Cystic FibrosisPhase 3CT03ExtNCT01111383
A Single Arm 48-Week Follow-on Safety Study to a Core Study Comparing the Efficacy and Tolerability of Tobrineb®/Actitob®/Bramitob® Versus TOBI®
A Phase 3 study testing tobramycin in people with Cystic Fibrosis, run by Chiesi Farmaceutici S.p.A. at 20 sites in 3 countries.
Trial summary
This is a 48-week extension study to CMA-0631-PR-0010 Core Study. Patients who have a positive culture for P. aeruginosa at visit 4 of the first 8-week core study period and/or if deemed appropriate by the Investigators will be able to be included in the 48-week follow-on period (Extension Study) to continue the… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- tobramycin
- How many people
- 209 participants
- Where
- 20 sitesin 3 countries
- Study dates
- Sep 2009to May 2011
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Successful completion of Core Study
- At least 6 years of age
- Males and females
Who cannot join
None listed.
Trial locations and contacts
20 sites in 3 countries.
20 sites in 3 countries
Ukraine9 sites
Dnipropetrovsk City Children Clinical Hospital # 2
Dnipropetrovsk, 49101
Donetsk Regional Children Clinical Hospital
Donetsk, 83052
Kriviy Rig City Clinical Hospital # 8
Kryvyi Rih, 50047
Institute of Phthysiology and Pulmonology n.a., F.G.Yanovskiy of the Academy of Medical Science of Ukraine
Kyiv, 03680
Institute of Pediatrics, Obstetrics and Gynecology of the Academy of Medical Science of Ukraine
Kyiv, 04050
Lviv Regional Children Specialized Clinical Hospital
Lviv, 79035
Odesa Regional Children Clinical Hospital
Odesa, 65031
Simferopol Central District Clinical Hospital
Simferopol, 95033
Zaporizhya Regional Clinical Children Hospital
Zaporizhya
Poland8 sites
Specjalistyczny ZOZ nad Matka i Dzieckiem, Poradnia Leczenia Mukowiscydozy
Gdansk, 80-308
I Oddzial Chorob Dzieciecych, Wojewodzki Specjalistyczny Szpital Dzieciecy
Kielce, 25-381
Oddzial Kliniczny Interny Dzieciecej i Alergologii, Wojewodzki Szpital Specjalistyczny
Lodz, 93-513
Dzieciecy Szpital Kliniczny Akademii Medycznej, Klinika Chorob Pluc I Reumatologii
Lublin, 20-093
Klinika Pneumonologii, Alergologii Dzieciecej i Immunologii Klinicznej Szpital Kliniczny Uniwersytetu Medycznego w Poznaniu
Poznan, 60-572
Klinika Pneumonologii i Mukowiscydozy, Instytut Gruzlicy i Chorob Pluc w Rabce Zdroj
Rabka-Zdrój, 34-700
Poradnia Mukowiscydozy Wojewodzkiej, Przychodni Specjalistycznej dla Dzieci, Szpitala Wojewodzkiego Nr 2
Rzeszów, 35-301
Klinika Pediatrii Instytut Matki I Dziecka
Warsaw, 01-211
France3 sites
CHR Clemenceau
Caen, 14 033
Hopital Arnaud de Villeneuve, Clinique des maladies respiratoires
Montpellier, 34 295
Hopital Necker
Paris, 75 015
About this study
Official title: A Single Arm 48-Week Follow-on Safety Study to the Core Study (A Multicentre, Multinational, Open-Label, Randomised, Parallel Group Clinical Trial of Tobrineb®/Actitob®/Bramitob® (Tobramycin Solution for Nebulisation, 300mg Twice Daily in 4mL Unit Dose Vials) Compared to TOBI® in the Treatment of Patients With Cystic Fibrosis and Chronic Infection With Pseudomonas Aeruginosa)
This is a 48-week extension study to CMA-0631-PR-0010 Core Study. Patients who have a positive culture for P. aeruginosa at visit 4 of the first 8-week core study period and/or if deemed appropriate by the Investigators will be able to be included in the 48-week follow-on period (Extension Study) to continue the treatment only with Bramitob® (tobramycin nebuliser solution, 300 mg twice daily in 4 mL unit dose vials).
