A randomised, multicentre, double-blind, placebo-controlled, crossover trial determining the efficacy of dry powder mannitol in improving lung function in subjects with Cystic Fibrosis aged six to seventeen years - DPM-CF-204 mannitol in CF aged 6-17 years

Pharmaxis Ltd.0 个研究点目标入组 160 人开始时间: 2013年2月15日最近更新: 2015年12月22日

适应症

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•1. Personally provide, or have a legal guardian provide written informed consent to participate in the trial, according to local regulations;
•2. rhDNase and maintenance antibiotic use is allowed but treatment must have been established at least 3months prior to screening. The subject must remain on rhDNase and / or maintenance antibiotics for the duration of the trial. The subject must not commence treatment with rhDNase or maintenance antibiotics during the trial;
•3. Have a confirmed diagnosis of cystic fibrosis (sweat test result >= 60 mEq/L chloride and/or genotyping showing two identifiable mutations consistent with a diagnosis of cystic fibrosis);
•4. Be aged >= 6 years and < 18 years;
•5. Have a percentage of predicted FEV1 of >= 30% and < 90% at Screening (Visit 0). Percentage of predicted FEV1 will be calculated using Wang for children aged < 8 years, and using NHanes III for those >= 8 years; and
•6. Be able to perform all the techniques necessary to measure lung function.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 160
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•1. Be using maintenance nebulised hypertonic saline Be considered terminally ill”; eligible for lung transplantation, or have received a lung transplant previously;
•2. Require home oxygen or assisted ventilation;
•3. Have had an episode of massive haemoptysis defined as acute bleeding =240 ml in a 24-hour period and/or recurrent bleeding =100 ml/day over several days in the three-months prior to Screening (Visit 0);
•4. Have a known intolerance to mannitol;
•5. Be taking non-selective beta blockers;
•6. In the three months prior to Screening (Visit 0) have had a myocardial infarction; a cerebral vascular accident; major ocular, abdominal, chest or brain surgery;
•7. Have a known cerebral, aortic or abdominal aneurysm;
•8. Be currently participating in, or have participated in another investigative drug trial within four weeks of Screening (Visit 0);
•9. Be pregnant or breastfeeding, or plan to become pregnant whilst in the trial;
•10. For females of childbearing potential (at the discretion of the investigator), be using an unreliable form of contraception;
•11. Have a failed” or incomplete” mannitol tolerance test; or
•12. Have any concomitant medical, psychiatric, or social condition that, in the Investigator’s opinion, would put the subject at significant risk, may confound the results or may significantly interfere with the subject’s participation in the trial.

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