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Tobramycin

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Novartis AG|Last Updated: Jun 2, 2021

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLED
Total Trials5
Total Enrollment734

FDA Designations

No designations recorded

Clinical trial landscape

Tobramycin · 8 trials · 4 indications

Phase 3 6Phase 1 2
NCT01069705Second Open Label Extension to Bridging Study CTBM100C2303Pulmonary Infections
COMPLETED49 Analytics
NCT00982930Open Label Extension to Bridging Study CTBM100C2303Pseudomonas Aeruginosa
COMPLETED55 Analytics
NCT00918957A Study of Tobramycin Inhalation Powder From a Modified Manufacturing Process Versus PlaceboCystic Fibrosis
COMPLETED62 Analytics
NCT00634192Pharmacokinetic Evaluation of an 8 -Week Treatment With Inhaled TobramycinPseudomonas Infections
COMPLETED50 Analytics
NCT00388505Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic FibrosisCystic Fibrosis
COMPLETED517 Analytics
NCT00391976Efficacy and Safety of 28 or 56 Day Treatment for Pseudomonas Aeruginosa in Children With Cystic FibrosisCystic Fibrosis
COMPLETED123 Analytics
PHASE3COMPLETED
Second Open Label Extension to Bridging Study CTBM100C2303
Pulmonary InfectionsUnlock trial analytics
PHASE3COMPLETED
Open Label Extension to Bridging Study CTBM100C2303
Pseudomonas AeruginosaUnlock trial analytics
PHASE3COMPLETED
A Study of Tobramycin Inhalation Powder From a Modified Manufacturing Process Versus Placebo
Cystic FibrosisUnlock trial analytics
PHASE3COMPLETED
Pharmacokinetic Evaluation of an 8 -Week Treatment With Inhaled Tobramycin
Pseudomonas InfectionsUnlock trial analytics
PHASE3COMPLETED
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
Cystic FibrosisUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of 28 or 56 Day Treatment for Pseudomonas Aeruginosa in Children With Cystic Fibrosis
Cystic FibrosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Adverse Events (AEs)
From time of first administration of study drug until study completion (up to 169 days)

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.

Number of Participants With Serious Adverse Events (SAEs)
From time of consent to 4 weeks after study completion (up to 199 days)

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.

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

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.

Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)

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

Percentage of Participants With Adverse Events (AEs)
From first administration of study drug to study completion (up to approximately 25 weeks)

AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product.

Percentage of Participants With Serious Adverse Events (SAEs)
From time of consent to 4 weeks after study completion (up to approximately 29 weeks)

SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.

Percentage of Death Cases
From time of consent to 4 weeks after study completion (up to approximately 29 weeks)
Percentage of Participants With Adverse Events and Serious Adverse Events Leading to Permanent Study Discontinuation
From first administration of study drug to study completion (up to approximately 25 weeks)

AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product. SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.

Shift From Baseline in Laboratory Parameters to Above Upper/Lower Limit of Normal
From baseline to study completion (up to approximately 25 weeks)

Shift from baseline in hematology and biochemistry values to above upper/below lower limit of normal at any time post-baseline were reported. Baseline for safety analyses was defined as the last measurement prior to first dose of study drug in the core study CTBM100C2303. Change to low referred to number of participants with normal or high values at baseline. Change to high referred to number of participants with normal or low values at baseline.

Acute Relative Change in Airways Reactivity [Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted] From Pre-dose to 30 Minutes After Completion of First Dose of Study Drug
Pre-dose and 30 minutes Post-dose on Day 1 and Day 29 of every Cycle (2, 3, 4)

Airway Reactivity was defined as ≥20% FEV1 relative decrease in percent predicted from pre dose to 30 minutes post dose. FEV1 was defined as the volume of air expired in 1 second. FEV1 % predicted was a normalized value of FEV1 calculated using the Knudsen equation, based upon participant's age, gender and height. Relative change in FEV1 % predicted = 100\* (30 minutes post dose - pre dose) / pre dose assessed by number and percentage of participants with a decrease in ≥20% FEV1 percent predicted from pre dose to 30 minutes post dose. Baseline for was defined as the last measurement prior to first dose of study drug in the core study CTBM100C2303.

