An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II Clinical Trial

Official title:

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03708965
• Other study ID #: JR-141-BR22
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: January 1, 2019
• Est. completion date: March 31, 2024

Study information

• Verified date: June 2023
• Source: JCR Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 19
• Est. completion date: March 31, 2024
• Est. primary completion date: March 31, 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 0 Years and older

Eligibility

Inclusion Criteria:

• Patients who will have completed clinical trial JR-141-BR21.
• Capable of providing written consent by himself, unless the patient is under the age of 18 years at the time of informed consent process, or it is not possible to obtain consent from the patient himself due to his intellectual disabilities associated with MPS II.
• In the case of a patient who is under the age of 18 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MSP II, he may be included if written consent can be provided by legal representative; however written consent should be obtained from the patient himself too, wherever possible.

Exclusion Criteria:

• Refusal to sign the informed consent form.
• Unable to perform the study procedures, except for neurocognitive testing.
• Previous engrafted BMT/HSCT.
• Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumber puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process.
• Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or sensitivity.
• Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study out of consideration for the subject safety.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis II

Intervention

Drug:
• JR-141
IV infusion (lyophilized powder)

Locations

Country	Name	City	State
Brazil	Grupo de Pesquisa Clínica em Genética Médica - HCPA	Porto Alegre
Brazil	Igeim - Unifesp	São Paulo

Sponsors (1)

Lead Sponsor	Collaborator
JCR Pharmaceuticals Co., Ltd.

Country where clinical trial is conducted

Brazil, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with Adverse Events		From screening up to the end of study, up to approximately 5 years
Secondary	Urinary heparan sulfate concentrations		through study completion, an average of 52 weeks, up to approximately 5 years
Secondary	Urinary dermatan sulfate concentrations		through study completion, an average of 52 weeks, up to approximately 5 years
Secondary	Serum heparan sulfate concentrations		through study completion, an average of 52 weeks, up to approximately 5 years
Secondary	Serum dermatan sulfate concentrations		through study completion, an average of 52 weeks, up to approximately 5 years
Secondary	Liver and spleen volumes (MRI)		through study completion, an average of 52 weeks, up to approximately 5 years
Secondary	Echocardiography		through study completion, an average of 52 weeks, up to approximately 5 years