Second Open Label Extension to Bridging Study CTBM100C2303

NCT01069705 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: CTBM100C2303E2
• Study Type: interventional
• Target: 49
• Locations: 7 countries
• Sites: 17

Brief Summary

This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who have completed their study participation in CTBM100C2303 and extension study one CTBM100C2303E1 (all visits), who were proven infected with Pseudomonas aeruginosa at enrollment into CTBM100C2303.

Conditions

Pulmonary Infections; Pseudomonas Aeruginosa

Keywords

Tobramycin Inhalation Powder; Cystic fibrosis; Lung diseases; Anti-Bacterial Agents; Treatment of pulmonary infections with P. aeruginosa in cystic fibrosis participants

Outcome Measures

Primary Outcomes (4)

• Number of Participants With Adverse Events (AEs)

  An AE was defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug.

  From time of first administration of study drug until study completion (up to 169 days)

• Number of Participants With Serious Adverse Events (SAEs)

  A SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes.

  From time of consent to 4 weeks after study completion (up to 199 days)

• Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose

  Airway Reactivity \>= 20% relative decrease in FEV1% predicted from pre-dose to 30 minutes post-dose. Relative Change = 100 \* (30 minutes Post-dose - Pre-dose)/Pre-dose assessed by the number and percentage of participants with a decrease of ≥ 20% in FEV1 % predicted from pre-dose to 30 minutes post-dose.

  Pre-dose and post-dose of Day 1 and Day 29 of every Cycle (5, 6, 7)

• Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests

  Auditory acuity of participants was measured using a standard dual-channel audiometer at frequencies from 250 to 8000 Hertz, and an audiogram (pure-tone air conduction) and tympanogram were performed by an audiologist. The categories reported includes \>= 10dB decrease in 3 consecutive frequencies in either ear, \>= 15dB decrease in 2 consecutive frequencies in either ear, and \>= 20dB decrease in at least one frequency in either ear

  Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

Secondary Outcomes (8)

• Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit

  Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

• Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit

  Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

• Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit

  Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

• Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit

  Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

• Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit

  Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

Study Arms (1)

Tobramycin Inhalation Powder (TIPnew)

EXPERIMENTAL

Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.

Drug: Tobramycin inhalation powder

Interventions

Tobramycin inhalation powder DRUG

Tobramycin dry powder for inhalation in capsules administered by the T-326 inhaler.

Tobramycin Inhalation Powder (TIPnew)

Eligibility Criteria

• Age: 6 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Completed all visits in study CTBM100C2303 and CTBM100C2303E1, and visit 11 of study CTBM100C2303E1 took place not more than 5 days before enrollment into this study.
• Confirmed diagnosis of cystic fibrosis participants with P. aeruginosa infection.
• Forced Expiratory Volume in one second (FEV1) at screening (at start of study CTBM100C2303) must be between 25% and 80% of normal predicted values.

You may not qualify if:

• Any use of inhaled anti-pseudomonal antibiotics between the termination of the trial CTMB100C2303E1 and the enrollment into this study.
• Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (17)

Novartis Investigative Site

Pleven, Bulgaria

Location

Novartis Investigative Site

Plovdiv, Bulgaria

Location

Novartis Investigative Site

Sofia, Bulgaria

Location

Novartis Investigative Site

Varna, Bulgaria

Location

Novartis Investigative Site

Tallinn, Estonia

Location

Novartis Investigative Site

Tartu, Estonia

Location

Novartis Investigative Site

Riga, Latvia

Location

Novartis Investigative Site

Kaunas, Lithuania

Location

Novartis Investigative Site

Vilnius, Lithuania

Location

Novartis Investigative Site

Bucharest, Romania

Location

Novartis Investigative Site

Timișoara, Romania

Location

Novartis Investigative Site

Kazan', Russia

Location

Novartis Investigative Site

Moscow, Russia

Location

Novartis Investigative Site

Saint Petersburg, Russia

Location

Novartis Investigative Site

Samara, Russia

Location

Novartis Investigative Site

Yaroslavl, Russia

Location

Novartis Investigative Site

Durban, South Africa

Location

Related Publications (1)

• Konstan MW, Flume PA, Galeva I, Wan R, Debonnett LM, Maykut RJ, Angyalosi G. One-year safety and efficacy of tobramycin powder for inhalation in patients with cystic fibrosis. Pediatr Pulmonol. 2016 Apr;51(4):372-8. doi: 10.1002/ppul.23358. Epub 2015 Dec 27.

  PMID: 26709158 RESULT

MeSH Terms

Conditions

Pseudomonas Infections; Cystic Fibrosis; Lung Diseases

Condition Hierarchy (Ancestors)

Gram-Negative Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections; Pancreatic Diseases; Digestive System Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 15, 2010
• First Posted: February 17, 2010
• Study Start: February 12, 2010
• Primary Completion: March 19, 2012
• Study Completion: March 19, 2012
• Last Updated: June 2, 2021
• Results First Posted: June 2, 2021

Record last verified: 2021-05

Locations

RO (2); LI (2); ES (2); RU (5); LA (1); ZA (1); BU (4)