Number of Participants With Adverse Events (AEs) Associated With the Use of New Antipseudomonal Antibiotic
From first administration of study drug to study completion (up to approximately 25 weeks)

AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not related to the medicinal product. SAEs included adverse events that resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.

Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing (Day 29)
Baseline, Day 29

Relative change in percentage predicted FEV1 in the Intent-to-treat (ITT), modified ITT (mITT) and Observed cases in the ITT populations were calculated from an adjusted analysis ANOVA model. ITT Patients with missing or unacceptable Day 29 spirometry measurements had their primary endpoint data imputed with zero. BSL = Baseline, defined as the latest measurement prior to the first dosing of study medication \- Relative change = 100 \* (value - baseline) / baseline There were 3 patients who had no screening nor baseline values (due to inadequate spirometry) and so were excluded from all change from baseline analyses.

Pre-planned Sensitivity Analysis: Absolute Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent (%) Predicted to End of Dosing (Day 29)
Baseline, Day 29

Absolute change in percentage predicted FEV1 in the Intent-to-treat (ITT), modified ITT (mITT) and Observed cases in the ITT populations were calculated from an adjusted analysis ANOVA model. In the adjusted analysis model: response = treatment + screening FEV1 % predicted (\<50 and \>=50) + age (\<13 and \>=13) + error. Significance for the FEV1 % predicted is reached for p-values \<= 0.05. There were 3 patients who had no screening nor baseline values (due to inadequate spirometry) and so were excluded from all change from baseline analyses.

Post-hoc: Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to End of Dosing (Day 29) Without Outlier
Baseline, Day 29

Relative change in percentage predicted FEV1 without outlier (outliers with respect to FEV1 values and PK data), in the Intent-to-treat (ITT), modified ITT (mITT) and Observed cases in the ITT populations were calculated from an adjusted analysis ANOVA model.

To evaluate the serum pharmacokinetics (PK) of inhaled tobramycin (AUC 0-90') of continuous daily dosing regimens with 2x300mg/d Tobramycin Nebuliser Solution (=TNS) inhaled with the PARI eFlow® rapid in Cystic Fibrosis (CF) subjects
8 wks
Number of Participants With Treatment-emergent Adverse Events
25 weeks

An adverse event (AE) is any untoward medical occurrence, including any unfavorable and unintended sign, symptom or disease temporally associated with the use of the study medication that does not necessarily have a causal relationship with study medication. A serious AE (SAE) is an event that results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability, is a congenital anomaly or defect, or is a significant medical event that may jeopardize the patient or require intervention to prevent one of the outcomes listed above.

Time to Recurrence of Pseudomonas (P.) Aeruginosa (Any Genotype) in Sputum or Deep Throat Cough Swab
From 1 month after the end of treatment (Day 56 for the 28-day treatment group and Month 3 for the 56-day treatment group) until the end of the study (Month 27)

Microbiological samples were obtained from sputum or by deep throat cough swab technique. Time to recurrence was defined as the time between the visit at 1 month after the end of treatment (when eradication was confirmed) and the time of the first positive culture with any genotype of P. aeruginosa. Time zero was Day 56 (Month 2) for the 28-day treatment group and Month 3 for the 56-day treatment group. Kaplan-Meier estimates were used.

Lung deposition of tobramycin when inhaled using either PARI LC PLUS jet nebulizer or PARI eFlow rapid Electronic Nebulizer
Aerosol delivery characteristics of tobramycin when inhaled using either PARI LC PLUS jet nebulizer or PARI eFlow rapid Electronic Nebulizer evaluated by serum concentrations

