Mucopolysaccharidosis IPhase 1/2NCT04453085
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
A Phase 1/2 study testing JR-171 in people with Mucopolysaccharidosis I, run by JCR Pharmaceuticals Co., Ltd. at 6 sites in 3 countries.
Trial summary
Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis I
- What is being tested?
- JR-171
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 28 Oct 2021to 2 May 2025
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who have completed the Part 2 of JR-171-101 study
- A patient from whom written informed consent can be obtained. If the patient is aged under 18 years (20 years in case of Japan) at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g., his/her parents or guardians) need to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible
- Female patient or male patient whose co-partners is of child-bearing potential agree to use a medically accepted, highly effective method of contraception, such as spermatocidal gel plus condom, an intrauterine device or oral contraceptives until one month after the final administration.
Who cannot join
- A patient who is unable to perform the study procedures, except for 6-minute walk test, neurocognitive testing, BVMT-R, HVLT-R, and T.O.V.A.
- Judged by the principal investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumbar puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process
- Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or hypersensitivity to any drugs
- Otherwise judged by the principal investigator or subinvestigator to be ineligible to participate in the study in consideration of patient's safety
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
Japan3 sites
Fukuoka Children's Hospital
Fukuoka
National Hospital Organization Kokura Medical Center
Kitakyushu
Osaka Metropolitan University Hospital
Osaka
Brazil2 sites
Hospital de Clínicas de Porto Alegre
Porto Alegre
Instituto de Genética e Erros Inatos do Metabolismo - IGEIM
São Paulo
United States1 site
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
Official title: An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I
Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I
Read the detailed description
Patients who have completed the Part2 of JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study.
- Conditions
- Mucopolysaccharidosis I
- Being tested
- JR-171
- Keywords
- Mucopolysaccharidosis I
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JR-171Drug
Also called lepunafusp alfa
Intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Dose level 1 | Experimental | 2.0 mg/kg/week Receives: Drug: JR-171 |
| Dose level 2 | Experimental | 4.0 mg/kg/week Receives: Drug: JR-171 |
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
Measured: 156 weeks
Incidence of abnormal laboratory test results
Hematology
Measured: 156 weeks
Incidence of abnormal laboratory test results
Biochemistry
Measured: 156 weeks
Incidence of abnormal laboratory test results
Serum iron tests
Measured: 156 weeks
Incidence of abnormal laboratory test results
Urinalysis
Measured: 156 weeks
Incidence of abnormal vital signs
Pulse rate
Measured: 156 weeks
Incidence of abnormal vital signs
Body temperature
Measured: 156 weeks
Incidence of abnormal vital signs
Blood pressure
Measured: 156 weeks
Incidence of abnormal vital signs
Body weight
Measured: 156 weeks
Incidence of abnormal EKG readings
Measured: 156 weeks
Number of participants with Adverse Events
Anti-human-α-L-iduronidase antibodies
Measured: 156 weeks
Number of participants with Adverse Events
Anti-JR-171 antibodies
Measured: 156 weeks
Number of participants with Adverse Events
Infusion associated reaction (IAR)
Measured: 156 weeks
Secondary outcomes (22)
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid (CSF)
Measured: Weeks 26, 52, 104, 156
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid
Measured: Weeks 26, 52, 104, 156
CSF opening pressure
Measured: Weeks 26, 52, 104, 156
Change From Baseline in Heparan Sulfate Levels in Urinary
Measured: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Dermatan Sulfate Levels in Urinary
Measured: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Heparan Sulfate Levels in Serum
Measured: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Dermatan Sulfate Levels in Serum
Measured: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Liver Volume
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Spleen Volume.
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Left ventricular posterior wall thickness
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
interventricular septal thickness
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
left ventricular mass index
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
left ventricular ejection fraction
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
left ventricular ejection fraction,
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
E/A ratio
Measured: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in 6-minute Walk Test Distance.