- Conditions
- Cystic Fibrosis
- Being tested
- tobramycin
- Keywords
- cystic fibrosis, P. aeruginosa, tobramycin
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tobramycinDrug
Also called Tobrineb®/Actitob®/Bramitob®
300mg/4ml solution, via a nebuliser, over a 48-week period in a twice-daily regimen, with 6 "on" cycles of 4 weeks duration during the 48-week period.
What is being measured
The primary outcome is the main question the study is designed to answer.
to assess safety profile in terms of incidence of adverse events/adverse drug reactions, frequency of cystic fibrosis exacerbations, audiometric test, laboratory parameters (hematology and blood chemistry), vitals signs (hr and bp), physical examination.
Measured: up to 48 weeks
Secondary outcomes (4)
to assess whether prolonged use of aerosolized tobramycin is required to sustain FEV1 increase (FEV1 expressed in liters and % predicted)
Measured: up to 48 weeks
Categorical results of microbiological tests referred to P. aeruginosa (negativisation, persistence, superinfection, re-infection); susceptibility testing of isolated P. aeruginosa strains (MIC90 and MIC50)
Measured: up to 48 weeks
Changes in body weight and BMI
Measured: up to 48 weeks
to assess health related quality of life
Measured: Initial visit, Week 20, Week 44
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2010 (estimated)
- Study startSep 2009
- Primary completionMay 2011
- Study completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (29)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 209 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Henryk Mazurek, Doctor, Principal investigator, Klinika Pneumonologii i Mukowiscydozy, Instytut Gruzlicy i Chorob Pluc w Rabce Zdroj
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01111383
- Sponsor's protocol ID
- CMA-0631-PR-0010 Extension
- Acronym
- CT03Ext
- Status last verified
- Jul 2020
- First submitted
- 13 Apr 2010
- Last updated
- 31 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
Wine JJ. Cystic fibrosis: How do CFTR mutations cause cystic fibrosis? Curr Biol. 1995 Dec 1;5(12):1357-9. doi: 10.1016/s0960-9822(95)00269-7. No abstract available.
PubMed 8749384Lewis PA. The epidemiology of cystic fibrosis. In: Hodson ME, Geddes DM. Cystic Fibrosis 2nd edition, Arnold, London 2000; 1a: 2-12.
Dodge JA, Morison S, Lewis PA, Coles EC, Geddes D, Russell G, Littlewood JM, Scott MT. Incidence, population, and survival of cystic fibrosis in the UK, 1968-95. UK Cystic Fibrosis Survey Management Committee. Arch Dis Child. 1997 Dec;77(6):493-6. doi: 10.1136/adc.77.6.493.
PubMed 9496181Lucotte G, Hazout S, De Braekeleer M. Complete map of cystic fibrosis mutation DF508 frequencies in Western Europe and correlation between mutation frequencies and incidence of disease. Hum Biol. 1995 Oct;67(5):797-803.
PubMed 8543293Bossi A, Battistini F, Braggion C, Magno EC, Cosimi A, de Candussio G, Gagliardini R, Giglio L, Giunta A, Grzincich GL, La Rosa M, Lombardo M, Lucidi V, Manca A, Mastella G, Moretti P, Padoan R, Pardo F, Quattrucci S, Raia V, Romano L, Salvatore D, Taccetti G, Zanda M. [Italian Cystic Fibrosis Registry: 10 years of activity]. Epidemiol Prev. 1999 Jan-Mar;23(1):5-16. Italian.
PubMed 10356860Yamashiro Y, Shimizu T, Oguchi S, Shioya T, Nagata S, Ohtsuka Y. The estimated incidence of cystic fibrosis in Japan. J Pediatr Gastroenterol Nutr. 1997 May;24(5):544-7. doi: 10.1097/00005176-199705000-00010.
PubMed 9161949Imaizumi Y. Incidence and mortality rates of cystic fibrosis in Japan, 1969-1992. Am J Med Genet. 1995 Aug 28;58(2):161-8. doi: 10.1002/ajmg.1320580215.
PubMed 8533810Boat TF, Welsh MJ, Beaudet AL. Cystic fibrosis, in the metabolic basis of inherited disease (7th edn). McGraw-Hill, New York,1995.
