The general objective of the study is to estimate the systemic pharmacokinetics of mannitol
after single and multiple dosing of IDPM 400 mg to adult and paediatric cystic fibrosis
patients.
Minimum age: 6 Years.
Maximum age: N/A.
Gender(s): Both.
Inclusion Criteria:
- Have given written informed consent to participate in this study in accordance with
local regulations
- Have a confirmed diagnosis of cystic fibrosis (sweat test and/or genotype)
- Be aged >6 years (6-11 for paediatrics, 12-17 for adolescents and 18 years for
adults)
- Have FEV1 > 30 % and < 90% predicted
Exclusion Criteria:
- Be investigators, site personnel directly affiliated with this study, or their
immediate families. Immediate family is defined as a spouse, parent, child or
sibling, whether biologically or legally adopted.
- Be considered "terminally ill" or listed for lung transplantation
- Have had a lung transplant
- Be using nebulised hypertonic saline
- Have had a significant episode of haemoptysis (> 60 mL) in the three months prior to
enrolment
- Have had a myocardial infarction in the three months prior to enrolment
- Have had a cerebral vascular accident in the three months prior to enrolment
- Have had major ocular surgery in the three months prior to enrolment
- Have had major abdominal, chest or brain surgery in the three months prior to
enrolment
- Have a known cerebral, aortic or abdominal aneurysm
- Be breast feeding or pregnant, or plan to become pregnant while in the study
- Be using an unreliable form of contraception (female patients at risk of pregnancy
only)
- Be participating in another investigative drug study, parallel to, or within 4 weeks
of study entry (except inhaled mannitol)
- Not able to maintain a mannitol free diet from Day -2 until Day 8 of the treatment
phase.
- Have a known allergy to mannitol
- Be using beta blockers
- Have uncontrolled hypertension - systolic blood pressure > 190 and / or diastolic
blood pressure > 100
- 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 patient's participation in the study
- Be MTT positive.
Sheffield Children's Clinical Foundation, Sheffield S102TH, United Kingdom
Southampton General Hospital, Southampton SO166YD, United Kingdom
Mater Adult Hospital, Brisbane, Queensland 4101, Australia
Royal Children's Hospital, Melbourne, Victoria 3052, Australia