Cystic fibrosis: a hereditary inflammatory process
| ISRCTN | ISRCTN03484127 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN03484127 |
| Secondary identifying numbers | NTR91 |
- Submission date
- 12/09/2005
- Registration date
- 12/09/2005
- Last edited
- 17/09/2008
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Mr S W J Terheggen-Lagro
Scientific
Scientific
Universitair Medisch Centrum, locatie AZU
CF-Centrum
Huispostnummer B03.237
P.O.Box 85500
Utrecht
3508 GA
Netherlands
| Phone | +31 (0)30 250 4000 |
|---|---|
| s.terheggen@umcutrecht.nl |
Study information
| Study design | Randomised, double blind, placebo controlled, parallel group trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Scientific title | |
| Study objectives | One out of 3600 new-born children in the Netherlands has cystic fibrosis (CF). It is an autosomal recessive disease and about 70% of the Dutch CF-patients are homozygous for the delta-F508 mutation. Although the genetic mutation is identical in this group of patients, the pulmonary disease is very diverse. Causative factors are environmental and also co-genetic ones. Morbidity is caused by chronic inflammation and infection of the lungs, which leads to irreversible lung damage. Neutrophils play a key role in the inflammatory cascade. It is assumed that parts of the acute inflammatory response of the neutrophil (chemotaxis/IL8 ± adhesion/selectines ± activation/TNFa ± production of e.g. superoxides or myeloperoxidase ±tissue destruction) play an important role in the inflammatory process in CF. There is a higher concentration of mediators (IL-8, sICAM1, sE-Selectin, TNFa) in patients with CF than in other patients with airway infections. The CFTR protein acts not only as a Cl channel but also as a Na/H antiport and influences the intracellular pH. This might affect the functional activity of the neutrophil. Recently, new activation markers (MoPhabs A17 and A27) located on leukocytes were described that may be an early sign of pulmonary inflammation. To be able to predict and intervene in the inflammatory process would improve the prognosis especially in young children before the process of irreversible lung damage. The use of new and powerful inhaled corticosteroid medication enables us to give anti-inflammatory therapy to young children without the systemic side-effects of orally administered steroids. |
| Ethics approval(s) | Ethics approval received from the local medical ethics committee |
| Health condition(s) or problem(s) studied | Cystic fibrosis |
| Intervention | Inhaled HFA-Beclomethasone Diproprionate (Qvar®) 200 mcg twice daily by aerochamber or a placebo (also inhaled by aerochamber). |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Inhaled HFA-Beclomethasone Diproprionate |
| Primary outcome measure | Pulmonary 1. Forced expiratory volume in one second (FEV1), forced vital capacity (FVC), residual volume (RV)/total lung capacity (TLC) % after 3 years 2. Rint measurements |
| Secondary outcome measures | Immunological: 1. Neutrophil markers: MoPhabs A17 and A27, CD11b, CD11a 2. Interleukin-8 (IL-8), soluble intercellular adhesion molecule 1 (sICAM1), sE-Selectin, tumour necrotising factor alpha (TNFa) 3. End tidal carbon monoxide in exhaled breath Microbiological: 1. Respiratory pathogens in culture Serological: 1. Seroconversion to anti-pseudomonal antibodies Clinical: 1. Adverse events 2. Clinical parameters (body weight, height, fat free mass) 3. Number of pulmonary exacerbations 4. Antimicrobial agent use 5. Quality of life questionnaire scores Radiological: 1. Chest radiograph scored by CF chest radiograph scoring systems |
| Overall study start date | 01/01/2002 |
| Completion date | 01/12/2005 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 2 Years |
| Upper age limit | 10 Years |
| Sex | Both |
| Target number of participants | 60 |
| Key inclusion criteria | For 3-years randomised controlled trial: 1. CF diagnosis as confirmed by sweat chloride test and/or genotyping 2. CF-patients 2 - 10 years old 3. Informed consent 4. Capable of using inhaled corticosteroids by aerochamber 5. Compliant to regular therapy |
| Key exclusion criteria | For 3-years randomised controlled trial: 1. CF-patients less than 2 years 2. CF-patients greater than 10 years 3. Use of anti-inflammatory therapy in a period of 2 months before inclusion (orally administered steroids, inhaled corticosteroids and non-steroid anti-inflammatory drugs, non-steroidal anti-inflammatory drugs [NSAIDs]) 4. Disease, other than CF, that affects growth 5. Participation in another study |
| Date of first enrolment | 01/01/2002 |
| Date of final enrolment | 01/12/2005 |
Locations
Countries of recruitment
- Netherlands
Study participating centre
Universitair Medisch Centrum, locatie AZU
Utrecht
3508 GA
Netherlands
3508 GA
Netherlands
Sponsor information
University Medical Centre Utrecht (UMCU) (The Netherlands)
University/education
University/education
P.O. Box 85500
Utrecht
3508 GA
Netherlands
"ROR" |
https://ror.org/04pp8hn57 |
|---|
Funders
Funder type
Government
The Netherlands Organization for Scientific Research (NWO) (The Netherlands)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
