No registrations found.
ID
Source
Brief title
Health condition
cystic fibrosis, mucoviscidosis, taaislijm ziekte
Sponsors and support
Intervention
Outcome measures
Primary outcome
The absolute change from treatment periodbaseline to week 8 of each treatment period in percentage of predicted FEV1.
Secondary outcome
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
Study objective
It is hypothesised that inhaled mannitol 400 mg b.d. will lead to a significant improvement in the absolute change in percentage of predicted FEV1 from baseline following eight-weeks of trial treatment compared to treatment with inhaled placebo b.d.
Any improvement in FEV1 is considered clinically meaningful; however, this trial has set a threshold of 3% for the purposes of determining an appropriate sample size for statistical power whilst retaining trial feasibility in an orphan disease population.
Study design
Trial started 21 Jun 2013
Trial end planned 11 Mar 2015
Intervention
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
The study drug is administered via a dry powder inhaler.
• Mannitol 400 mg twice a day for 8 weeks, followed by an 8-week washout period followed by 400 mg placebo /twice a day for 8 weeks
or
• 400 mg placebo twice a day for 8 weeks, followed by an 8-week washout period, followed by Mannitol 400 mg/twice a day for 8 weeks.
S. Saadatmand
Rotterdam 3075 EA
The Netherlands
+31 (0)10 7041223
s.saadatmand@erasmusmc.nl
S. Saadatmand
Rotterdam 3075 EA
The Netherlands
+31 (0)10 7041223
s.saadatmand@erasmusmc.nl
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
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
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL4215 |
| NTR-old | NTR4453 |
| Other | NCT01883531 : DPM-CF-204 |