EUCTR2022-000422-16-HU进行中（未招募）1 期
A randomised, double-blind, placebo-controlled, two-part study to evaluate the pharmacokinetics, safety and tolerability, and preliminary efficacy of two dose levels of golexanolone in subjects with primary biliary cholangitis, fatigue, and cognitive dysfunction.
mecrine Cognition AB0 个研究点目标入组 101 人开始时间: 2022年5月24日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 101
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. Male and female subjects age = 18 and =75 years.
- •2. Diagnosis of PBC based on the presence of =2 of the 3 key disease characteristics:
- •a. Anti-mitochondrial antibody or PBC-specific anti-nuclear antibody titre =1/40
- •b. Elevated alkaline phosphatase (ALP) (> upper limit of normal [ULN] for the relevant laboratory)
- •c. Compatible or diagnostic liver biopsy
- •3. Clinically significant fatigue defined for the purposes of this study as a PBC-40 fatigue domain score of =29 at screening.
- •4. Clinically significant cognitive symptoms, defined for the purposes of this study as a PBC-40 cognitive domain =16 at screening.
- •5. Stable PBC SoC therapy, which may include UDCA, OCA, bezafibrate and/or fenofibrate. for at least 3 months prior to randomisation.
- •6. For all women of childbearing potential (WOCBP) a negative pregnancy test at screening and a negative urine dip-stick pregnancy test at baseline, prior to first dose of IMP will be required.
- •7. WOCBP must be willing to use a contraceptive method with a failure rate of < 1% (intrauterine device [IUD], intrauterine hormone-releasing system [IUS], bilateral tubal occlusion, vasectomised partner, sexual abstinence), and agree to continue use of this method for the duration of the study and thereafter for 1 month after the last dosing of the IMP. Note that IUS is the only hormonal contraceptive method allowed due to possible interactions with the IMP.
- •8. Females of non-childbearing potential must have documented tubal ligation or hysterectomy; or be post-menopausal (defined as 12 months of amenorrhoea [in questionable cases a blood sample with simultaneous follicle stimulating hormone (FSH) 25-140 IE/L and oestradiol <200 pmol/L is confirmatory]).
- •9. Fertile male subjects must be willing to use condom and assure that their female partner will use contraceptive methods with a failure rate of < 1%1 to prevent pregnancy and drug exposure of a fertile female partner and refrain from donating sperm from the date of dosing until 1 month after dosing of the IMP. Men who are surgically sterile may be included without they/their partner fulfilling the above criteria on birth control.
- •10. Willing and able to give informed consent.
- •11. The subject should be judged by the Investigator to be lucid and oriented to person, place, time, and situation when giving the informed consent.
- •Are the trial subjects under 18? no
- •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 81
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 20
排除标准
- •1. Child-Pugh class B or C cirrhosis.
- •2. Clinical evidence of hepatic decompensation (e.g. current or prior HE, ascites, or variceal bleeding).
- •3. History of hepatocellular carcinoma.
- •4. Bilirubin >1.5 x ULN.
- •5. Glomerular filtration rate (GFR) <35 ml/min/1.73m2 estimated using the CKD-EPI equation.
- •6. Haemoglobin (HB) <110 g/L, i.e. subjects with moderate/severe anaemia.
- •7. S-B12 < 125 pmol/L and/or P-folate < 10 nmol/L.
- •8. Evidence of biliary obstruction.
- •9. Any positive result on screening for human immunodeficiency virus (HIV), hepatitis B (serum hepatitis B surface antigen positive), hepatitis C (e.g. HCV RNA positive).
- •10. Prolonged QTcF (>500 ms), cardiac arrhythmia, or any clinically significant abnormality in the resting ECG, as judged by the Investigator (at screening).
- •11. Concomitant disease characterised by chronic fatigue and/or cognitive impairment (e.g. Alzheimer’s, Parkinson’s, etc.) which in the judgement of the Investigator would either limit the potential benefit to the subject and/or confound the interpretation of results.
- •12. Clinically significant bowel disease, including obstruction, inflammatory bowel disease, or malabsorption.
- •13. Clinically significant sleep apnoea, as defined by >5 episodes/hour more than 70% of occasions, despite use of continuous positive airway pressure (CPAP).
- •14. An uncontrolled thyroid disorder:
- •a. Uncontrolled hyperthyroidism: defined as any history of hyperthyroidism that has either not been treated with either radioactive iodine and/or surgery or that has been treated with radioactive iodine and/or surgery but has required ongoing continuous or intermittent use of thyroid hormone synthesis inhibitors (i.e. methimazole or propylthiouracil) in the 24 weeks before screening.
- •b. Uncontrolled hypothyroidism: defined as initiation of thyroid hormone replacement therapy or dose adjustment of replacement therapy in the 12 weeks before screening.
- •c. Subjects without a diagnosed thyroid disease should be excluded if thyroid-stimulating hormone (TSH)-value is above ULN, or/and if free triiodothyronine (FT3)- or free thyroxine (FT4)-values are outside normal limits.
- •15. Subjects with a history of or currently active immune disorders other that PBC (including autoimmune disease) and/or diseases requiring immunosuppressive drugs (including azathioprine, prednisone, prednisolone, budesonide, cyclosporine, tacrolimus, methotrexate, or mycophenolate mofetil).
- •16. Clinical diagnosis of autoimmune hepatitis overlap defined using the Paris overlap criteria (see Appendix 13.2) [30, 31].
- •17. Concurrent liver disease of another aetiology.
- •18. The presence, as judged by the Investigator, of clinically significant concomitant illness which would jeopardise safe participation in the study and /or the interpretation of study findings, e.g. major psychiatric disorder and major depressive disorder (HADS-D >10) formally diagnosed by a psychiatrist.
- •19. Regular use of prescribed or over the counter medications known to cause fatigue or cognitive dysfunction (including, but not limited to, benzodiazepines, opioids other than codeine phosphate, sleeping pills, regular (daily) antihistamine use in the last 4 weeks, anti-psychotic agents, barbiturates, or recreational drug use).
- •Confidential 50 (94)
- •20. Use of prohibited medications within 14 days prior to randomisation, including medications sensitive to CYP3A4 and/or Pgp inhibition/induction with a narrow therapeutic window, including use of warfarin and warfarin
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