Clinical trial NCT01455675: Efficacy Study of IgY (Antibody Against Pseudomonas) in Cystic Fibrosis Patients
Official title: Phase III Study to Evaluate Clinical Efficacy and Safety of Avian Polyclonal Anti-Pseudomonas Antibodies (IgY) in Prevention of Recurrence of Pseudomonas Aeruginosa Infection in Cystic Fibrosis Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Keywords: cystic fibrosis
Lead sponsor: Mukoviszidose Institut gGmbH
Sponsor class: OTHER
First posted: 2011-10-20
Enrollment: 164
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: The purpose of this study is to prolong the time to reinfection with Pseudomonas aeruginosa after successfully treated acute or intermittent infection.

Detailed description: This is a double -blind, placebo controlled study in which the investigational drug and the reference placebo group are gargled and swallowed. 70 ml IgY/ placebo solution is gargled every night for two minutes (for maximal 24 months) The design will include the recruitment of 144 patients randomized in two groups (72 per treatment group) In order to compensate for dropouts (i.e. patients dropping out prior to 24 months without having an event) the total sample size was planned to be approximately 180 (i.e. \~20 % dropout rate). After the actual drop-out rate has been low throughout the study, only 144 plus approx. 10% potential drop-outs were included into the study.

During the two years of treatment, subjects will be examined at the clinic every 3 months regarding safety and efficacy of the medication.

For more information please see www.impactt.eu The IMPACTT Project is funded by EU within the Framework 7 Program. PsAer-IgY Studies is part of IMPACTT Project (Workpackage 2).

Eligibility criteria: Inclusion Criteria:

* CF patients diagnosed according to specific clinical features and either a positive sweat chloride in double proofs or presence of disease-associated CFTR mutations in both alleles
* Males and females 5 years of age and above (being able to gargle)
* CF patients having a FEV1 value between 50% and 130% of predicted value (according to Knudson formula)
* CF patients who have had one to several sputum or throat cough swabs or endolaryngeal suction cultures positive for PA within the last three years and for whom PA has been successfully eradicated.
* Sputum / throat cough swab/ endolaryngeal suction culture negative for PA and other gram-negative bacteria on study entry.
* Patients and/ or their legal representative who are willing and able to give informed consent/ assent to participate in the study after thorough information
* Subjects of child bearing potential and who are sexually active must meet the contraception requirements (i.e. oral or injectable contraceptives, intrauterine devices, double-barrier method, contraceptive patch, male partner sterilization or condoms).

Exclusion Criteria:

* Microbiologic or serologic evidence of chronic infection with PA. Definition of chronic PA infection: Three cultures (sputum or throat cough swabs or endolaryngeal suction) have been positive for PA for 6 consecutive months (at least 3 cultures have to be taken) or more, .
* Patients, who have positive sputum culture or throat cough swab or endolaryngeal suction culture for gram-negative bacteria, such as PA, S. maltophilia, B. cepacia, A. xylosoxidans (eradication before entry in study is possible), Patients, who have positive sputum culture or throat cough swab or endolaryngeal suction culture for atypical Mycobacteria and / or Aspergillus fumigates, associated with clinical symptoms that may necessitate specific treatment.
* History of allergy/hypersensitivity to hens' egg proteins (including medication allergy) that is deemed relevant to the trial by the investigator. "Relevance" in this context refers to any increased risk of hypersensitivity reaction to trial medication.
* Patient with a known relevant substance abuse, including alcohol or drug abuse.
* Start of a new concomitant or chronic medication for CF within 4 weeks before inclusion.
* Clinically relevant diseases or medical conditions other than CF or CF-related conditions that, in the opinion of the investigator, would compromise the safety of the patient or the quality of the data. This includes, but is not limited to, significant hematological, hepatic, renal, cardiovascular, and neurological diseases (diabetic patients may participate if their disease is under good control prior to inclusion).
* Participation in another study with an investigational drug within one month or 6 half-lives (whichever is greater) preceding the inclusion.
* The patient is an employee of the investigator or the institution with direct involvement in the trial or other trials under the direction of the investigator or their members.
* Patients who are pregnant cannot be included into the study. This will be tested at inclusion visit with a urine pregnancy test (in female patients older than 10 years with secondary sexual characteristics)

