A Phase III study of JR-141 in MPS II (Hunter Syndrome) Patients
Research type
Research Study
Full title
A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients.
IRAS ID
1003734
Contact name
Tatsuyoshi Yamamoto
Contact email
Sponsor organisation
JCR Pharmaceuticals Co., Ltd.
Eudract number
2020-003200-14
Clinicaltrials.gov Identifier
Research summary
MPS II (Hunter syndrome) is a rare genetic and progressive disorder affecting many body parts. It is caused by a malfunctioning enzyme called Iduronate 2-sulfatase (IDS) which breakdown certain complex molecules such as glycosaminoglycans (GAGs). Due to lack of IDS, GAGs accumulate in different organs leading to an increase in the size of these organs.
Idursulfase (ELAPRASE®) is the only approved product and first-line treatment for MPS II patients in the US. Idursulfase enters cells in surrounding organs and breaks down GAGs in lysosomes (membrane-bound sacs of digestive enzymes) which has shown to improve clinical symptoms. However, it does not penetrate the blood-brain barrier (BBB), thus does not breakdown GAG accumulation in the central nervous system (CNS) which causes the neurologic symptoms of MPS II.
The study drug, JR-141 is a humanized monoclonal antibody (combined human and non-human antibody) which binds to transferrin receptor (TfR), a protein expressed on the cell surface of several tissues including components of the BBB. This binding allows JR-141 to cross the BBB and be distributed to tissues of the CNS.
This trial is designed to show changes in several biological markers and symptoms after up to two-year intravenous treatment with JR-141 in MPS II patients. JR-141 was designed to treat bodily as well as CNS symptoms. Completed Phase II/III study in Japan and Phase II study in Brazil have shown the reduction in GAG and the maintenance or improvement of neurocognitive development, in addition to bodily symptom improvements compared to idursulfase.
This study contains two cohorts to evaluate the effectiveness of JR-141 on both CNS symptoms and bodily symptoms. This study is conducted in several countries including the United Kingdom, countries in Europe, the United States and South America and will enroll 50 participants world-wide.
This study is sponsored by JCR Pharmaceuticals Co., Ltd.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
21/NE/0229
Date of REC Opinion
8 Feb 2022
REC opinion
Further Information Favourable Opinion