Wood RE, Boat TF, Doershuk CF. Cystic fibrosis. Am Rev Respir Dis. 1976 Jun;113(6):833-78. doi: 10.1164/arrd.1976.113.6.833. No abstract available.
PubMed 779549Rommens JM, Iannuzzi MC, Kerem B, Drumm ML, Melmer G, Dean M, Rozmahel R, Cole JL, Kennedy D, Hidaka N, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science. 1989 Sep 8;245(4922):1059-65. doi: 10.1126/science.2772657.
PubMed 2772657Riordan JR, Rommens JM, Kerem B, Alon N, Rozmahel R, Grzelczak Z, Zielenski J, Lok S, Plavsic N, Chou JL, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989 Sep 8;245(4922):1066-73. doi: 10.1126/science.2475911.
PubMed 2475911Anderson MP, Gregory RJ, Thompson S, Souza DW, Paul S, Mulligan RC, Smith AE, Welsh MJ. Demonstration that CFTR is a chloride channel by alteration of its anion selectivity. Science. 1991 Jul 12;253(5016):202-5. doi: 10.1126/science.1712984.
PubMed 1712984Trapnell BC, Chu CS, Paakko PK, Banks TC, Yoshimura K, Ferrans VJ, Chernick MS, Crystal RG. Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis. Proc Natl Acad Sci U S A. 1991 Aug 1;88(15):6565-9. doi: 10.1073/pnas.88.15.6565.
PubMed 1713683Høiby N. Microbiology of lung infections in cystic fibrosis patients. Acta Paediatr Scand 1982; 301: 33-54.
Moss RB, Babin S, Hsu YP, Blessing-Moore J, Lewiston NJ. Allergy to semisynthetic penicillins in cystic fibrosis. J Pediatr. 1984 Mar;104(3):460-6. doi: 10.1016/s0022-3476(84)81117-8.
PubMed 6323664
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm 48-Week Follow-on Safety Study to the Core Study (A Multicentre, Multinational, Open-Label, Randomised, Parallel Group Clinical Trial of Tobrineb®/Actitob®/Bramitob® (Tobramycin Solution for Nebulisation, 300mg Twice Daily in 4mL Unit Dose Vials) Compared to TOBI® in the Treatment of Patients With Cystic Fibrosis and Chronic Infection With Pseudomonas Aeruginosa)
- ClinicalTrials.gov ID
- NCT01111383
- Acronym / sponsor ID
- CT03Ext / CMA-0631-PR-0010 Extension
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, P. aeruginosa, tobramycin
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 209 participants (actual)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- Sep 2009
- Primary completion
- May 2011 (actual)
- Study completion
- May 2011 (actual)
- First posted
- 27 Apr 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 48-week extension study to CMA-0631-PR-0010 Core Study. Patients who have a positive culture for P. aeruginosa at visit 4 of the first 8-week core study period and/or if deemed appropriate by the Investigators will be able to be included in the 48-week follow-on period (Extension Study) to continue the treatment only with Bramitob® (tobramycin nebuliser solution, 300 mg twice daily in 4 mL unit dose vials).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Successful completion of Core Study
- At least 6 years of age
- Males and females
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| tobramycinAlso: Tobrineb®/Actitob®/Bramitob®300mg/4ml solution, via a nebuliser, over a 48-week period in a twice-daily regimen, with 6 "on" cycles of 4 weeks duration during the 48-week period. | Drug | – |
Primary outcome
to assess safety profile in terms of incidence of adverse events/adverse drug reactions, frequency of cystic fibrosis exacerbations, audiometric test, laboratory parameters (hematology and blood chemistry), vitals signs (hr and bp), physical examination.
Time frame: up to 48 weeks
4 secondary outcomes
to assess whether prolonged use of aerosolized tobramycin is required to sustain FEV1 increase (FEV1 expressed in liters and % predicted)
Time frame: up to 48 weeks
Categorical results of microbiological tests referred to P. aeruginosa (negativisation, persistence, superinfection, re-infection); susceptibility testing of isolated P. aeruginosa strains (MIC90 and MIC50)
Time frame: up to 48 weeks
Changes in body weight and BMI
Time frame: up to 48 weeks
to assess health related quality of life
Time frame: Initial visit, Week 20, Week 44
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2020.
