cystic fibrosis, mucoviscidosis, taaislijm ziekte
Conditions
Interventions
Study 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
Sponsors
Pharmaxis Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 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 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 ¡Ý 60 mEq/L chloride and/or genotyping showing two identifiable mutations consistent with a diagnosis of cystic fibrosis); 4. Be aged ¡Ý 6 years and < 18 years; 5. Have a percentage of predicted FEV1 of ¡Ý 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 ¡Ý 8 years; and 6. Be able to perform all the techniques necessary to measure lung function.
Exclusion criteria
Exclusion criteria: 1. 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 ¡Ý 240 ml in a 24-hour period and/or recurrent bleeding ¡Ý100 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The absolute change from treatment periodbaseline to week 8 of each treatment period in percentage of predicted FEV1. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change 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 | — |
Contacts
Public ContactS. Saadatmand
Erasmus MC, Daniel den Hoed Clinic, Faculty of Medicine, Department of Surgical Oncology, Groene Hilledijk 301
Outcome results
None listed