Mucopolysaccharidosis IIPhase 2/3NCT03568175
A Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase 2/3 study testing JR-141 in people with Mucopolysaccharidosis II, run by JCR Pharmaceuticals Co., Ltd. at 19 sites in Japan.
Trial summary
A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II. Read more about this study
- Who is it for?
- Any age, 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
- 28 participants
- Where
- 19 sitesJapan
- Study dates
- 1 Aug 2018to 20 Feb 2020
- Phase
- Phase 2/3
- 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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
- In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to intellectual disabilities associated with MPS 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, whenever possible)
- Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
- Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.
Who cannot join
- Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
- Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely 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 serious drug allergy or sensitivity.
- Patients who have received other investigational product within 4 months before enrollment in the study.
- Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
Japan19 sites
Fukui Clinical site
Fukui, 910-1193
Fukuoka Clinical site 2
Fukuoka, 813-0017
Fukuoka Clinical site
Fukuoka, 830-0011
Gifu Clinical site
Gifu, 501-1194
Hokkaido Clinical site
Hokkaido, 063-0005
Kanagawa Clinical site
Kanagawa, 232-8555
Kumamoto Clinical site
Kumamoto, 860-8556
Okayama Clinical site
Okayama, 701-1192
Okayama Clinical site 2
Okayama, 710-8602
Okinawa Clinical site
Okinawa, 903-0215
Osaka Clinical site 3
Osaka, 534-0021
Osaka Clinical site 2
Osaka, 545-8586
Osaka Clinical site
Osaka, 565-0871
Saitama Clinical site
Saitama, 330-8777
Shizuoka Clinical site
Shizuoka, 420-8660
Shizuoka Clinical site 2
Shizuoka, 426-8677
Tochigi Clinical site
Tochigi, 329-0498
Tokyo Clinical site
Tokyo, 157-8535
Tottori Clinical site
Tottori, 683-8504
About this study
Official title: A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety 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, 2.0 mg/kg/week
| Study group | Role | What they receive |
|---|---|---|
| JR-141 2.0 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.
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Measured: Baseline to 52 weeks
Secondary outcomes (14)
Change From Baseline in Serum Heparan Sulfate Levels.
Measured: Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Serum Dermatan Sulfate Levels.
Measured: Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Urinary Heparan Sulfate Levels.
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in Urinary Dermatan Sulfate Levels.
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in Liver Volumes.
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in Spleen Volumes.
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in Cardiac Function.
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in 6-minute Walk Test Distance.
Item 9 will be administrated only in patients judged by the investigator or subinvestigator to be possible to perform the 6-minutes walk test
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in Joint Range of Motion.
Measured: Baseline, 25 weeks, 52 weeks
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.
Measured: Baseline to 25 weeks
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.
Measured: Baseline, 25 weeks, 52 weeks
Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)
Measured: 25, 52 weeks
Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )
Measured: 25, 52 weeks
Drug concentration in Cerebrospinal Fluid.
*Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .
Measured: 25, 52*weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2018
- Study start1 Aug 2018
- Primary completion20 Feb 2020
- Study completion20 Feb 2020
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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 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
- NCT03568175
- Sponsor's protocol ID
- JR-141-301
- Status last verified
- Nov 2022
- First submitted
- 1 Jun 2018
- Last updated
- 14 Nov 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
- ClinicalTrials.gov ID
- NCT03568175
- Acronym / sponsor ID
- JR-141-301
- Conditions
- Mucopolysaccharidosis II
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Aug 2018 (actual)
- Primary completion
- 20 Feb 2020 (actual)
- Study completion
- 20 Feb 2020 (actual)
- First posted
- 26 Jun 2018 (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/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| JR-141 2.0 mg/kg/week | Experimental | Drug: JR-141 |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
- In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to intellectual disabilities associated with MPS 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, whenever possible)
- Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
- Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.
Key exclusion criteria (5 listed)
- Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
- Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely 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 serious drug allergy or sensitivity.
- Patients who have received other investigational product within 4 months before enrollment in the study.
- Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.
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, 2.0 mg/kg/week | Drug | JR-141 2.0 mg/kg/week |
Primary outcome
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Time frame: Baseline to 52 weeks
14 secondary outcomes
Change From Baseline in Serum Heparan Sulfate Levels.
Time frame: Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Serum Dermatan Sulfate Levels.
Time frame: Baseline, 24-26 weeks, 50-52 weeks
Change From Baseline in Urinary Heparan Sulfate Levels.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in Urinary Dermatan Sulfate Levels.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in Liver Volumes.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in Spleen Volumes.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in Cardiac Function.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in 6-minute Walk Test Distance.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in Joint Range of Motion.
Time frame: Baseline, 25 weeks, 52 weeks
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.
Time frame: Baseline to 25 weeks
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.
Time frame: Baseline, 25 weeks, 52 weeks
Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)
Time frame: 25, 52 weeks
Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )
Time frame: 25, 52 weeks
Drug concentration in Cerebrospinal Fluid.
Time frame: 25, 52*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 14 Nov 2022.
- A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-IILink provided by the sponsor.
Related trials for Mucopolysaccharidosis II
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
Enrolling by invitationPhase 2/3
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Mucopolysaccharidosis II
Active, not recruitingPhase 3
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
Mucopolysaccharidosis II
Active, not recruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome
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.
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing JR-141 in people with Mucopolysaccharidosis II, run by JCR Pharmaceuticals Co., Ltd. at 19 sites in Japan.
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 4 inclusion and 5 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 14 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in Japan. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid (time frame: Baseline to 52 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 14 Nov 2022.