Measured: Weeks 13, 26, 52, 78, 104, 156
Changes from baseline in cognitive age equivalent score of neurocognitive testing
Wechsler Intelligence Quotient (IQ) test or the Bayley scales of infant and toddler development
Measured: Weeks 52, 104, 156
Changes from baseline in outcome of adaptive behavioral function
Vineland adaptive behavior scales
Measured: Weeks 52, 104, 156
Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised
Measured: Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised
Measured: Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Test of Variables of Attention
Measured: Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Pediatric Quality of Life Inventory Family Impact Module (PedsQL-FIM)
Measured: Weeks 13, 26, 52, 104 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2020
- Study start28 Oct 2021
- Primary completion2 May 2025
- Study completion2 May 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IStudies 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
- Randomised
- Intervention model
- Parallel groups
- 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
- NCT04453085
- Sponsor's protocol ID
- JR-171-102
- Status last verified
- Aug 2025
- First submitted
- 4 Jun 2020
- Last updated
- 28 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I
- ClinicalTrials.gov ID
- NCT04453085
- Acronym / sponsor ID
- JR-171-102
- Conditions
- Mucopolysaccharidosis I
- Keywords
- Mucopolysaccharidosis I
- 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
- 28 Oct 2021 (actual)
- Primary completion
- 2 May 2025 (actual)
- Study completion
- 2 May 2025 (actual)
- First posted
- 1 Jul 2020 (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.
Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I
Detailed description
Patients who have completed the Part2 of JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose level 1 | Experimental | Drug: JR-1712.0 mg/kg/week |
| Dose level 2 | Experimental | Drug: JR-1714.0 mg/kg/week |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients who have completed the Part 2 of JR-171-101 study
- A patient from whom written informed consent can be obtained. If the patient is aged under 18 years (20 years in case of Japan) at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g., his/her parents or guardians) need to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible
- Female patient or male patient whose co-partners is of child-bearing potential agree to use a medically accepted, highly effective method of contraception, such as spermatocidal gel plus condom, an intrauterine device or oral contraceptives until one month after the final administration.
Key exclusion criteria (4 listed)
- A patient who is unable to perform the study procedures, except for 6-minute walk test, neurocognitive testing, BVMT-R, HVLT-R, and T.O.V.A.
- Judged by the principal investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumbar puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process
- Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or hypersensitivity to any drugs
- Otherwise judged by the principal investigator or subinvestigator to be ineligible to participate in the study in consideration of patient's safety
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-171Also: lepunafusp alfaIntravenous (IV) infusion | Drug | Dose level 1, Dose level 2 |
Primary outcome
Number of participants with Adverse Events
Time frame: 156 weeks
Adverse events
Incidence of abnormal laboratory test results
Time frame: 156 weeks
Hematology
Incidence of abnormal laboratory test results
Time frame: 156 weeks
Biochemistry
Incidence of abnormal laboratory test results
Time frame: 156 weeks
Serum iron tests
Incidence of abnormal laboratory test results
Time frame: 156 weeks
Urinalysis
Incidence of abnormal vital signs
Time frame: 156 weeks
Pulse rate
Incidence of abnormal vital signs
Time frame: 156 weeks
Body temperature
Incidence of abnormal vital signs
Time frame: 156 weeks
Blood pressure
Incidence of abnormal vital signs
Time frame: 156 weeks
Body weight
Incidence of abnormal EKG readings
Time frame: 156 weeks
Number of participants with Adverse Events
Time frame: 156 weeks
Anti-human-α-L-iduronidase antibodies
Number of participants with Adverse Events
Time frame: 156 weeks
Anti-JR-171 antibodies
Number of participants with Adverse Events
Time frame: 156 weeks
Infusion associated reaction (IAR)
22 secondary outcomes
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid (CSF)
Time frame: Weeks 26, 52, 104, 156
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid
Time frame: Weeks 26, 52, 104, 156
CSF opening pressure
Time frame: Weeks 26, 52, 104, 156
Change From Baseline in Heparan Sulfate Levels in Urinary
Time frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Dermatan Sulfate Levels in Urinary
Time frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Heparan Sulfate Levels in Serum
Time frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Dermatan Sulfate Levels in Serum
Time frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Liver Volume
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Spleen Volume.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in 6-minute Walk Test Distance.
Time frame: Weeks 13, 26, 52, 78, 104, 156
Changes from baseline in cognitive age equivalent score of neurocognitive testing
Time frame: Weeks 52, 104, 156
Changes from baseline in outcome of adaptive behavioral function
Time frame: Weeks 52, 104, 156
Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised
Time frame: Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised
Time frame: Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Test of Variables of Attention
Time frame: Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Pediatric Quality of Life Inventory Family Impact Module (PedsQL-FIM)
Time frame: Weeks 13, 26, 52, 104 156
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 28 Aug 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.
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing JR-171 in people with Mucopolysaccharidosis I, run by JCR Pharmaceuticals Co., Ltd. at 6 sites in 3 countries.
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 28 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Japan, Brazil, United States. 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: 156 weeks). The study lists 13 primary outcomes in all. 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 28 Aug 2025.