A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects

Launched by SYNTARA · May 7, 2014

Keywords

ClinConnect Summary

This is a double-blind, randomized, parallel arm, controlled, multicenter, and interventional clinical trial. Potential subjects will sign the informed consent form (ICF) and be assessed for eligibility. After satisfying all inclusion \& exclusion criteria, subjects will be given a mannitol tolerance test (MTT). Those subjects that pass the MTT will be randomized to receive inhaled mannitol (400 mg b.i.d.) or control b.i.d. for a period of 26-weeks.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Have given written informed consent to participate in this trial in accordance with local regulations;
• • 2. Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value ≥ 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype);
• • 3. Be aged at least 18 years old;
• • 4. Have FEV1 \> 40 % and \< 90% predicted (using NHanes III \[1\]);
• • 5. Be able to perform all the techniques necessary to measure lung function;
• • 6. Be adherent with maintenance therapies (antibiotics and or rhDNase), if used, for at least 80% of the time in the two weeks prior to visit 1 and
• • 7. If rhDNase and/or maintenance antibiotic are being used treatment must have been established at least 1 month prior to screening (Visit 0). The subject should remain on the rhDNase and / or maintenance antibiotics for the duration of the trial. The subject should not commence treatment with rhDNase or maintenance antibiotics during the trial
• Exclusion Criteria:
• • 1. Be investigators, site personnel directly affiliated with this trial, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted;
• • 2. Be considered "terminally ill" or eligible for lung transplantation;
• • 3. Have had a lung transplant;
• • 4. Be using maintenance nebulized hypertonic saline in the 2 weeks prior to visit 1;
• • 5. Have had a significant episode of hemoptysis (\> 60 mL) in the three months prior to Visit 0;
• • 6. Have had a myocardial infarction in the three months prior to Visit 0;
• • 7. Have had a cerebral vascular accident in the three months prior to Visit 0;
• • 8. Have had major ocular surgery in the three months prior to Visit 0;
• • 9. Have had major abdominal, chest or brain surgery in the three months prior to Visit 0;
• • 10. Have a known cerebral, aortic or abdominal aneurysm;
• • 11. Be breast feeding or pregnant, or plan to become pregnant while in the trial;
• • 12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only);
• • 13. Be participating in another investigative drug trial, parallel to, or within 4 weeks of screening (Visit 0);
• • 14. Have a known allergy to mannitol;
• • 15. Be using non-selective oral beta blockers;
• • 16. Have uncontrolled hypertension -i.e. systolic BP \> 190 and / or diastolic BP \> 100;
• • 17. Have a condition or be in a situation which in the Investigator's opinion may put the subject at significant risk, may confound results or may interfere significantly with the subject's participation in the trial;or
• • 18. Have a failed or incomplete MTT at trial entry (as evaluated in Section 8.1.1.1).
• • 19. The subject must not commence treatment with rhDNase or maintenance antibiotics during the trial.