An extension of a clinical study to investigate long term safety of tobramycin inhalation powder (TIP) in patients with Cystic Fibrosis

• Register: EUCTR
• Last refreshed on: 23 February 2015
• Main ID: EUCTR2012-003532-23-HU
• Date of registration: 31/10/2012
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: An extension of a clinical study to investigate long term safety of tobramycin inhalation powder (TIP) in patients with Cystic Fibrosis
• Scientific title: A 48 week extension to CTBM100C2401, a single arm open-label, multicenter, phase IV trial, to assess long term safety of tobramycin inhalation powder (TIP) in patients with Cystic Fibrosis
• Date of first enrolment: 19/12/2012
• Target sample size: 120
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-003532-23
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Argentina, Australia, Canada, France, Germany, Hungary, Italy, Mexico, Spain, United States

Contacts

• Name: Public Information Desk
• Affiliation: Novartis Hungária Kft., Pharma

• Name: Public Information Desk
• Affiliation: Novartis Hungária Kft., Pharma

Key inclusion & exclusion criteria

Inclusion criteria:
1. Provide written informed consent, HIPAA (Health Insurance Portability and Accountability Act) authorization (where
applicable), and assent (as appropriate) prior to the performance of any study-related procedure.

2. Completed the core study CTBM100C2401 and able to comply with all protocol requirements of the extension study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 25
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 93
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion criteria:
1. Serum creatinine 2mg/dl or more, BUN 40mg/dl or more, or an abnormal urinalysis defined as 2+ or greater proteinuria at entry
into the extension study (visit 15).

2. Use of loop diuretics within 7 days prior to entry into the extension
study.

3. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the
termination of gestation, confirmed by a positive HCG laboratory test (> 5 mIU/mL) at Visit 15.

4. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are
using a highly effective method of contraception during dosing of
study treatment as defined in full protocol.


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

Health Condition(s) or Problem(s) studied

Chronic lung infection with Pseudomonas aeruginosa in cystic fibrosis patients
MedDRA version: 14.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: LLT Classification code 10021860 Term: Infection pseudomonas aeruginosa System Organ Class: 100000004862

Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]

Intervention(s)

Trade Name: TOBI Podhaler
Product Name: TOBI Podhaler
Product Code: TBM100C
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: Tobramycin
CAS Number: 32986-56-4
Current Sponsor code: TBM100
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 28-

Primary Outcome(s)

Secondary Objective: To evaluate endpoints of interest across 12 cycles (6 treatment cycles in the core study and 6 treatment cycles in the extension study) of TIP treatment:

• change in FEV1 % predicted (relative and absolute)
• absolute change in P. aeruginosa CFU per gram of sputum
• change of P. aeruginosa tobramycin MIC
• time to first and the rate of the usage (overall, oral, intravenous) of antipseudomonal antibiotic (other than those regularly scheduled as prophylactic
treatment)
• time to first and the rate of hospitalization due to serious respiratory-related AEs
• safety profile of TIP in terms of clinical laboratory results and audiology (in a sub-set of patients)
• safety profile of TIP in terms of acute change in FEV1 from pre-dose to 30
minutes post dose
• To evaluate the above endpoints of interest (including the primary endpoint of incidence of AEs) across the 6 cycles of treatment in this extension study.

Timepoint(s) of evaluation of this end point: Across 12 cycles of treatment (core study and extension) and within the extension alone

Main Objective: To assess the safety of Tobramycin Inhalation Powder (TIP) across 12 treatment
cycles (6 treatment cycles in the core study and 6 treatment cycles in the extension
study) in terms of the incidence of treatment emergent adverse events (AEs).

Primary end point(s): Incidence of treatment-emergent adverse event

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1) Visits 15 (baseline of extension), 16, 18, 20, 22, 24, 26, 27

2) Visits 15 (baseline of extension), 16, 18, 20, 22, 24, 26, 27

3) Visits 15 (baseline of extension), 16, 18, 20, 22, 24, 26, 27

4) across 12 cycles of treatment (core study and extension) and within the extension alone

5) across 12 cycles of treatment (core study and extension) and within the extension alone

6) Visits 16, 18, 20, 22, 24, 26

7) Evaluation of clinical laboratory results and (in selected study sites) audiology: across 12 cycles of treatment (core study and extension) and within the extension alone

Secondary end point(s): 1) Relative change in FEV1% predicted, FVC % predicted and FEF25-75 % predicted from baseline

2) Relative change in P aeruginosa CFU in sputum from baseline

3) Change in P aeruginosa tobramycin MIC from baseline

4) Rate of and time to the first hospitalization due to serious respiratory-related AE

5) Rate of and time to the first use of anti-pseudomonal antibiotics (overall, oral, intravenous)

6) Acute change in FEV1% predicted from pre-dose to post-dose 30 minutes

7) Evaluation of clinical laboratory results and (in selected study sites) audiology

Secondary ID(s)

CTBM100C2401E1

Source(s) of Monetary Support

Novartis Pharma Services AG