NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JRCT ID: jRCT2080225015

Registered date:14/01/2020

A Study of JR-171 in Patients With Mucopolysaccharidosis Type I

Basic Information

Recruitment status completed
Health condition(s) or Problem(s) studiedMucopolysaccharidosis I
Date of first enrollment01/09/2020
Target sample size19
Countries of recruitmentJapan,US,Brazil
Study typeInterventional
Intervention(s)investigational material(s) Generic name etc : INN of investigational material : - Therapeutic category code : 395 Enzyme preparations Dosage and Administration for Investigational material : control material(s) Generic name etc : INN of investigational material : - Therapeutic category code : Dosage and Administration for Investigational material :

Outcome(s)

Primary Outcomesafety
Secondary Outcomeefficacy pharmacokinetics

Key inclusion & exclusion criteria

Age minimumNot applicable
Age maximumNot applicable
GenderBoth
Include criteria1. A patient aged 18 years or older in Part 1 or any age in Part 2, at the time of informed consent. 2. 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 or assent should be obtained from the patient, wherever possible 3. A patient diagnosed with MPS I based on any one of the following criteria: Activity of IDUA enzyme below 10% of lower reference level in leucocytes or cultured skin fibroblasts, AND increased age-related urinary levels of GAGs (before enzyme replacement therapy) Activity of IDUA enzyme below 10% of lower reference level in leucocytes or cultured skin fibroblasts, AND presence of one pathogenic mutation in each of the alleles of the IDUA gene Increased age-related urinary levels of GAGs (before enzyme replacement therapy), AND presence of one pathogenic mutation in each of the alleles of the IDUA gene 4. A patient diagnosed as having no or mild MPS I-related intellectual disability (able to report their own subjective symptoms) by the principal investigator or subinvestigator (Part 1 only) 5. A patient who has received laronidase continuously for at least 12 weeks and has received laronidase on a stable dosage for 2 weeks immediately before the initial administration of JR-171, except for a laronidase naive patient or a patient who has previously been treated by HSCT) 6. Female patient or male patient whose co-partner is of child-bearing potential agrees 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
Exclude criteria1. A patient who received gene therapy treatment 2. A patient who, in the opinion of the principal investigator or subinvestigator, cannot undergo lumbar puncture, including those who have a difficulty in taking a position for lumbar puncture due to joint contracture and those who are likely to develop dyspnea during lumbar puncture 3. A patient who is pregnant or lactating 4. A patient who has developed serious drug allergy or hypersensitivity to any drugs, in the opinion of the principal investigator or subinvestigator, is inappropriate for participation in the study 5. A patient who has received another investigational product within 12 months before enrollment in the study 6. A patient who, in the opinion of the principal investigator or subinvestigator, is ineligible to participate in the study out of consideration for the participant safety.

Related Information

Contact

Public contact
Name Satoshi Kawashima
Address 11-18 Kusunoki-cho, Ashiya-shi, Hyogo 659-0015, Japan
Telephone +81-797-32-8582
E-mail clinical_development@jp.jcrpharm.com
Affiliation JCR Pharmaceuticals Co., Ltd,
Scientific contact
Name Satoshi Kawashima
Address 11-18 Kusunoki-cho, Ashiya-shi, Hyogo 659-0015, Japan
Telephone +81-797-32-8582
E-mail clinical_development@jp.jcrpharm.com
Affiliation JCR Pharmaceuticals Co., Ltd,