EUCTR2018-000648-25-FR进行中（未招募）1 期
A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 76
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
入选标准
- •1. Aged = 18 years of age at the time of Screening.
- •2. Documented diagnosis of PKU.
- •3. Documented failure to maintain control of plasma Phe using MNT in the judgement of the investigator.
- •4. Documented non-response to treatment with sapropterin (Kuvan) prior to Screening, defined as:
- •a. Lack of clinical response, or
- •b. Intolerance to treatment, or
- •c. Negative result from sapropterin (Kuvan) response test, or
- •d. Genetic determination of non-response to sapropterin with two null mutations of the phenylalanine hydroxylase (PAH) gene.
- •5. Average plasma Phe concentration of > 600 µmol/L over Screening Visits 1 and 2.
- •6. Any plasma Phe concentration of > 600 µmol/L within the 6 months prior to Screening.
- •7. Has identified a competent person or persons = 18 years of age who can observe the subject during study drug administration and for a minimum of 1 hour following administration for at least the first 6 months of self-administration.
- •8. According to the investigator, subject has identified a person who knows the subject well and who will be able to complete the observer-reported patient reported outcomes (PROs) in this study.
- •9. For females of childbearing potential, must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. (Females are considered not to have childbearing potential if they have been in menopause for at least 2 years, have had a tubal ligation at least 1 year prior to Screening, or have had a total hysterectomy).
- •10. If sexually active, female subjects must be willing to use one highly effective method and one acceptable method of contraception while participating in the study and 4 weeks after completion of the study:
- •a. Highly effective methods of contraception may include: (1) primary forms: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal) or progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable); (2) secondary forms: include intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion.
- •b. Acceptable (but not highly effective) methods of contraception may include: progestogen-only oral hormonal contraception*, where inhibition of ovulation is not the primary mode of action; male or female condom with or without spermicide; cap, diaphragm or sponge with spermicide.
- •c. Females who have been in menopause for at least 2 years, have had a tubal ligation at least 1 year prior to Screening, have a vasectomised partner (with medical assessment of surgical success), have had a total hysterectomy or are sexually abstinent do not need to use any other forms of contraception during the study.
- •*Note that the use of two methods of hormonal contraception is not advised.
- •11. If applicable, has maintained a stable dose of medication for attention deficit hyperactivity disorder (ADHD), depression, anxiety, or other psychiatric disorder for = 8 weeks prior to enrolment and is willing to maintain a stable dose throughout the study unless a change is medically indicated.
- •12. Is willing and
排除标准
- •1. Use of any medication that is intended to treat PKU including the use of sapropterin (Kuvan), or large neutral amino acids, within 14 days prior to administration of study drug (Day 1; first dose of pegvaliase).
- •2. Not willing or unlikely to be able to perform independent self-administration (or to allow carer administration) or not have the ability to recognise the signs and symptoms of acute systemic hypersensitivity reactions (ASHRs).
- •3. Not willing or unlikely to be able to use adrenaline injection device (auto injector or prefilled syringe) and have them readily available at all times throughout pegvaliase treatment.
- •4. Not willing or unlikely to be able to maintain their diet in accordance with dietary information presented in the protocol.
- •5. Based on the clinical judgement of the investigator, does not have the neurocognitive and linguistic capacities to comprehend and answer the patient-reported outcome questionnaires.
- •6. Not willing or unlikely to be able to complete the study diary.
- •7. Use or planned use of any injectable drugs containing PEG (other than pegvaliase), including medroxyprogesterone injection, within 3 months prior to Screening (or within five elimination half-lives, whichever is longer) and during study participation.
- •8. Concurrent disease or condition that would interfere with study participation or safety.
- •9. Alanine aminotransferase (ALT) concentration = 2 times the upper limit of normal (ULN).
- •10. Subjects with known renal pathology, persistent albuminuria (elevated urine albumin creatinine ratio above the ULN measured on first morning void, confirmed on a repeat measurement), or creatinine > 1.5 times the ULN.
- •11. Pregnant or breastfeeding at Screening or planning to become pregnant (subject or partner of subject) or breastfeed at any time during the study.
- •12. Use of any investigational product or investigational medical device within 30 days prior to Screening or requirement for any investigational agent prior to completion of all scheduled study assessments.
- •13. Previous exposure to pegvaliase.
- •14. Any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or terminating early from study.
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