JR-141 for Hunter Syndrome
Palo Alto (17 mi)Age: Any Age
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: JCR Pharmaceuticals Co., Ltd.
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 1 jurisdiction
Trial Summary
What is the purpose of this trial?This trial is testing a new medicine for patients with MPS II to see if it is safe and effective. Previous research in animals helped design this trial to evaluate the new medicine for MPS II patients.
Do I need to stop my current medications to join the trial?The trial does not specify if you need to stop your current medications. However, if you are on stable enzyme replacement therapy with idursulfase, you can continue it. There is no mention of a washout period for other medications.
Is the drug JR-141 a promising treatment for Hunter Syndrome?The drug JR-141, also known as IZCARGO or pabinafusp alfa, is considered a promising treatment for Hunter Syndrome because it offers potential benefits for patients with this condition.1251013
What data supports the idea that JR-141 for Hunter Syndrome is an effective treatment?The available research does not provide any data on JR-141 for Hunter Syndrome. Instead, it focuses on treatments for juvenile idiopathic arthritis, which is a different condition. Therefore, there is no information here to support the effectiveness of JR-141 for Hunter Syndrome.67111214
What safety data exists for JR-141 treatment for Hunter Syndrome?The provided research does not contain safety data for JR-141, Idursulfase, Elaprase, IZCARGO, or pabinafusp alfa related to Hunter Syndrome. The studies focus on treatments for juvenile chronic arthritis and juvenile idiopathic arthritis, not Hunter Syndrome.348914
Eligibility Criteria
This trial is for patients with Hunter Syndrome (MPS II). Eligible participants include those diagnosed with MPS II, either treatment-naïve or on stable enzyme therapy. They must agree to use effective contraception and sign consent forms. There are specific age-related cognitive criteria for two separate cohorts: Cohort A includes children aged 36-71 months with certain developmental scores, while Cohort B includes individuals aged 6 years or older with an IQ of 70 or higher.Inclusion Criteria
I have been diagnosed with MPS II.
Exclusion Criteria
I have had a successful stem cell transplant.
I have a genetic mutation linked to developmental delays or seizures.
I have had brain surgery or a shunt placed in the last 30 days, or my shunt isn't working well.
I have received gene therapy treatment before.
I have a documented loss of sulfatase activity, not including IDS.
I cannot have a lumbar puncture procedure.
I may have an infection or a higher risk of bleeding due to my health condition or treatments.
Treatment Details
The STARLIGHT Phase III trial is testing the safety and effectiveness of a drug called JR-141 compared to Idursulfase in treating MPS II. Participants will be randomly assigned to receive either JR-141 alone, JR-141 combined with Idursulfase, or only Idursulfase in a blinded manner where assessors do not know which treatment each participant receives.
3Treatment groups
Experimental Treatment
Group I: administered as the standard of care: idursulfase (ELAPRASE®)Experimental Treatment1 Intervention
standard of care-controlled study
Group II: Rescue armExperimental Treatment1 Intervention
Group III: JR-141 2.0 mg/kg/weekExperimental Treatment1 Intervention
JR-141 is already approved in Japan for the following indications:
🇯🇵 Approved in Japan as IZCARGO for:
- Mucopolysaccharidosis type II (MPS II)
- Hunter syndrome
Find a clinic near you
Research locations nearbySelect from list below to view details:
Columbia UniversityNew York, NY
Phoenix Children's HospitalPhoenix, AZ
Columbia UniversityNew York City, NY
Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, IL
More Trial Locations
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Who is running the clinical trial?
JCR Pharmaceuticals Co., Ltd.Lead Sponsor
References
Separation and characterization of complement-fixing immune complexes in juvenile rheumatoid arthritis patients. [2019]Immune complexes (IC) in sera from patients with juvenile rheumatoid arthritis (JRA) were isolated by the use of immunoabsorbent columns. Sera from 14 JRA patients (four seropositive for 19S IgM RF and 10 seronegative, but nine having hidden 19S IgM RF) were analyzed by the anti-human Clq (alpha HClq) and anti-human C3 (alpha HC3) columns. The columns were sequentially eluted with veronal buffer, 0.02 M EDTA, 0.5 M NaCl, and 1 M propionic acid. By the alpha HClq column, IgM RF were detected in at least one of the separated IC fractions of 13 of 14 patients and IgG RF in three patients. By the alpha HC3 column, only five patients demonstrated IgM RF and only one IgG RF in the eluted fractions. On sucrose density gradient analysis (SDGA), all IC were demonstrated in the peaks greater than or equal to 19S.19S IgM RF were demonstrated by ELISA in all 14 patients, but IgG RF in only three. These studies demonstrate that complement-fixing 19S IgM RF, IgG, and IgG RF containing IC can be detected in the serum of JRA patients.
Influence of maternal antibody anti-Jra on the baby: a case report and pedigree chart. [2019]A Jr(a-) female (proposita) who has developed anti-Jra during her second pregnancy and her pedigree chart are reported. The antibody anti-Jra of the proposita and her baby was determined to be IgG and was eluted from their red cells. The baby's bilirubin rose to a peak on day 4 without clinical problem except for prophylactic phototherapy carried out. Two other examples of Jr(a-) blood type were detected in her elder sister and the first baby of the proposita with investigation of her pedigree chart.
