Non Cystic Fibrosis Bronchiectasis in ChildrenPhase 3AZI-STOPNCT02531984
The Efficacy of Azithromycin in Treating Children With Non Cystic Fibrosis Bronchiectasis
A Phase 3 study testing Azithromycin in people with Non Cystic Fibrosis Bronchiectasis in Children, run by Assistance Publique Hopitaux De Marseille.
Trial summary
Bronchiectasis is characterized by a permanent and abnormal dilatation of a part of the bronchial tree. An accumulation of mucus in the respiratory tract ensues, resulting in frequent bacterial infections and eventual destruction of the lungs. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Non Cystic Fibrosis Bronchiectasis in Children
- What is being tested?
- Azithromycin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- Aug 2015to Aug 2018
- 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 3 to 17 years
- No healthy volunteers
- Child
Who may be able to join
- Presenting with non cystic fibrosis bronchiectasis documented by chest CT scan , diagnosed since at least 2 years
- Already treated with azithromycin for at least 6 months
- Presented no respiratory exacerbation since one month
- Benefit from health insurance
- Have signed a consent (if appropriate age )
- Parents or legal guardian have given their written consent
Who cannot join
- Patients presenting a history of allergic reaction to azithromycin, érythromycine, another macrolide or Ketolide or an excipient of Zithromax
- Patients treated with dihydroergotamine or ergotamine , cisapride, colchicine
- Patients presenting severe hepatic failure
- Patients who are unlikely to adhere to the protocol and hence participate in the entire study ( as judged by the investigator )
- Patients who have not signed the protocol
- Patients whose parents are unable to understand the purpose and conditions of the study or are unable to give their consent.
- Patients already included in another clinical trial or are in an exclusion period from a previous clinical trial
- Patients whose ECG shows a QTc > 450 msec ( Zithromax is contraindicated )
About this study
Official title: Open Trial With Randomized Withdrawal of Treatment, to Evaluate the Efficacy of Azithromycin in the Treatment of Children With Non Cystic Fibrosis Bronchiectasis ( AZI-STOP Study )
Bronchiectasis is characterized by a permanent and abnormal dilatation of a part of the bronchial tree. An accumulation of mucus in the respiratory tract ensues, resulting in frequent bacterial infections and eventual destruction of the lungs. Clinically, patients present with a chronic productive cough and episodes of acute respiratory exacerbations. Chronic respiratory failure can follow. Although its prevalence is largely unknown, bronchiectasis is considered to be a rare and orphan disease. There are numerous causes for this disease: sequelae of respiratory infections, immunodeficiency, genetic diseases like cystic fibrosis, primary ciliary dyskinesia….The focus of this study will be on non cystic fibrosis bronchiectasis in children.
Due to a lack of pediatric clinical trials, the management of children with this disease is widely based on the management of adults or patients with cystic fibrosis or pan-bronchiolitis. The treatment is based on respiratory physiotherapy, prevention of infections, administration of inhaled corticosteroids and anti biotherapy for acute exacerbations.
Read more
Recently, some studies have demonstrated the efficacy of a family of antibiotics, the macrolides, in the treatment of cystic fibrosis or pan-bronchiolitis in children. Indeed, taken 3 times a week during a long period of time, the macrolides, and specifically the azithromycin have shown some anti-inflammatory and tissue repairing properties, in addition to their antimicrobial properties.
Moreover, several studies conducted in the adult population have shown that the use of azithromycin has led to significant reduction in the frequency of respiratory exacerbations as well as an improvement in the quality of life.
The efficacy of azithromycin in these respiratory diseases has led to enlarge its use for the long term treatment of bronchiectasis. Yet, this type of treatment has no marketing authorization application.
No studies have been conducted in children, but individual examination of patients with bronchiectasis treated with azithromycin suggests an improvement of their symptoms.
Thus, the use of azithromycin in the treatment of bronchiectasis in children seems to be a promising therapy. However, its efficacy needs to be demonstrated by clinical trials led on a pediatric population with an adequate number of patients and a strong methodology in order to ensure validity and reliability of the results.
Therefore the investigators decided to conduct a comparative, prospective multicenter randomized study in this population.
This study intends to include 100 patients already treated by azithromycin for at least 6 months. After inclusion, they will be randomized in two groups of 50 patients each. The first group will continue the treatment and the other one will discontinue it.
The patients will be followed from the inclusion period (M0) until the onset of the first exacerbation for a maximum of 6 months (M6), with an intermediate health care visit after 3 months (M3).
In case of exacerbation, a health care visit will be scheduled; the patient will receive the appropriate treatment and he will be suspended from the study.
The duration of follow up for each patient is dictated by the occurrence of the first respiratory exacerbation, a maximum of 6 months .The total inclusion period is 12 months and the total duration of the study is 18 months.