Primary outcomes:
measure: Time from start of treatment (=Day 0) to the first recurrence of PA (Pseudomonas aeruginosa) in the sputum or throat cough swab or endolaryngeal suction
timeFrame: max. 24 months
Secondary outcomes:
measure: • Change in FEV 1.0 from day 0 to each visit
timeFrame: max. 24 months
measure: • Change in BMI from day 0 to each visit
timeFrame: max. 24 months
measure: • Number of exacerbations
timeFrame: max. 24 months
measure: • Number of days of illness in hospital and at home, i.e. out of school or work
timeFrame: max. 24 months
measure: • Control of use of antibiotics, especially anti-pseudomonas antibiotics -measured as days with antibiotic treatment
timeFrame: max. 24 months
measure: • Change in values of serologic tests for PA precipitins from day 0 to each visit (if applicable)
timeFrame: max. 24 months
measure: • Good tolerability and comparable number and quality of adverse events like placebo group
timeFrame: max. 24 months
measure: • Sputum or throat cough swab or endolaryngeal suction cultures for bacteria and fungi
timeFrame: max. 24 months
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: Medizinische Universität Innsbruck Department für Kinderheilkunde, Päd III CF Zentrum
city: Salzburg
country: Austria
facility: (SALK) Universitätsklink für Kinder- und Jugendheilkunde, Ambulanz für Allergien und Lungenerkrankungen
city: Brussels
country: Belgium
facility: Clinic of Pediatric Respiratory Diseases, Infectious Diseases and Travel Clinic
city: Brussels
country: Belgium
facility: Hôpital Universitaire Erasme, Service de Pneumologie
city: Leuven
country: Belgium
facility: University Hospital Leuven, Kindergeneeskunde
city: Berlin
country: Germany
facility: Charité, Christiane Herzog Zentrum
city: Bochum
country: Germany
facility: Klinikum der Ruhr Universität Bochum
city: Dresden
country: Germany
facility: University Dresden
city: Düsseldorf
country: Germany
facility: Universitätsklinikum Düsseldorf
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Frankfurt
country: Germany
facility: Klinikum der Johann-Wolfgang- Goethe Universität Frankfurt
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin
city: Gießen
country: Germany
facility: Universitätsklinikum Gießen und Marburg GmbH
city: Hannover
country: Germany
facility: MH Hannover (adults)
city: Hannover
country: Germany
facility: MH Hannover (children)
city: Jena
country: Germany
facility: Universitätsklinik Jena, Mukoviszidosezentrum
city: Kiel
country: Germany
facility: Städtisches Krankenhaus Kiel GmbH
city: Köln
country: Germany
facility: Universitätsklinik Köln
city: Mainz
country: Germany
facility: Universitätsklinikum Mainz
city: Tübingen
country: Germany
facility: Universitätsklinik Tübingen
city: Würzburg
country: Germany
facility: Universitäts-Kinderklinik Würzburg
city: Budapest
country: Hungary
facility: Heim Pal Hospital for Children
city: Budapest
country: Hungary
facility: Országos Korányi TBC és Pulm. Intézet, XIX. J fsz. Kronikus-CF care
city: Cork
country: Ireland
facility: Cork University Hospital
city: Dublin
country: Ireland
facility: Our Lady´s Children´s Hospital
city: Dublin
country: Ireland
facility: Tallagh Hospital
city: Limerick
country: Ireland
facility: Mid-Western Regional Hospital
city: Firenze
country: Italy
facility: Centro Regionale Toscano di Riferimento per la Fibrosi Cistica
city: Genova
country: Italy
facility: Istituto Ospedale Giannina Gaslini
city: Roma
country: Italy
facility: Centro Regionale Fibrosi Cisica Lazio
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitaria Integrata di Verona
city: Gdansk
country: Poland
facility: Szpital Dzieciecy Polanki im. Macieja Plazynskiego w Gdansku sp Z o.o. Poradnia Leczenia
city: Karpacz
country: Poland
facility: Centrum Medyczne Karpacz Spólka Akcyjna
city: Rabka - Zdrój
country: Poland
facility: NZOZ Sanatorium Cassia Villa Medica
city: Warsaw
country: Poland
facility: Instytut Matki i Dziecka Zaklad Mukowiscydozy
city: Łódź
country: Poland
facility: Wojewódzki Szpital Specjalistyczny im. M.Kopernika Ośrodek Pediatryczny im. dr J.Korczaka
city: Barakaldo (Vizcaya)
country: Spain
facility: Hospital Universitario Cruces Neumologia, Pediatric pulmonology
city: Barcelona
country: Spain
facility: Passeig Vall d´Hebron 119
city: Madrid
country: Spain
facility: Hospital Infantil la Paz Sección de Neumologia Pediátrica
city: Málaga
country: Spain
facility: Hospital Materno-Infantil Servicio de Pediatria
city: Stockholm
country: Sweden
facility: Karolinska University Hospital, Huddinge - CF-Centre
city: Uppsala
country: Sweden
facility: Uppsala University Childrens Hospital, Akademiska sjukhuset, CF center
Study officials:
affiliation: University Düsseldorf
name: Antje Schuster, Prof. Dr.
role: PRINCIPAL_INVESTIGATOR
References: