NCT01883531

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
95

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Jun 2013

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2013

Completed
16 days until next milestone

First Submitted

Initial submission to the registry

June 17, 2013

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 21, 2013

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2015

Completed
Last Updated

October 14, 2015

Status Verified

October 1, 2015

Enrollment Period

2.3 years

First QC Date

June 17, 2013

Last Update Submit

October 12, 2015

Conditions

Cystic Fibrosis

Keywords

improving lung functionpaediatric and adolescentFEV1

Outcome Measures

Primary Outcomes (1)

  • Effect on lung function (FEV1)

    To determine the effect of eight weeks of twice-daily treatment with inhaled dry powder mannitol on lung function (FEV1) in subjects with CF who are aged six to seventeen years.

    The absolute change from each treatment period baseline to week 8 of each treatment period in percentage of predicted FEV1.

Secondary Outcomes (4)

  • Effect on FVC

    The absolute change from each treatment period baseline to week 8 of each treatment period in percentage of predicted FVC.

  • Effect of inhaled mannitol on FEF25-75

    The absolute change from each treatment period baseline to week 8 of each treatment period in percentage of predicted FEF25-75.

  • Assess safety

    From each treatment period baseline to week 8 of each treatment period.

  • Sputum weight

    The absolute change from each treatment period baseline to week 8 of each treatment period in sputum weight.

Study Arms (2)

Inhaled Placebo

PLACEBO COMPARATOR

Eight-week treatment period with inhaled placebo b.d.

Drug: Inhaled Placebo

Inhaled Mannitol

ACTIVE COMPARATOR

Eight-week treatment period Inhaled Mannitol 400 mg b.d.

Drug: Inhaled Mannitol

Interventions

Inhaled MannitolDRUG

Active treatment is inhaled mannitol with a particle size of 3-4 microns

Also known as: Mannitol, IDPM, Dry Powder Mannitol for Inhalation, Bronchitol
Inhaled Mannitol
Inhaled PlaceboDRUG

The PLacebo is non respirable mannitol due to the big size particle

Also known as: Control
Inhaled Placebo

Eligibility Criteria

Age6 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Personally provide, or have a legal guardian provide written informed consent to participate in the trial, according to local regulations;
  • 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;
  • Have a confirmed diagnosis of cystic fibrosis (sweat test result greater than or equal to 60 mEq/L chloride and/or genotyping showing two identifiable mutations consistent with a diagnosis of cystic fibrosis);
  • Be aged greater than or equal to 6 years and \< 18 years;
  • Have a percentage of predicted FEV1 of greater than or equal to 30% and less than or equal to 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 greater than or equal to 8 years; and
  • Be able to perform all the techniques necessary to measure lung function.

You may not qualify if:

  • Be using maintenance nebulised hypertonic saline;
  • Be considered "terminally ill"; eligible for lung transplantation, or have received a lung transplant previously;
  • Require home oxygen or assisted ventilation;
  • Have had an episode of massive haemoptysis defined as acute bleeding ≥240 ml in a 24-hour period and/or recurrent bleeding ≥100 ml/day over several days in the three-months prior to Screening (Visit 0);
  • Have a known intolerance to mannitol;
  • Be taking non-selective beta-blockers;
  • 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;
  • Have a known cerebral, aortic or abdominal aneurysm;
  • Be currently participating in, or have participated in another investigative drug trial within four weeks of Screening (Visit 0);
  • Be pregnant or breastfeeding, or plan to become pregnant whilst in the trial;
  • For females of childbearing potential, be using an unreliable form of contraception, (at the discretion of the investigator);
  • 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
  • Have a "failed" or "incomplete" mannitol tolerance test (as described in Section 8.3.1.1).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

John Radcliffe Hospital

Oxford, Oxford, OX3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

MannitolInhalation

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Sugar AlcoholsAlcoholsOrganic ChemicalsCarbohydratesRespiratory MechanicsRespirationRespiratory Physiological PhenomenaCirculatory and Respiratory Physiological Phenomena

Study Officials

  • Christiane De Boeck

    UZ Leuven, Belgium

    PRINCIPAL INVESTIGATOR

  • Jeremy Hull, Dr

    John Radcliffe Hospital, Oxford, UK

    PRINCIPAL INVESTIGATOR

  • Anne Munck, Dr

    Hôpital Robert Debré, France

    PRINCIPAL INVESTIGATOR

  • Joachim Riethmuller, Dr

    Universitats Kinderklinik Tubingen, Germany

    PRINCIPAL INVESTIGATOR

  • Larry Lands, MD

    'Montreal Children's Hospital, Montreal, Canada

    PRINCIPAL INVESTIGATOR

  • Alexander Möller, MD

    University Childrens Hospital Zurich

    PRINCIPAL INVESTIGATOR

  • Sonia Volpi, MD

    Azienda Ospedaliera Universitaria Integrata Verona Italy

    PRINCIPAL INVESTIGATOR

  • Harm Tiddens, MD

    Erasmus MC-Sophia Children's Hospital, Rotterdam, Netherlands

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 17, 2013

First Posted

June 21, 2013

Study Start

June 1, 2013

Primary Completion

October 1, 2015

Study Completion

October 1, 2015

Last Updated

October 14, 2015

Record last verified: 2015-10

Locations

GB(1)