruiting
01.04.2024

Trial locations

Investigated drugs:

JR-141 is a medication being studied for its long-term safety and effectiveness in treating symptoms of Mucopolysaccharidosis Type II, also known as Hunter Syndrome. This medication is specifically being evaluated for its impact on central nervous system symptoms in male patients with this condition.

Investigated diseases:

Mucopolysaccharidosis II – This is a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. It leads to the accumulation of glycosaminoglycans in the body’s cells, which can cause a variety of symptoms. These symptoms often include developmental delays, distinctive facial features, and joint stiffness. Over time, individuals may experience progressive physical and neurological decline. The disease primarily affects males and can vary in severity. It is also known as Hunter Syndrome.

Trial ID:
2022-503142-41-00
Protocol code:
JR-141-GS32
NCT ID:
NCT05594992
Trial Phase:
Therapeutic confirmatory (Phase III)

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