Mucopolysaccharidosis IIPhase 1/2NCT03128593
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
A Phase 1/2 study testing JR-141 in people with Mucopolysaccharidosis II, run by JCR Pharmaceuticals Co., Ltd. at 8 sites in Japan.
Trial summary
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below, to collect the safety information of JR-141 to evaluate the plasma pharmacokinetics of JR-141 to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms Read more about this study
- Who is it for?
- Ages 6 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
- 14 participants
- Where
- 8 sitesJapan
- Study dates
- 30 Mar 2017to 4 Oct 2017
- Phase
- Phase 1/2
- 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 6 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients aged 6 years or older at the time of informed consent.
- Patients diagnosed with MPS II.
- Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.
Who cannot join
- Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.
- Patients in whom lumbar puncture cannot be performed.
- Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.
- Patients who have received other investigational products within 4 months before enrollment in the study.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
Japan8 sites
Gifu Clinical site
Gifu, 501-1194
Fukuoka Clinical site
Kurume, 830-0011
Tokyo clinical site1
Minato, 105-8471
Osaka Clinical site1
Osaka, 545-8586
Saitama Clinical site
Saitama, 330-8777
Tokyo clinical site2
Setagaya City, 157-8535
Osaka Clinical site2
Suita, 157-8535
Tottori Clinical site
Yonago, 683-8504
About this study
Official title: A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,
- to collect the safety information of JR-141
- to evaluate the plasma pharmacokinetics of JR-141
- to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms
- 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), 0.01-2.0 mg/kg/week
| Study group | Role | What they receive |
|---|---|---|
| Experimental: JR-141 | 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
- Laboratory tests
- Vital signs
- 12-lead electrocardiogram
- Antibody
- Infusion associated reaction
Measured: 4 weeks
Secondary outcomes (7)
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Plasma concentration of JR-141
Measured: 4 weeks
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Plasma concentration of JR-141
Measured: 4 weeks
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
Measured: 4 weeks
HS/DS in CSF
JR-141 concentration in CSF
Measured: 4 weeks
Urinary total GAG
Measured: 4 weeks
Liver and spleen volumes
Measured: 4 weeks
Cardiac function
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2017
- Study start30 Mar 2017
- Primary completion4 Oct 2017
- Study completion4 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 14 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
- NCT03128593
- Sponsor's protocol ID
- JR-141-101
- Status last verified
- Nov 2022
- First submitted
- 10 Apr 2017
- Last updated
- 14 Nov 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y. Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2019 Feb 6;27(2):456-464. doi: 10.1016/j.ymthe.2018.12.005. Epub 2018 Dec 8.
PubMed 30595526
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 I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
- ClinicalTrials.gov ID
- NCT03128593
- Acronym / sponsor ID
- JR-141-101
- Conditions
- Mucopolysaccharidosis II
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 30 Mar 2017 (actual)
- Primary completion
- 4 Oct 2017 (actual)
- Study completion
- 4 Oct 2017 (actual)
- First posted
- 25 Apr 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.
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,
- to collect the safety information of JR-141
- to evaluate the plasma pharmacokinetics of JR-141
- to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: JR-141 | Experimental | Drug: JR-141 |
Who may qualify
- Ages
- 6 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients aged 6 years or older at the time of informed consent.
- Patients diagnosed with MPS II.
- Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.
Key exclusion criteria (4 listed)
- Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.
- Patients in whom lumbar puncture cannot be performed.
- Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.
- Patients who have received other investigational products within 4 months before enrollment 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 (lyophilized powder), 0.01-2.0 mg/kg/week | Drug | Experimental: JR-141 |
Primary outcome
Number of participants with Adverse Events
Time frame: 4 weeks
* Adverse events * Laboratory tests * Vital signs * 12-lead electrocardiogram * Antibody * Infusion associated reaction
7 secondary outcomes
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Time frame: 4 weeks
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Time frame: 4 weeks
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
Time frame: 4 weeks
HS/DS in CSF
Time frame: 4 weeks
Urinary total GAG
Time frame: 4 weeks
Liver and spleen volumes
Time frame: 4 weeks
Cardiac function
Time frame: 4 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.
- Derived from this study: PubMed 30595526Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y. Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2019 Feb 6;27(2):456-464. doi: 10.1016/j.ymthe.2018.12.005. Epub 2018 Dec 8.
- Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 TrialLink provided by the sponsor.
Related trials for Mucopolysaccharidosis II
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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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
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Active, not recruitingPhase 3
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
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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.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing JR-141 in people with Mucopolysaccharidosis II, run by JCR Pharmaceuticals Co., Ltd. at 8 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 3 inclusion and 4 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 8 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03128593, which also lists 1 linked publication.
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.