EUCTR2020-003200-14-FR进行中（未招募）1 期
A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 50
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. A patient from whom a voluntarily signed written IRB or Independent Ethics Committee (IEC)-approved ICF can be obtained. Informed permission from the patient’s legally accepted representative (e.g. his parent[s] or guardian[s]) needs to be obtained for a patient who is under 18 years of age (aged under 16 years in the UK) at the time of assent and/or for a patient where willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability. In these cases, written informed consent or assent should still be obtained from the patient, whenever possible
- •2. Males with confirmed diagnosis of MPS II, based on the documented mutation identified in the IDS gene and meeting one of the following criteria:
- •1) Deficient activity of IDS in leucocyte or fibroblasts
- •2) Increased levels of urinary glycosaminoglycans (GAGs) (or uronic acid) measured before the start of specific therapy
- •3. Naïve patients or patients who are receiving stable enzyme replacement therapy (ERT) with idursulfase for more than 12 weeks before starting administration of the JR-141 or idursulfase for this study.
- •4. Cohort A
- •- Males aged 36-71 months old at the time of ICF signed and whose development quotient (DQ) on the cognitive domain by the BSID-III or the nonverbal index by the KABC-II is between 55-75 at screening; OR
- •- Males aged 30-35 months old at the time of randomization and who are judged as having the severe phenotype by the Expert Board based on presence of one of the following mutations in the IDS gene and other information such as high CSF HS concentrations:
- •1) Large deletion or rearrangement
- •2) Small insertions or deletions that are out of frame
- •3) Missense mutations, nonsense mutations, in frame inserts or deletions that involve either active site residues or have been identified to be associated with severe, neuronopathic disease in either another family or other family members.
- •5. Cohort B
- •- Males aged 6 years or older at the time of ICF signed and whose intelligence quotient (IQ) on a Wechsler test (either WPPSI-IV, WISC-V, or WAIS-IV) is 70 and higher at screening
- •- Patients with 1SD deficiency in the omission errors or variability domains of the T.O.V.A. test or Processing Speed or Working memory on the Wechsler tests at screening
- •6. Male patients whose partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception being a condom plus an approved method of effective contraception from the time of informed consent.
- •The following methods are acceptable:
- •Partner’s use of combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation:
- •- intravaginal
- •- transdermal
- •Partner’s use of progestogen-only hormonal contraception:
- •- injectable/implantable
- •- intrauterine hormone-releasing system (IUS)
- •Partner’s use of implantable intrauterine device (IUD)
- •Surgical sterilization (for example, vasectomy or bilateral tubal occlusion)
- •Partner’s use of female cap or diaphragm (double barrier)
- •Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle. If a subject is usually not sexually active but becomes active, they, with their partner, must comply with the contraceptive requirements detailed above.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 26
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of sub
排除标准
- •1. A patient with a history of engrafted hematopoietic stem cell transplantation (HSCT), with successful engraftment.
- •2. A patient who has received gene therapy treatment at any point.
- •3. A patient who is judged by the principal investigator or sub-investigator 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 breathing difficulties during the lumbar puncture process.
- •4. A patient who is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) within 4 months before obtaining informed consent.
- •5. A patient who is unable to comply with the protocol (e.g. is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator.
- •6. A patient who is judged by the principal investigator or sub-investigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including anesthesia or hypersensitivity to any component of JR-141 or idursulfase.
- •7. A patient who has a known or suspected local or general infection or is at risk of abnormal bleeding due to a medical condition* or therapy.
- •8. A patient who otherwise is judged by the principal investigator or sub-investigator to be ineligible to participate in the study.
- •* Medical Conditions:
- •1. Clinically significant multiple or severe drug allergies, intolerance to topical corticosteroids, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear immunoglobulin A [IgA] dermatosis, toxic epidermal necrolysis, and exfoliative dermatitis)
- •2. Evidence or history of significant active bleeding or coagulation disorder or use of non-steroidal anti-inflammatory drugs or other drugs that affect coagulation or platelet function within 14 days prior to lumbar catheter insertion
- •3. Allergy to lidocaine (Xylocaine®) or its derivatives
研究者
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