An exploratory, randomized, double-blind, placebo controlled study to assess the efficacy of multiple doses of omalizumab in cystic fibrosis complicated with allergic bronchopulmonary aspergillosis

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2007-006648-23-BE
• Date of registration: 28/08/2008
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: An exploratory, randomized, double-blind, placebo controlled study to assess the efficacy of multiple doses of omalizumab in cystic fibrosis complicated with allergic bronchopulmonary aspergillosis
• Scientific title: An exploratory, randomized, double-blind, placebo controlled study to assess the efficacy of multiple doses of omalizumab in cystic fibrosis complicated with allergic bronchopulmonary aspergillosis
• Date of first enrolment: 30/09/2008
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-006648-23
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: yes Other trial design description: There is an optional6 mo open label treatment period, following the first 6 months of blinded t'ment If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Belgium, Germany, Ireland, Italy, Netherlands, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
1. Males/females age more than or equal to 12 years
2. Cystic Fibrosis diagnosed by either gene profiling and/or sweat test
3. ABPA previously diagnosed according to the Cystic Fibrosis Foundation Consensus Conference recommendations – minimal diagnostic criteria for diagnosis of ABPA in cystic fibrosis [Stevens, et al 2003]
4. Total serum IgE level of >500 IU/ml
5. Patients who are being treated for ABPA by any oral corticosteroid (OCS) (for at least 8 weeks prior to the first dose) with an OCS entry dose of minimum 5mg/maximum 40mg per day (in prednisolone equivalence) and a history of at least one unsuccessful attempt to taper steroids, defined as in the clinician’s judgment, an ABPA exacerbation during taper, OR patients with a new or recurrent acute ABPA flare, max OCS dose of 20mg.
6. Patients with an FEV1 no lower than 90% of their previous best FEV1 at Screening Visit
7. FEV1 >40% (>30% is acceptable = 16 years old) of predicted (after 12 hour washout of LABA \ 6 hours of SABA)

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:
1. History of cancer in the past 10 years (except surgically-cured basal cell or squamous cell skin cancer).
2. Any previous history of anaphylaxis.
3. Any other medical condition that in the opinion of the investigator may cause the patient to be unsuitable for completion of the study or place the patient at potential risk from being in the study.
4. Pregnant & lactating women.
5. Prior Xolair exposure.
6. Lung or other transplant.



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

Health Condition(s) or Problem(s) studied

Patients with cystic fibrosis complicated by allergic bronchopulmonary aspergillosis.
MedDRA version: 9.1 Level: LLT Classification code 10011763 Term: Cystic fibrosis lung
MedDRA version: 9.1 Level: LLT Classification code 10000244 Term: ABPA

Intervention(s)

Trade Name: Xolair
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: omalizumab
Current Sponsor code: IGE025
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-mg
Pharmaceutical form of the placebo: Powder for solution for injection
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Main Objective: To assess the efficacy of omalizumab (Xolair) in adolescent and adult patients with cystic fibrosis (CF) complicated by acute or chronic allergic bronchopulmonary aspergillosis (ABPA)
•as measured by the proportion of patients requiring rescue with corticosteroids following 6 months of study treatment
•as measured by time to deviation from the protocol prescribed steroid tapering regime

Primary end point(s): To assess the efficacy of Xolair in adolescent and adult patients with cystic fibrosis (CF) complicated by acute or chronic allergic bronchopulmonary aspergillosis (ABPA)
• as measured by the proportion of patients requiring rescue with corticosteroids following 6 months of study treatment
• as measured by time to deviation from the protocol prescribed steroid tapering regime

Secondary Objective: To assess:
1. ABPA exacerbation rates during the treatment periods
2. Changes in FEV1 from baseline, measured after 3 and 6 months of treatment, in particular changes between FEV1 measured pre and post first dose in both treatment periods.
3. Proportion of patients responding to Xolair treatment, where a responder is defined by a reduction in systemic corticosteroid dose of 50% or more compared to baseline.
4. To measure time to steroid free state.
To assess:
5. Change from baseline in average steroid dose.
6. Proportion of patients in each treatment group whose steroid dose has reduced to 5mg following 6 months of treatment.
To measure:
7. Number of steps to reduce steroid dose to zero (or 5mg or less) following 6 months of treatment
8. Immunogenicity
9. PK/PD
Safety Objective:To explore the safety and tolerability of higher doses of omalizumab in this patient population

Secondary ID(s)

CIGE025A2437

2007-006648-23-DE