Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis – A Safety and Efficacy Study

• Register: EUCTR
• Last refreshed on: 21 August 2017
• Main ID: EUCTR2006-004078-28-GB
• Date of registration: 15/02/2012
• Prospective Registration: No
• Primary sponsor: Pharmaxis UK Limited
• Public title: Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis – A Safety and Efficacy Study
• Scientific title: Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis – A Safety and Efficacy Study
• Date of first enrolment: 14/03/2007
• Target sample size: 250
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2006-004078-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: Double blind: yes Parallel group: yes Cross over: Other: If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: yes Other specify the comparator: "Placebo" = Sub-therapeutic control
• Phase: Human pharmacology (Phase I): Therapeutic exploratory (Phase II): Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV):

Countries of recruitment

United Kingdom

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
3. Be aged >= 6 years
4. Have FEV1 >=30 % and <90% predicted
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, and 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 listed for lung transplantation
3. Have had a lung transplant
4. Be using nebulised hypertonic saline concurrently or in the 2 weeks prior to visit 1
5. Have had a significant episode of haemoptysis (>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 study entry
14. Have a known allergy to mannitol
15. Be using beta blockers
16. Have uncontrolled hypertension – 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 ‘Aridol-MTT test positive’.


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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis
MedDRA version: 14.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: IDPM: 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 type: equal
Concentration number: 40-
Pharmaceutical form of the placebo: Inhalation powder, hard capsule
Route of administration of the placebo: Inhalation use

Primary Outcome(s)

Secondary Objective: • To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF on existing RhDNase treatment compared to control. (key objective)
To assess whether IDPM treatment:
• Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
• Improves quality of life (key objective)
• 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, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
• Reduces hospital and community care costs

Main Objective: To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in all patients with CF compared to control.

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

Secondary ID(s)

DPM-CF-301