- Background: PubMed 8749384Wine JJ. Cystic fibrosis: How do CFTR mutations cause cystic fibrosis? Curr Biol. 1995 Dec 1;5(12):1357-9. doi: 10.1016/s0960-9822(95)00269-7. No abstract available.
- BackgroundLewis PA. The epidemiology of cystic fibrosis. In: Hodson ME, Geddes DM. Cystic Fibrosis 2nd edition, Arnold, London 2000; 1a: 2-12.
- Background: PubMed 9496181Dodge JA, Morison S, Lewis PA, Coles EC, Geddes D, Russell G, Littlewood JM, Scott MT. Incidence, population, and survival of cystic fibrosis in the UK, 1968-95. UK Cystic Fibrosis Survey Management Committee. Arch Dis Child. 1997 Dec;77(6):493-6. doi: 10.1136/adc.77.6.493.
- Background: PubMed 8543293Lucotte G, Hazout S, De Braekeleer M. Complete map of cystic fibrosis mutation DF508 frequencies in Western Europe and correlation between mutation frequencies and incidence of disease. Hum Biol. 1995 Oct;67(5):797-803.
- Background: PubMed 10356860Bossi A, Battistini F, Braggion C, Magno EC, Cosimi A, de Candussio G, Gagliardini R, Giglio L, Giunta A, Grzincich GL, La Rosa M, Lombardo M, Lucidi V, Manca A, Mastella G, Moretti P, Padoan R, Pardo F, Quattrucci S, Raia V, Romano L, Salvatore D, Taccetti G, Zanda M. [Italian Cystic Fibrosis Registry: 10 years of activity]. Epidemiol Prev. 1999 Jan-Mar;23(1):5-16. Italian.
- Background: PubMed 9161949Yamashiro Y, Shimizu T, Oguchi S, Shioya T, Nagata S, Ohtsuka Y. The estimated incidence of cystic fibrosis in Japan. J Pediatr Gastroenterol Nutr. 1997 May;24(5):544-7. doi: 10.1097/00005176-199705000-00010.
- Background: PubMed 8533810Imaizumi Y. Incidence and mortality rates of cystic fibrosis in Japan, 1969-1992. Am J Med Genet. 1995 Aug 28;58(2):161-8. doi: 10.1002/ajmg.1320580215.
- BackgroundBoat TF, Welsh MJ, Beaudet AL. Cystic fibrosis, in the metabolic basis of inherited disease (7th edn). McGraw-Hill, New York,1995.
- Background: PubMed 779549Wood RE, Boat TF, Doershuk CF. Cystic fibrosis. Am Rev Respir Dis. 1976 Jun;113(6):833-78. doi: 10.1164/arrd.1976.113.6.833. No abstract available.
- Background: PubMed 2772657Rommens JM, Iannuzzi MC, Kerem B, Drumm ML, Melmer G, Dean M, Rozmahel R, Cole JL, Kennedy D, Hidaka N, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science. 1989 Sep 8;245(4922):1059-65. doi: 10.1126/science.2772657.
- Background: PubMed 2475911Riordan JR, Rommens JM, Kerem B, Alon N, Rozmahel R, Grzelczak Z, Zielenski J, Lok S, Plavsic N, Chou JL, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989 Sep 8;245(4922):1066-73. doi: 10.1126/science.2475911.
- Background: PubMed 1712984Anderson MP, Gregory RJ, Thompson S, Souza DW, Paul S, Mulligan RC, Smith AE, Welsh MJ. Demonstration that CFTR is a chloride channel by alteration of its anion selectivity. Science. 1991 Jul 12;253(5016):202-5. doi: 10.1126/science.1712984.
- Study Record on EU Clinical Trials Register including resultsLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Henryk Mazurek, Doctor principal investigator
Klinika Pneumonologii i Mukowiscydozy, Instytut Gruzlicy i Chorob Pluc w Rabce Zdroj
20 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing tobramycin in people with Cystic Fibrosis, run by Chiesi Farmaceutici S.p.A. at 20 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Ukraine, Poland, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
to assess safety profile in terms of incidence of adverse events/adverse drug reactions, frequency of cystic fibrosis exacerbations, audiometric test, laboratory parameters (hematology and blood chemistry), vitals signs (hr and bp), physical examination. (time frame: up to 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01111383, which also lists 29 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2020.