EUCTR2015-001967-38-GB进行中（未招募）1 期
A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD) - SMTC11004 - Phase 1 study in healthy volunteers and DMD patients
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
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. Males of any ethnic origin aged 18 to 55 years inclusive and with body weight of 60 to 85 kg inclusive and a body mass index (BMI) of 18.5 to 30 kg/m2 inclusive.
- •2. In good health, as determined by a medical history, physical examination, 12-lead ECG and clinical laboratory evaluations (congenital non-haemolytic hyperbilirubinaemia, Gilbert’s syndrome, is acceptable).
- •3. Subjects who are non-smokers and do not use other tobacco or nicotine products for at least 60 days preceding screening and are willing and able to refrain from smoking, tobacco, and nicotine products until after completion of the follow up visit.
- •4. Willing and able to give written informed consent to participate in the study and to abide by the study restrictions.
- •1. Male children aged 5 to 9 years inclusive of any ethnic origin with a genetic diagnosis of DMD.
- •2. The subject is willing to give verbal or written age appropriate assent to participate. A parent/legal guardian must date and sign a written consent on behalf of the subject, according to ICH and local regulations. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team. For safety reasons, the subject’s parent/legal guardian must have a good understanding of the English language, in which the consent/assent forms are available, and understand the requirements for reporting of any AE to the Investigator.
- •3. The subject has 3 months or more stable systemic (subjects using an intermittent regimen of steroid are allowed to be enrolled) corticosteroid therapy prior to Screening. Dose modifications for body weight are permitted.
- •4. The subject has a North Star Ambulatory Assessment (NSAA) score; a historical NSAA score maybe provided if performed within 30 days prior to the Screening visit. The subject should have a minimum NSAA score of 17.
- •5. The subject and parent are willing for the subject to adhere to a balanced diet from 1 week prior to dosing until the end of the follow-up period.
- •6. Subjects must agree to not have sexual intercourse during the study treatment phases and until the end of their participation in the study
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 8
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 16
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1. Not willing, or partners not willing, to use appropriate contraception, or not willing to avoid donating sperm from time of first dose until 3 months after final dose
- •2. Received any prescribed systemic or topical medication within 14 days of first dose unless in the opinion of the Investigator it will not interfere with the study procedures or compromise safety
- •3. Used any non-prescribed systemic or topical medication (including herbal remedies) within 7 days of the first dose (with the exception of vitamin/mineral supplements) unless in the opinion of the Investigator it will not interfere with the study procedures or compromise safety
- •4. Received any medications, including St John’s Wort, known to chronically alter drug absorption or elimination processes within 30 days of first dose unless in the opinion of the Investigator it will not interfere with the study procedures or compromise safety
- •5. Still participating or has participated in a clinical study involving administration of an investigational drug in the past 3 months
- •6. Has donated any blood, plasma or platelets in the 3 months prior to screening or who have made donations on more than two occasions within the 12 months preceding first dose
- •7. Significant history of drug allergy as determined by the Investigator, or any clinically significant allergic disease (excluding non-active hay fever) as determined by the Investigator
- •8. Known hypersensitivity to the excipients of the study drug or potential reconstitution vehicle
- •9. Subject is dairy or lactose intolerant, has an allergy to egg or nuts or any other dietary restrictions that might interfere with the study conduct
- •10. Supine blood pressure and/or supine pulse rate higher than 150/90 mmHg and 110 beats per minute (bpm), or lower than 90/40 mmHg and/or 40 bpm, after 5 minutes rest, confirmed by a repeat assessment
- •11. Consumes more than 28 units of alcohol per week or has a significant history of alcoholism or drug/chemical abuse as determined by the Investigator
- •12. Positive urine drug, alcohol or cotinine result at screening or admission to the clinical unit
- •13. History of, any clinically significant neurological, gastrointestinal, renal, hepatic, cardiovascular, psychiatric, respiratory, metabolic, endocrine, haematological or other major disorders as determined by the Investigator
- •14. Clinically significant illness within 4 weeks of start of dose
- •15. Known to have serum hepatitis, or tests positive for hepatitis B surface antigen, hepatitis C antibody or human immunodeficiency virus (HIV) I or II tests at screening
- •16. Abnormality in the 12-lead ECG that, in the opinion of the investigator, increases the risk of participating in the study
- •17. Subjects who, in the opinion of the Investigator, should not participate in the study
- •18. Vegans or medical dietary restrictions
- •19. Cannot communicate reliably with the Investigator
- •20. Unlikely to co-operate with the study requirements
- •1. Enrolment or participation in any therapeutic clinical trial within prior 3 months or 5 times the half-life (whichever is longer)
- •2. Known hype
研究者
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