A Study of JR-141 in Patients With Mucopolysaccharidosis II

• Register: ClinicalTrials.gov
• Last refreshed on: 21 November 2022
• Main ID: NCT03359213
• Date of registration: 17/11/2017
• Prospective Registration: Yes
• Primary sponsor: JCR Pharmaceuticals Co., Ltd.
• Public title: A Study of JR-141 in Patients With Mucopolysaccharidosis II
• Scientific title: Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
• Date of first enrolment: July 26, 2018
• Target sample size: 18
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03359213
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Brazil

Key inclusion & exclusion criteria

Inclusion Criteria:

- Males with confirmed diagnosis of MPS II, based on deficient activity of IDS in
leucocytes or fibroblasts and/or pathogenic mutations identified in the IDS gene (if
enzyme diagnosis was in dried blood spots or plasma, molecular genetics confirmation
is mandatory).

- One of the following age groups:

1. 0 to 3 years and 11 months old (6 patients, 2 in each dose)

2. 4 years to 7 years and 11 months old (6 patients, 2 in each dose)

3. 8 years or older (6 patients, 2 in each dose)

- 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.

- Naïve patients or patients who are receiving enzyme replacement therapy with
idursulfase could be included if provided treatment has been stable in the last 6
months and agree to interrupt the treatment at least one week before the first study
drug infusion, and agree in suspending this treatment for the duration of the trial.

Exclusion Criteria:

- Refusal to sign the informed consent form.

- Unable to perform the study procedures, except for neurocognitive testing.

- Previous engrafted BMT/HSCT.

- Surgical or other major medical intervention planned to occur before week 26.

- Participation in a clinical trial with an investigational drug in the last 12 months.

- 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.



Age minimum: 0 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis II

Intervention(s)

Drug: JR-141

Primary Outcome(s)

Number of participants with Adverse Events [Time Frame: 26 weeks]

Secondary Outcome(s)

Urinary heparan sulfate concentrations [Time Frame: 26 weeks]

Serum dermatan sulfate concentrations [Time Frame: 26 weeks]

Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] [Time Frame: 21 hours after dosing at the first and last infusions]

Liver and spleen volumes (MRI) [Time Frame: 26 weeks]

Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] [Time Frame: 21 hours after dosing at the first and last infusions]

Left ventricular mass by a standard 2-dimensional Doppler echocardiogram [Time Frame: 26 weeks]

Urinary dermatan sulfate concentrations [Time Frame: 26 weeks]

Serum heparan sulfate concentrations [Time Frame: 26 weeks]

Secondary ID(s)

JR-141-BR21

Source(s) of Monetary Support

Please refer to primary and secondary sponsors