A Multi-centre, Multiple-dose, Open-label Study to Investigate the Safety, Tolerability, Pharmacodynamics, and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase (EE-TP) in Patients with Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)

St George’s University of London0 个研究点目标入组 20 人开始时间: 2018年11月9日最近更新: 2019年6月30日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: St George’s University of London
入组人数: 20

研究概览入排标准研究者记录与标识符相似试验

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. Patients must be male or female, of any race, aged 18 years or
•older at Screening.
•Having reviewed the benefit-risk profile and with appropriate approval from ECs and/or regulatory agencies, the age range may be extended to include patients:
•- aged 16 years or older after at least 12 patient-months of exposure in patients aged 18 years or over.
•- aged 12 years or older after at least 12 patient-months of exposure in patients aged <18 years at the time of enrolment.
•2. Patients must be diagnosed with MNGIE by demonstrating ALL of the following:
•<18% normal TP activity in the buffy coat;
•>3 µmol/L plasma thymidine;
•>5 µmol/L plasma deoxyuridine;
•confirmation of the presence of a pathogenic mutation in TYMP gene by sequencing.
•3. Patients must be able to undergo study procedures. Patients must agree to either remain completely true abstinent (because of the patient’s lifestyle choice; the patient should not become abstinent just for the purpose of study participation) or to use two effective contraceptive methods from Screening until completion of the Follow-up visit:
•Male patients with partners of childbearing potential must use a male barrier method of contraception (i.e., male condom with spermicide) in addition to a second method of acceptable contraception used by their female partners (refer to Section 4.6.6).
•Female patients of childbearing potential must be willing to use a highly effective method of birth control (i.e., contraceptive measures with a failure rate of <1% per year):
•- IUD (e.g., Mirena) which was implanted at least 2 months prior to Screening. Steel or copper IUDs are not acceptable.
•- Established use of oral, implanted, transdermal, or hormonal method of contraception associated with inhibition of ovulation.
•- Male sterilisation, with verbal confirmation of surgical success (for female patients on the study, the vasectomised male partner should be the sole partner for that patient).
•- Bilateral tubal ligation.
•5. Patients must be willing to sign and date the written ICF after the benefits and risks of taking part in this study have been explained to them, and to comply with the study restrictions.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 12
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

排除标准

•1. Patients who have received a successful liver or bone marrow transplant.
•2. Patients with a known history of human immunodeficiency virus (HIV), hepatitis B infection, or an active hepatitis C infection.
•3. Patients who are severely disabled (e.g., patient bed-bound, incontinent, and unable to carry out any daily activities), or with a life expectancy of less than 12 months at Screening, based on the Investigator’s judgment.
•4. Female patients who are:
•pregnant, planning a pregnancy, or are unwilling to use contraception
•breastfeeding or lactating.
•5. Patients who have donated blood in the 90 days prior to Screening.
•6. Patients with a confirmed RBC count of <3.0 × 109 per mL.
•7. Patients who have a significant history of alcoholism or drug/chemical abuse within 1 year prior to Screening, as determined by the Investigator.
•8. Patients who have an abnormality in heart rate, blood pressure, or body temperature at Screening that, in the opinion of the Investigator, increases the risk of participating in the study.
•9. Patients who have an abnormality in the 12-lead electrocardiogram (ECG) at Screening that, in the opinion of the Investigator, increases the risk of participating in the study.
•10. Patients who have, or have a history of, any clinically significant neurological, gastrointestinal, renal, hepatic, cardiovascular, psychiatric, respiratory, metabolic, endocrine, haematological, or other major disorder (except for MNGIE, or disorders associated with MNGIE that, in the Investigator’s opinion, do not constitute a risk when taking study medication and would not interfere with the study objectives) as determined by the Investigator.
•11. Patients with any current malignancy, or a history of malignancy within 5 years prior to Screening, with the exception of adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.
•12. Patients who are currently enrolled in, or are planning to participate in, or discontinued within the last 30 days from a clinical study involving an investigational medicinal product (IMP) or concurrently enrolled in medical research judged not to be scientifically or medically compatible with EE-TP.
•13. Patients with any medical condition, which in the opinion of the Investigator, would make the patient unsuitable for enrolment or could interfere with the patient’s participation in, or completion of, the study

研究者

发起方

St George’s University of London

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