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A trial to determine the efficacy of dry powder mannitol in improving lung function in subjects with Cystic Fibrosis aged six to seventeen years


- candidate number16574
- NTR NumberNTR4453
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR27-feb-2014
- Secondary IDsDPM-CF-204 NCT01883531
- Public TitleA trial to determine the efficacy of dry powder mannitol in improving lung function in subjects with Cystic Fibrosis aged six to seventeen years
- Scientific TitleA 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
- ACRONYMDPM-CF-204
- hypothesisIt is hypothesised that inhaled mannitol 400 mg b.d. will lead to a significant improvement in the absolute change in percentage of predicted FEV1 from baseline following eight-weeks of trial treatment compared to treatment with inhaled placebo b.d. Any improvement in FEV1 is considered clinically meaningful; however, this trial has set a threshold of 3% for the purposes of determining an appropriate sample size for statistical power whilst retaining trial feasibility in an orphan disease population.
- Healt Condition(s) or Problem(s) studiedCystic Fibrosis (CF)
- Inclusion criteria1. 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 3 months 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 ¡İ 60 mEq/L chloride and/or genotyping showing two identifiable mutations consistent with a diagnosis of cystic fibrosis); 4. Be aged ¡İ 6 years and < 18 years; 5. Have a percentage of predicted FEV1 of ¡İ 30% and ¡Ü 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 ¡İ 8 years; and 6. Be able to perform all the techniques necessary to measure lung function.
- Exclusion criteria1. Be using maintenance nebulised hypertonic saline; 2. Be considered ¡°terminally ill¡±; eligible for lung transplantation, or have received a lung transplant previously; 3. Require home oxygen or assisted ventilation; 4. Have had an episode of massive haemoptysis defined as acute bleeding ¡İ 240 ml in a 24-hour period and/or recurrent bleeding ¡İ100 ml/day over several days in the three-months prior to Screening (Visit 0); 5. Have a known intolerance to mannitol; 6. Be taking non-selective ¦Â blockers; 7. 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; 8. Have a known cerebral, aortic or abdominal aneurysm; 9. Be currently participating in, or have participated in another investigative drug trial within four weeks of Screening (Visit 0); 10. Be pregnant or breastfeeding, or plan to become pregnant whilst in the trial; 11. For females of childbearing potential, be using an unreliable form of contraception (at the discretion of the investigator); 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; or 13. Have a ¡°failed¡± or ¡°incomplete¡± mannitol tolerance test.
- mec approval receivedyes
- multicenter trialyes
- randomisedyes
- masking/blindingDouble
- controlPlacebo
- groupCrossover
- Type2 or more arms, randomized
- Studytypeintervention
- planned startdate 21-jun-2013
- planned closingdate11-mrt-2015
- Target number of participants160
- InterventionsStudy Drug = Mannitol 400mg/twice a day Placebo = non active Mannitol 400mg/twice a day, that means: the placebo consists of larger particles of Mannitol and therefore it is not inhaled into the lungs The study drug is administered via a dry powder inhaler. • Mannitol 400 mg twice a day for 8 weeks, followed by an 8-week washout period followed by 400 mg placebo /twice a day for 8 weeks or • 400 mg placebo twice a day for 8 weeks, followed by an 8-week washout period, followed by Mannitol 400 mg/twice a day for 8 weeks.
- Primary outcomeThe absolute change from treatment periodbaseline to week 8 of each treatment period in percentage of predicted FEV1.
- Secondary outcomeChange from treatment period baseline in percentage of predicted FVC t Change from treatment period baseline in percentage of predicted FEF25-75 (exploratory endpoint) Adverse events, vital signs and physical examination Treatment induced sputum weight
- TimepointsTrial started 21 Jun 2013 Trial end planned 11 Mar 2015
- Trial web site
- statusopen: patient inclusion
- CONTACT FOR PUBLIC QUERIESDrs. S. Saadatmand
- CONTACT for SCIENTIFIC QUERIESDr. H.A.W.M. Tiddens
- Sponsor/Initiator Pharmaxis Pharmaceuticals Limited
- Funding
(Source(s) of Monetary or Material Support)
Pharmaxis Pharmaceuticals Limited
- Publications
- Brief summary
- Main changes (audit trail)
- RECORD27-feb-2014 - 8-apr-2014


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