NCT01082367

Brief Summary

This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.

Trial Health

98
On Track

Trial Health Score

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

Enrollment
50

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Apr 2010

Longer than P75 for phase_3

Geographic Reach
10 countries

19 active sites

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

First Submitted

Initial submission to the registry

March 5, 2010

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 8, 2010

Completed
24 days until next milestone

Study Start

First participant enrolled

April 1, 2010

Completed
5.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2015

Completed
11 months until next milestone

Results Posted

Study results publicly available

April 27, 2016

Completed
Last Updated

July 15, 2016

Status Verified

June 1, 2016

Enrollment Period

5.2 years

First QC Date

March 5, 2010

Results QC Date

December 22, 2015

Last Update Submit

June 6, 2016

Conditions

Keywords

Tobramycin Inhalation solutionCystic fibrosisLung diseaseanti-bacterial agents

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle

    Sputum/throat swab cultures were assessed.

    Day 29

Secondary Outcomes (2)

  • Percentage of Participants Free From P. Aeruginosa 28 Days After Termination of the Second Treatment Cycle

    Day 91

  • Percentage of Participants P Aeruginosa-free at Termination of the Double Blind Period

    Day 91

Study Arms (2)

TOBI (tobramycin inhaled solution)/Placebo

EXPERIMENTAL

Participants randomized to TOBI received the investigational treatment for 28 days twice daily (bi)d in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the open label (OL) phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received placebo for 28 days bid (second treatment cycle).

Drug: TOBIDrug: Placebo

Placebo/TOBI

PLACEBO COMPARATOR

Participants randomized to placebo group received 0.9 % saline (NaCl) for 28 days bid in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the OL phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received TOBI for 28 days bid (second treatment cycle).

Drug: TOBIDrug: Placebo

Interventions

TOBIDRUG

TOBI (tobramycin inhaled solution)

Placebo/TOBITOBI (tobramycin inhaled solution)/Placebo

Placebo

Placebo/TOBITOBI (tobramycin inhaled solution)/Placebo

Eligibility Criteria

Age3 Months - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Diagnosis of cystic fibrosis
  • Early lower respiratory tract infection with P. aeruginosa,

You may not qualify if:

  • Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.
  • Administration of loop diuretics within 7 days prior to study drug administration.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

Novartis Investigative Site

Toronto, Ontario, M5G 1X8, Canada

Location

Novartis Investigative Site

Montreal, Quebec, H3T1C5, Canada

Location

Novartis Investigative Site

Alexandria, 21131, Egypt

Location

Novartis Investigative Site

Bordeaux, 33076, France

Location

Novartis Investigative Site

Paris, 75019, France

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Pátrai, Greece, 265 00, Greece

Location

Novartis Investigative Site

Thessaloniki, GR, 546 29, Greece

Location

Novartis Investigative Site

Thessaloniki, GR, 546 42, Greece

Location

Novartis Investigative Site

Debrecen, Hungary, 4032, Hungary

Location

Novartis Investigative Site

Kaposvár, Hungary, 7400, Hungary

Location

Novartis Investigative Site

Szeged, Hungary, 6725, Hungary

Location

Novartis Investigative Site

Debrecen, 4043, Hungary

Location

Novartis Investigative Site

Milan, MI, 20122, Italy

Location

Novartis Investigative Site

Bucharest, 20395, Romania

Location

Novartis Investigative Site

Kazan', 420012, Russia

Location

Novartis Investigative Site

Voronezh, 394036, Russia

Location

Novartis Investigative Site

Yaroslavl, 150003, Russia

Location

Novartis Investigative Site

Zurich, Switzerland, 8032, Switzerland

Location

Related Publications (1)

  • Ratjen F, Moeller A, McKinney ML, Asherova I, Alon N, Maykut R, Angyalosi G; EARLY study group. Eradication of early P. aeruginosa infection in children <7 years of age with cystic fibrosis: The early study. J Cyst Fibros. 2019 Jan;18(1):78-85. doi: 10.1016/j.jcf.2018.04.002. Epub 2018 Apr 21.

MeSH Terms

Conditions

Cystic FibrosisLung Diseases

Condition Hierarchy (Ancestors)

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

Results Point of Contact

Title
Study Director
Organization
Novartis

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 5, 2010

First Posted

March 8, 2010

Study Start

April 1, 2010

Primary Completion

June 1, 2015

Study Completion

June 1, 2015

Last Updated

July 15, 2016

Results First Posted

April 27, 2016

Record last verified: 2016-06

Locations