Secondary Endpoints

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)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Tobramycin Inhalation Powder (TIPnew)EXPERIMENTALParticipants 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.
Tobramycin Inhalation Powder (TIP)EXPERIMENTALParticipants received 112 mg (four 28 mg capsules) of TIP administered by the T-326 Inhaler, twice a day (b.i.d.), given in a cycle of 28 days on treatment followed by 28 days off treatment (one cycle = 56 days) for up to 3 cycles.
TIP (Tobramycin Inhalation Powder)EXPERIMENTALTobramycin 28 mg powder. The TIP dose of 112 mg twice a day (bis in diem = b.i.d.), given in a cycle of 28 days on treatment followed by 28 days off treatment.
PlaceboPLACEBO_COMPARATORPlacebo 20 mg powder capsules. The dose regimen for the reference product was inhaling the contents of four capsules twice a day (bis in diem = b.i.d.), in the morning and in the evening for 28 days (on treatment), followed by 28 days of no study treatment (off treatment).
1EXPERIMENTAL -
2EXPERIMENTAL -
Tobramycin solution for inhalation (TOBI)ACTIVE_COMPARATORParticipants received one 300 mg (in 5 mL) ampoule of tobramycin solution for inhalation (TOBI) delivered with a nebulizer twice daily for 28 days followed by 28 days off therapy (one cycle) for a total of three cycles.
Tobramycin 300 mg for 28 daysEXPERIMENTALPatients inhaled tobramycin 300 mg bis in die (bid, twice a day) for 28 days using the PARI LC PLUS™ jet nebulizer and a suitable compressor. The 2 daily doses were taken approximately 12 hours apart and no less than 6 hours apart.
Tobramycin 300 mg for 56 daysEXPERIMENTALPatients inhaled tobramycin 300 mg bis in die (bid, twice a day) for 56 days using the PARI LC PLUS™ jet nebulizer and a suitable compressor. The 2 daily doses were taken approximately 12 hours apart and no less than 6 hours apart.

Interventions

NameTypeDescription
Tobramycin inhalation powderDRUGTobramycin dry powder for inhalation in capsules administered by the T-326 inhaler.
PlaceboDRUGPlacebo inhalation powder consisting of the excipients used for TIP. Placebo was provided in hard capsules, containing 20 mg placebo powder, which were packaged in blister cards, matching in appearance to TIP. Capsules were administered by the T-326 Inhaler.
tobramycinDRUG1x300mg/d inhaled
Tobramycin Solution for InhalationDRUGTobramycin solution for inhalation (TOBI), supplied as 300 mg/5mL ampoules administered with a nebulizer
Tobramycin solution for inhalation 300 mgDRUGTobramycin solution for inhalation was supplied in 5 mL liquid-filled low-density polyethylene ampoules containing 300 mg tobramycin. Patients used a nebulizer to inhale the contents of the ampoules.
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Eligibility Criteria

Age Range6 Years to 21 Years
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: * 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...

Countries:BulgariaEstoniaLatviaLithuaniaRomaniaRussiaSouth AfricaEgyptIndiaGermanyUnited StatesAustraliaCanadaChileColombiaFranceHungaryIsraelItalyMexicoNetherlandsSpainUnited Kingdom
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Frequently asked questions about Tobramycin

What is Tobramycin used for?

Tobramycin is an investigational small molecule being developed for pulmonary infections, cystic fibrosis, Pseudomonas infections, and Pseudomonas aeruginosa. It is administered via inhalation and is currently in Phase 3 clinical development. The drug is being studied in patients with cystic fibrosis who have Pseudomonas aeruginosa infections.

Who makes Tobramycin?

Tobramycin is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the safety and efficacy of Tobramycin inhalation formulations in patients with cystic fibrosis.

What phase is Tobramycin in?

Tobramycin is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 3 trials are studying the safety and efficacy of Tobramycin inhalation powder and solution in patients with cystic fibrosis and Pseudomonas aeruginosa infections.

What clinical trials is Tobramycin in?

Tobramycin has completed five clinical trials, including two Phase 3 studies. NCT00388505 evaluated the safety of Tobramycin inhalation powder versus solution in 517 cystic fibrosis patients. NCT00391976 studied 28 or 56 day treatment in 123 children with cystic fibrosis and Pseudomonas aeruginosa. Two Phase 1 trials examined nebulizer delivery.

How does Tobramycin work?

Tobramycin is an aminoglycoside antibiotic that works by inhibiting bacterial protein synthesis. It binds to the bacterial 30S ribosomal subunit, causing misreading of the genetic code and producing nonfunctional proteins. This bactericidal action is effective against Pseudomonas aeruginosa, a common pathogen in cystic fibrosis pulmonary infections.