EUCTR2018-004837-34-DE进行中（未招募）1 期
PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults with Arginase 1 Deficiency
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
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
- All
入选标准
- •1. The subject and/or parent/guardian provides written informed
- •consent/assent, which includes compliance with the requirements and
- •restrictions listed in the informed consent form (ICF) and in this
- •2. A current diagnosis of ARG1-D as documented in medical records,
- •which must include 1 of the following: elevated plasma arginine levels, a
- •mutation analysis that results in s pathogenic variant, or reduced RBC arginase
- •activity. For entry into this study, subjects must also fulfill the following
- •plasma arginine criteria:
- •a. The average of all measured values of plasma arginine during the
- •screening period prior to the randomization visit (Visit 1, Study Day 1) is
- •= 250 µmol/L.
- •b. If a subject is re-screened, the only values that are considered for eligibility assessment are those in the current screening period.
- •3. Subjects must be = 2 years of age on the date of informed consent /
- •assent.4. The subject must be assessable for clinically meaningful withinsubject
- •change (clinical response) on at least 1 component of 1
- •assessment included in the key secondary/other secondary endpoints. To be considered
- •assessable, the subject must be able to complete the assessment, and
- •must have a Baseline deficit in at least 1 component as defined in the
- •5. Have received documented confirmation from the investigator and/or
- •dietician that the subject can maintain their diet in accordance with
- •dietary information presented in the protocol, ie, can maintain the
- •current level of protein consumption, including natural protein and
- •essential amino acid supplementation.
- •6. Subjects receiving ammonia scavenger therapy, anti-epileptic drugs,
- •and/or medications for spasticity (eg, baclofen) must be on a stable
- •dose of the medication for at least 4 weeks prior to randomization and
- •be willing to remain on a stable dose during the double-blind portion and
- •blinded follow-up portions of the study.
- •7. Female and male subjects may participate. Female subjects of childbearing
- •potential must have a negative serum pregnancy test during the
- •screening period before receiving the first dose of study treatment, and
- •a negative urine pregnancy test on the day of the first dose, prior to the
- •first dose. If the subject (male or female) is engaging in sexual activity
- •that could lead to pregnancy, must be surgically sterile, postmenopausal
- •(no menses for 12 months without an alternative medical cause or a high
- •FSH level in the postmenopausal range in women not using hormonal
- •contraception or hormonal replacement therapy), or must agree to use a
- •highly effective method of birth control during the study and for a
- •minimum of 30 days after the last study drug administration. Highly
- •effective methods of contraception include: combined (estrogen and
- •progestogen containing) hormonal contraception associated with
- •inhibition of ovulation; progesterone-only hormonal contraception
- •associated with inhibition of ovulation; intrauterine device (IUD);
- •intrauterine hormone-releasing system (IUS); or abstinence (refraining
- •from heterosexual intercourse during the entire period of risk associated
- •with study treatment).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 30
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- 另有 2 项未显示
排除标准
- •1. Hyperammonemic episode (defined as an event in which a subject has an ammonia level =100 uM with one or more symptoms related to hyperammonemia requiring hospitalization or emergency room management) within the 6 weeks before the first dose of study drug is administered.
- •2. Active infection requiring anti-infective therapy within 3 weeks prior to first dose.
- •3. Known active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C.
- •4. Extreme mobility deficit, defined as either the inability to be assessed on the GFAQ or a score of 1 on the GFAQ.
- •5. Other medical conditions or comorbidities that, in the opinion of the investigator would interfere with study compliance or data interpretation (eg., severe intellectual disability precluding required study assessments).
- •6. Has participated in a previous interventional study with pegzilarginase.
- •7. Has a history of hypersensitivity to polyethylene glycol (PEG), that, in the judgment of the investigator, puts the subject at unacceptable risk for adverse events.
- •8. Subject is being treated with botulinum-toxin containing regimens or
- •plans to initiate such regimens during the double-blind or blinded followup
- •portions of the study or received surgical or botulinum-toxin
- •treatment for spasticity-related complications within the 16 weeks prior
- •to the first dose of study treatment in this study.
- •9. Is currently participating in another therapeutic clinical trial or has
- •received any investigational agent within 30 days (or 5 half-lives
- •whichever is longer) prior to the first dose of study treatment in this
- •10. Previous liver or hematopoietic transplant procedure.
研究者
相似试验
进行中（未招募）
1 期
A research study in patients with Arginase I Deficiency to investigate the safety of a new drug and its ability to lower arginine levelsArginase 1 Deficiency HyperargininemiaMedDRA version: 20.0 Level: PT Classification code 10062695 Term: Arginase deficiency System Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2018-004837-34-FRAeglea Biotherapeutics, Inc.30
进行中（未招募）
1 期
A research study in patients with Arginase I Deficiency to investigate the safety of a new drug and its ability to lower arginine levelsEUCTR2018-004837-34-GBAeglea Biotherapeutics, Inc.40
进行中（未招募）
1 期
A research study in patients with Arginase I Deficiency to investigate the safety of a new drug and its ability to lower arginine levelsArginase 1 DeficiencyHyperargininemiaMedDRA version: 20.0Level: PTClassification code 10062695Term: Arginase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2018-004837-34-ITAeglea Biotherapeutics, Inc40
进行中（未招募）
1 期
A research study in patients with Arginase I Deficiency to investigate the safety of a new drug and its ability to lower arginine levelsEUCTR2018-004837-34-ATAeglea Biotherapeutics, Inc.40
已完成
3 期
Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 DeficiencyArginase I DeficiencyHyperargininemiaNCT03921541Immedica Pharma AB32