Long Term Administration of Inahled Mannitol in Cystic Fibrosis - A Safety and Efficacy Study

• Register: EUCTR
• Last refreshed on: 3 September 2012
• Main ID: EUCTR2008-002740-42-DE
• Date of registration: 31/07/2008
• Prospective Registration: Yes
• Primary sponsor: Pharmaxis Pharmaceuticals Limited
• Public title: Long Term Administration of Inahled Mannitol in Cystic Fibrosis - A Safety and Efficacy Study
• Scientific title: Long Term Administration of Inahled Mannitol in Cystic Fibrosis - A Safety and Efficacy Study
• Date of first enrolment: 13/11/2008
• Target sample size: 300
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-002740-42
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Sub-therapeutic control

Countries of recruitment

Belgium, France, Germany, Netherlands

Key inclusion & exclusion criteria

Inclusion criteria:
Subjects may be included in the study if all of the following criteria are met. The subject must:
1. Have given written informed consent to participate in this study 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 > 6 years old. 4. Have FEV1 >40 % and < 90% predicted (using Wang32 <8 years and NHanes III33 >8years). 5. 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:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Subjects are excluded from participating in this study if one or more of the following criteria are met. The subject must NOT:
1. 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. 2. Be considered “terminally ill” or eligible for lung transplantation. 3. Have had a lung transplant. 4. Be using nebulized hypertonic saline in the 4 weeks prior to visit 1. 5. Have had a significant episode of hemoptysis (>60 mL) in the three months prior to enrolment. 6. Have had a myocardial infarction in the three months prior to enrolment. 7. Have had a cerebral vascular accident in the three months prior to enrolment. 8. Have had major ocular surgery in the three months prior to enrolment. 9. Have had major abdominal, chest or brain surgery in the three months prior to enrolment. 10. Have a known cerebral, aortic or abdominal aneurysm. 11. Be breast feeding or pregnant, or plan to become pregnant while in the study. 12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only). 13. Be participating in another investigative drug study, parallel to, or within 4 weeks of visit 0. 14. Have a known allergy to mannitol.
15. Be using beta blockers. 16. Have uncontrolled hypertension –e.g. for adults: 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 patient’s participation in the study. 18. Be ‘MTT positive or incomplete’. (As evaluated in section 3.3.8.5).



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic Fibrosis
MedDRA version: 9.1 Level: LLT Classification code 10011762 Term: Cystic fibrosis

Intervention(s)

Product Name: Inhaled Dry Powder Mannitol
Product Code: IDPM
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: Mannitol
CAS Number: 69658
Concentration unit: mg milligram(s)
Concentration number: 40-

Product Name: Inhaled dry powder mannitol
Product Code: IDPM
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: Mannitol
CAS Number: 69658
Concentration unit: mg milligram(s)
Concentration number: 5-

Product Name: Inhaled dry powder mannitol
Product Code: MTT
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: Mannitol
CAS Number: 69658
Concentration unit: mg milligram(s)
Concentration number: 40-

Primary Outcome(s)

Main Objective: To determine whether inhaled mannitol compared to control improves FEV1 in patients with Cystic Fibrosis.

Secondary Objective: To determine whether inhaled mannitol compared to control improves FEV1 in patients with CF on existing RhDNase treatment.
To assess whether inhaled mannitol treatment:
•Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort
•Improves quality of life
•Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
•Reduces days in hospital due to pulmonary exacerbations
•Improves other measures of lung function
•Demonstrates an appropriate safety profile (adverse events, hematology, biochemistry, sputum microbiology, physical examination)
•Reduces hospital and community care costs

Primary end point(s): Change in absolute FEV1 (forced expiratory volume in 1 second)

Secondary ID(s)

DPM-CF-302