Use of recombinant interferon gamma in pediatric patients with advanced juvenile chronic arthritis. [2019]Recombinant interferon (IFN) gamma was used in 10 patients, 6 to 15 years old, with juvenile chronic arthritis (JCA) for 5 to 11 years, resistant or with severe side effects to other treatments. Six patients had systemic JCA and 4 started as pauciarticular. Three of the latter became polyarticular. Treatment schedule was 50,000 IU-kg daily for 4 weeks, then 3 times per week for 3 months and twice a week up to 2 years. Eight cases had favourable clinical response. Prolonged steroid regime could be suspended in 7/8 cases who previously received it. Two patients with systemic JCA did not respond to IFN treatment. Side effects were fever (9), headache (8), chills (6), distal cyanosis, hypotension, leukopenia and myalgia (2), and vomiting (1). All were mild or moderate. IFN gamma was more tolerable than other drugs and seems to be beneficial for patients with JCA resistant to other treatments.
[Side effects of anti-TNFalpha therapy in juvenile idiopathic arthritis]. [2019]To report adverse events registered in our population affected by JIA and treated with anti-TNFalpha blockers.
Defining the Jr(a-) phenotype in the Japanese population. [2022]The Jr(a-) phenotype is rare in European and North American populations but is not so rare in Japanese and other Asian populations. Recently, two groups have established the connection between the Jr(a-) phenotype and the ATP-binding cassette, member G2 (ABCG2) gene and concluded that ABCG2-null alleles encode the Jr(a-) phenotype. In Japanese Red Cross Blood Centers, the Jr(a-) phenotype is found with a prevalence of 0.05% among blood donors, and we applied DNA-based genotyping to investigate the molecular basis of the Jr(a-) phenotype in Japan, in addition to serologic typing.
Biologic treatment response among adults with juvenile idiopathic arthritis: results from the British Society for Rheumatology Biologics Register. [2023]To describe the use of and response to biologic therapies commenced in adults with JIA.
Catch-up growth during tocilizumab therapy for systemic juvenile idiopathic arthritis: results from a phase III trial. [2022]To investigate the impact of tocilizumab treatment on growth and growth-related laboratory parameters in patients with systemic juvenile idiopathic arthritis (JIA) enrolled in a phase III clinical trial.
Short and long-term immunogenicity and safety following the 23-valent polysaccharide pneumococcal vaccine in juvenile idiopathic arthritis patients under conventional DMARDs with or without anti-TNF therapy. [2015]To assess immunogenicity and safety of the 23-valent polysaccharide pneumococcal vaccine (PPV23) in juvenile idiopathic arthritis (JIA) patients under conventional DMARDs with or without anti-TNF therapy. The influences of demographic data, disease activity and treatment on immune response and the potential deleterious effects of vaccine on disease itself were also evaluated.
Effectiveness and long-term retention of anti-tumour necrosis factor treatment in juvenile and adult patients with juvenile idiopathic arthritis: data from Reuma.pt. [2016]Assess the effectiveness and safety of biologic therapy as well as predictors of response at 1 year of therapy, retention rate in biologic treatment and predictors of drug discontinuation in JIA patients in the Portuguese register of rheumatic diseases.
Increased Prevalence of Human Polyomavirus JC Viruria in Chronic Inflammatory Rheumatic Diseases Patients in Treatment with Anti-TNF α: A 18 Month Follow-Up Study. [2020]Chronic inflammatory rheumatic diseases (CIRDs) are immune-mediated pathologies involving joints. To date, TNFα-blocking agents administration is the most promising therapy, although these treatments are associated with an increased Polyomavirus JC (JCPyV) reactivation, the etiological agent of the Progressive Multifocal Leukoencephalopathy (PML). The aim of this study was the recruitment and the analysis of a CIRDs cohort in order to investigate a possible correlation between JCPyV presence and the influence of anti-TNF-α agents on viral loads. Blood and urine samples were collected from 34 CIRDs subjects prior the first anti-TNF-α infusion (T0) and after 3 (T3), 6 (T6), 12 (T12), and 18 (T18) months. Results showed persistent JC viruria significantly higher than JC viremia throughout the 18 month follow-up study (p = 0.002). In JCPyV positive samples, the non-coding control region (NCCR) was analyzed. Results evidenced archetypal structures (type II-S) in all isolates with the exception of a sequence isolated from a plasma sample, that corresponds to the type II-R found in PML subjects. Finally, the viral protein 1 (VP1) genotyping was performed and results showed the prevalence of the European genotypes 1A, 1B, and 4. Since only few studies have been carried out to understand whether there is a PML risk in CIRDs population infected by JCPyV, this study contributes to enrich literature insight on JCPyV biology in this cluster. Further investigations are necessary in order to recognize the real impact of biologics on JCPyV life cycle and to identify possible and specific viral variants related to increased virulence in CIRDs patients.
Growth Outcomes After GH Therapy of Patients Given Long-Term Corticosteroids for Juvenile Idiopathic Arthritis. [2018]Growth hormone (GH) therapy may improve statural growth outcomes in patients with severe juvenile idiopathic arthritis (JIA).
Outcome of Juvenile Idiopathic Arthritis Associated Uveitis in Two Disease Subtypes. [2022]This study aims to evaluate the efficacy of adalimumab as a first line biologic agent in specific subtypes of juvenile idiopathic arthritis (JIA) patients with associated uveitis.
Fatal hemolytic disease of the fetus and newborn caused by anti-Jra antibody: A case report and literature review. [2020]The Jra antigen of the JR blood group system is a highly prevalent red blood cell antigen. Although anti-Jra-associated hemolytic disease of the fetus and newborn (HDFN) is generally considered mild-to-moderate, a rare fatal case was recently reported. We report the third example of HDFN-related anti-Jra with fatal outcomes. The clinical significance of anti-Jra antibody as a cause of HDFN should be reassessed.
Comparative effectiveness and persistence of TNFi and non-TNFi in juvenile idiopathic arthritis: a large paediatric rheumatology centre in the USA. [2023]To evaluate the persistence and effectiveness of TNF inhibitors (TNFi) vs non-TNFi among newly diagnosed JIA patients after initiation of biologic DMARD (bDMARD).