Mucopolysaccharidosis IIPhase 2NCT03359213
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
A Phase 2 study testing JR-141 in people with Mucopolysaccharidosis II, run by JCR Pharmaceuticals Co., Ltd. at 2 sites in Brazil.
Trial summary
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II. Read more about this study
- Who is it for?
- Ages 0 years and older, men only
- Conditions the study is for
- Mucopolysaccharidosis II
- What is being tested?
- JR-141
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesBrazil
- Study dates
- 26 Jul 2018to 4 Oct 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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:
- 0 to 3 years and 11 months old (6 patients, 2 in each dose)
- 4 years to 7 years and 11 months old (6 patients, 2 in each dose)
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Brazil2 sites
Grupo de Pesquisa Clínica em Genética Médica - HCPA
Porto Alegre
Igeim - Unifesp
São Paulo
About this study
Official title: Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.
- Conditions
- Mucopolysaccharidosis II
- Being tested
- JR-141
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JR-141Drug
IV infusion (lyophilized powder), 1.0 mg/kg/week
JR-141Drug
IV infusion (lyophilized powder), 2.0 mg/kg/week
JR-141Drug
IV infusion (lyophilized powder), 4.0 mg/kg/week
| Study group | Role | What they receive |
|---|---|---|
| JR-141 1.0 mg/kg/week | Experimental | Not described Receives: Drug: JR-141 |
| JR-141 2.0 mg/kg/week | Experimental | Not described Receives: Drug: JR-141 |
| JR-141 4.0 mg/kg/week | Experimental | Not described Receives: Drug: JR-141 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Adverse Events
- Adverse events
- Antidrug antibodies
- Blood pressures in mmHg
- Heart rate in beats/minute
- Respiratory rate in breaths/minute
- Temperature in °C
- Presence or absence of abnormalities for physical examination
- Presence or absence of abnormalities for 12-lead electrocardiogram
- Routine laboratory tests in blood (hematology, liver function, renal function, iron-related levels) and urine (urinalysis)
Measured: 26 weeks
Secondary outcomes (8)
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Plasma concentration of JR-141
Measured: 21 hours after dosing at the first and last infusions
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Plasma concentration of JR-141
Measured: 21 hours after dosing at the first and last infusions
Liver and spleen volumes (MRI)
Measured: 26 weeks
Left ventricular mass by a standard 2-dimensional Doppler echocardiogram
Measured: 26 weeks
Urinary heparan sulfate concentrations
Measured: 26 weeks
Urinary dermatan sulfate concentrations
Measured: 26 weeks
Serum heparan sulfate concentrations
Measured: 26 weeks
Serum dermatan sulfate concentrations
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2017
- Study start26 Jul 2018
- Primary completion4 Oct 2019
- Study completion4 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IIStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03359213
- Sponsor's protocol ID
- JR-141-BR21
- Status last verified
- Jul 2025
- First submitted
- 17 Nov 2017
- Last updated
- 24 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
- ClinicalTrials.gov ID
- NCT03359213
- Acronym / sponsor ID
- JR-141-BR21
- Conditions
- Mucopolysaccharidosis II
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 26 Jul 2018 (actual)
- Primary completion
- 4 Oct 2019 (actual)
- Study completion
- 4 Oct 2019 (actual)
- First posted
- 2 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| JR-141 1.0 mg/kg/week | Experimental | Drug: JR-141 |
| JR-141 2.0 mg/kg/week | Experimental | Drug: JR-141 |
| JR-141 4.0 mg/kg/week | Experimental | Drug: JR-141 |
Who may qualify
- Ages
- 0 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| JR-141IV infusion (lyophilized powder), 1.0 mg/kg/week | Drug | JR-141 1.0 mg/kg/week |
| JR-141IV infusion (lyophilized powder), 2.0 mg/kg/week | Drug | JR-141 2.0 mg/kg/week |
| JR-141IV infusion (lyophilized powder), 4.0 mg/kg/week | Drug | JR-141 4.0 mg/kg/week |
Primary outcome
Number of participants with Adverse Events
Time frame: 26 weeks
* Adverse events * Antidrug antibodies * Blood pressures in mmHg * Heart rate in beats/minute * Respiratory rate in breaths/minute * Temperature in °C * Presence or absence of abnormalities for physical examination * Presence or absence of abnormalities for 12-lead electrocardiogram * Routine laboratory tests in blood (hematology, liver function, renal function, iron-related levels) and urine (urinalysis)
8 secondary outcomes
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Time frame: 21 hours after dosing at the first and last infusions
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
Left ventricular mass by a standard 2-dimensional Doppler echocardiogram
Time frame: 26 weeks
Urinary heparan sulfate concentrations
Time frame: 26 weeks
Urinary dermatan sulfate concentrations
Time frame: 26 weeks
Serum heparan sulfate concentrations
Time frame: 26 weeks
Serum dermatan sulfate concentrations
Time frame: 26 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing JR-141 in people with Mucopolysaccharidosis II, run by JCR Pharmaceuticals Co., Ltd. at 2 sites in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Adverse Events (time frame: 26 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2025.