- Being tested
- Azithromycin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzithromycinDrug
| Study group | Role | What they receive |
|---|---|---|
| Withdrawal of Azithromycin treatment | Experimental | Not described Receives: Drug: Azithromycin |
| ongoing Azithromycin treatment | Other | Not described Receives: Drug: Azithromycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time of onset of the first respiratory exacerbation in the two groups of patients: those who continued treatment with azithromycin and those who stopped the treatment
Exacerbations are defined by a combination of major and minor clinical criteria (Kapur et al, 2012) and the need for an antibiotic therapy for lower respiratory tract in case of acute infection. These criteria are evaluated by parents with the help of a daily diary; respiratory exacerbation is confirmed by a clinical examination.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2015 (estimated)
- Study startAug 2015
- Primary completionAug 2017 (estimated)
- Study completionAug 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non Cystic Fibrosis Bronchiectasis in ChildrenStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Urielle DESALBRES, Study director, Assistance Publique Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02531984
- Sponsor's protocol ID
- 2014-26
- Other IDs
- RC12_3643
- Acronym
- AZI-STOP
- Status last verified
- Aug 2015
- First submitted
- 21 Aug 2015
- Last updated
- 25 Aug 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Trial With Randomized Withdrawal of Treatment, to Evaluate the Efficacy of Azithromycin in the Treatment of Children With Non Cystic Fibrosis Bronchiectasis ( AZI-STOP Study )
- ClinicalTrials.gov ID
- NCT02531984
- Acronym / sponsor ID
- AZI-STOP / 2014-26
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- Aug 2015
- Primary completion
- Aug 2017 (estimated)
- Study completion
- Aug 2018 (estimated)
- First posted
- 25 Aug 2015 (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.
Bronchiectasis is characterized by a permanent and abnormal dilatation of a part of the bronchial tree. An accumulation of mucus in the respiratory tract ensues, resulting in frequent bacterial infections and eventual destruction of the lungs. Clinically, patients present with a chronic productive cough and episodes of acute respiratory exacerbations. Chronic respiratory failure can follow. Although its prevalence is largely unknown, bronchiectasis is considered to be a rare and orphan disease. There are numerous causes for this disease: sequelae of respiratory infections, immunodeficiency, genetic diseases like cystic fibrosis, primary ciliary dyskinesia….The focus of this study will be on non cystic fibrosis bronchiectasis in children.
Due to a lack of pediatric clinical trials, the management of children with this disease is widely based on the management of adults or patients with cystic fibrosis or pan-bronchiolitis. The treatment is based on respiratory physiotherapy, prevention of infections, administration of inhaled corticosteroids and anti biotherapy for acute exacerbations.
Recently, some studies have demonstrated the efficacy of a family of antibiotics, the macrolides, in the treatment of cystic fibrosis or pan-bronchiolitis in children. Indeed, taken 3 times a week during a long period of time, the macrolides, and specifically the azithromycin have shown some anti-inflammatory and tissue repairing properties, in addition to their antimicrobial properties.
Moreover, several studies conducted in the adult population have shown that the use of azithromycin has led to significant reduction in the frequency of respiratory exacerbations as well as an improvement in the quality of life.
The efficacy of azithromycin in these respiratory diseases has led to enlarge its use for the long term treatment of bronchiectasis. Yet, this type of treatment has no marketing authorization application.
No studies have been conducted in children, but individual examination of patients with bronchiectasis treated with azithromycin suggests an improvement of their symptoms.
Thus, the use of azithromycin in the treatment of bronchiectasis in children seems to be a promising therapy. However, its efficacy needs to be demonstrated by clinical trials led on a pediatric population with an adequate number of patients and a strong methodology in order to ensure validity and reliability of the results.
Therefore the investigators decided to conduct a comparative, prospective multicenter randomized study in this population.
This study intends to include 100 patients already treated by azithromycin for at least 6 months. After inclusion, they will be randomized in two groups of 50 patients each. The first group will continue the treatment and the other one will discontinue it.
The patients will be followed from the inclusion period (M0) until the onset of the first exacerbation for a maximum of 6 months (M6), with an intermediate health care visit after 3 months (M3).
In case of exacerbation, a health care visit will be scheduled; the patient will receive the appropriate treatment and he will be suspended from the study.
The duration of follow up for each patient is dictated by the occurrence of the first respiratory exacerbation, a maximum of 6 months .The total inclusion period is 12 months and the total duration of the study is 18 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Withdrawal of Azithromycin treatment | Experimental | Drug: Azithromycin |
| ongoing Azithromycin treatment | Other | Drug: Azithromycin |
Who may qualify
- Ages
- 3 to 17 years
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Presenting with non cystic fibrosis bronchiectasis documented by chest CT scan , diagnosed since at least 2 years
- Already treated with azithromycin for at least 6 months
- Presented no respiratory exacerbation since one month
- Benefit from health insurance
- Have signed a consent (if appropriate age )
Key exclusion criteria (8 listed)
- Patients presenting a history of allergic reaction to azithromycin, érythromycine, another macrolide or Ketolide or an excipient of Zithromax
- Patients treated with dihydroergotamine or ergotamine , cisapride, colchicine
- Patients presenting severe hepatic failure
- Patients who are unlikely to adhere to the protocol and hence participate in the entire study ( as judged by the investigator )
- Patients who have not signed the protocol
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 |
|---|---|---|
| Azithromycin | Drug | Withdrawal of Azithromycin treatment, ongoing Azithromycin treatment |
Primary outcome
Time of onset of the first respiratory exacerbation in the two groups of patients: those who continued treatment with azithromycin and those who stopped the treatment
Time frame: 6 months
Exacerbations are defined by a combination of major and minor clinical criteria (Kapur et al, 2012) and the need for an antibiotic therapy for lower respiratory tract in case of acute infection. These criteria are evaluated by parents with the help of a daily diary; respiratory exacerbation is confirmed by a clinical examination.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2015.
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Non Cystic Fibrosis Bronchiectasis
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.
Urielle DESALBRES study director
Assistance Publique Hôpitaux de Marseille
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Azithromycin in people with Non Cystic Fibrosis Bronchiectasis in Children, run by Assistance Publique Hopitaux De Marseille.
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 6 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 25 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
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?
Time of onset of the first respiratory exacerbation in the two groups of patients: those who continued treatment with azithromycin and those who stopped the treatment (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Aug 2015.