Cystic FibrosisPhase 2MUCOLAXNCT04458129
Polyethylene Glycol and Intestinal Inflammation in Cystic Fibrosis
A Phase 2 study testing Treatment with polyethylene glycol (Macrogol 4000) in people with Cystic Fibrosis, run by University Hospital, Bordeaux at 1 site in France.
Trial summary
The main objective of the study is to evaluate the effectiveness of polyethylene glycol treatment on intestinal inflammation in children with cystic fibrosis. In this test, a method adapted from the Fleming one-step scheme will be used. Read more about this study
- Who is it for?
- Ages 4 to 17 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Treatment with polyethylene glycol (Macrogol 4000)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteFrance
- Study dates
- 29 Sep 2020to 8 Jul 2022
- Phase
- Phase 2
- 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 4 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 4 years old and <18 years old ;
- Patient with cystic fibrosis (sweat test > 60 mmol/l and/or molecular biology identifying mutations in the CFTR gene) with associated pancreatic insufficiency (fecal elastase <100 µg/g);
- With a rapid calprotectin assay result via the IBDoc test (Bühlmann®) superior or equal to 250 µg/g;
- Person affiliated or benefiting from a social security scheme;
- Free, informed and written consent signed by the holders of parental authority and the investigator before any examination required by the research and oral and/or written consent by the participant (depending on his/her age).
Who cannot join
- Ongoing processing that can modulate the functionality of the CFTR (such as lumacaftor-ivacaftor protein therapy);
- Patient already on polyethylene glycol or other laxative within 3 months before the inclusion visit;
- Patient with diarrhea at inclusion (diarrhea will be defined as the presence of 3 or more stools / day in the 7 days prior to the inclusion visit);
- Acute viral or bacterial diarrhea in the month prior to the inclusion visit (associated with fever);
- Cure of oral or intravenous antibiotics or antifungals in the month preceding the collection of samples;
- Change in background treatment in the month prior to the inclusion visit (oral or inhaled corticosteroid therapy, azithromycin, inhaled antibiotic therapy, inhaled antifungal agent, proton pump inhibitors);
Show all 13Show fewer
- Taking probiotics in the month before the inclusion visit;
- Transplanted patient (on immunosuppressants);
- Patient with IBD or celiac disease;
- Patient with digestive perforation or risk of digestive perforation;
- Patient with ileus or suspicion of intestinal obstruction, symptomatic stenosis;
- History of hypersensitivity to macrogol or any of the excipients
- Holders of parental authority enjoying judicial protection.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effect of Polyethylene Glycol Treatment on Intestinal Inflammation Associated With Cystic Fibrosis Inchildren
The main objective of the study is to evaluate the effectiveness of polyethylene glycol treatment on intestinal inflammation in children with cystic fibrosis. In this test, a method adapted from the Fleming one-step scheme will be used. The success rate is measured by the proportion of patients with fecal calprotectin levels < 250 µg/g at 3 months after treatment initiation.
Read the detailed description
Cystic fibrosis is one of the most frequent serious genetic diseases in France (7000 patients). It is the consequence of mutations in the CFTR gene, encoding a protein involved in the hydro-electrolytic balance of secretions. Beyond the well-known lung damage in these patients, intestinal inflammation is present in the majority of patients.
While advances in the management of cystic fibrosis are increasing patient life expectancy, other issues are emerging, including the impact of this chronic intestinal inflammation on the nutritional status and high risk of digestive cancers.
Currently, no management is proposed to treat this intestinal inflammation. The use of laxatives to fluidize digestive secretions and restore a digestive ecosystem close to the healthy subject could constitute a new therapeutic approach to this intestinal inflammation, as previously shown in the mouse model of cystic fibrosis. However, to date, to the investigator's knowledge, no studies have evaluated the effect of laxative treatment on intestinal inflammation of cystic fibrosis in humans.
This study is a bi-centric, non-comparative, prospective study for a phase II trial according to a Fleming scheme.
Study participants will take a 3-month laxative treatment with polyethylene glycol for 3 months. In addition to the inclusion visit, a follow-up visit will take place at 3 months and 3 intermediate telephone calls will be made to ensure efficacy, tolerance and compliance.
- Conditions
- Cystic Fibrosis
- Being tested
- Treatment with polyethylene glycol (Macrogol 4000)
- Keywords
- Cystic Fibrosis, intestinal inflammation, fecal calprotectin, microbiota, children, polyethylene glycol
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Treatment with polyethylene glycol (Macrogol 4000)Drug
3-month treatment with polyethylene glycol (Macrogol 4000), powder for oral solution, in 4g and 10g sachets. Dosage of 0.7 g/kg/day, with a maximum dose of 20 g/day.
| Study group | Role | What they receive |
|---|---|---|
| Polyethylene glycol treatment | Experimental | Study participants will take a 3-month laxative treatment with polyethylene glycol for 3 months. Receives: Drug: Treatment with polyethylene glycol (Macrogol 4000) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Impact of a 3-month polyethylene glycol treatment on intestinal inflammation assessed by measurement of fecal calprotectin (µg / g) by an ELISA biological test
proportion of patients with fecal calprotectin <250 µg / g measured by an ELISA test 3 months after initiation of treatment with polyethylene glycol, testifying to the absence of intestinal inflammation or slight inflammation.
Measured: 3 months after the inclusion visit i.e. 3 months after the initiation of treatment with polyethylene glycol
Secondary outcomes (7)
Evolution of the digestive inflammatory response, assessed by measurement of fecal calprotectin (µg / g)
Evolution of the digestive inflammatory response, assessed by measurement of fecal calprotectin (µg / g) by an ELISA biological test between the initiation of treatment (D0) and 3 months of treatment with polyethylene glycol (M3)
Measured: Change from Baseline fecal calprotectin measurement at 3 months
Evolution of the digestive inflammatory response, assessed by Analysis of the expression of genes involved in the inflammatory and pro-oncogenic response
Analysis of the expression of genes involved in the inflammatory and pro-oncogenic response, using NanoString Technology and the nCounters® PanCancer Immune Profiling Panel
Measured: Change from Baseline measurement at 3 months
Evolution of the digestive symptoms, assessed by the JenAbdomen CF-score
The JenAbdomen CF-score has been described in the following publication Tabori H. et al. Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings. PloS One 12,e0174463 (2017).
the JenAbdomen CF-Score is based on a CF patient-reported outcome measure (PROM) that includes all relevant gastrointestinal symptoms and their impact on subjective quality of life
Measured: Change from Baseline measurement at 3 months
Evolution of the quality of life scores, assessed by the CFQ-R questionnaire
The CFQ-R questionnaire has been described in the following publication: Modi, A. C. \& Quittner, A. L. Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis. J. Pediatr. Psychol. 28, 535-545 (2003).
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific health-related quality of life (HRQOL) measure for children, adolescents and adults with cystic fibrosis (CF). It is a profile measure of HRQOL with several different domains. It has undergone extensive reliability and validity testing.
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) application calculates the score derived from the CFQ-R, on a 0-100 scale with higher scores indicating better HRQoL.
Measured: Change from Baseline measurement at 3 months
Evolution of the composition of the bacterial intestinal microbiota
The composition of the bacterial intestinal microbiota will be assessed by High-throughput sequencing and phylogenetic allocation of the bacterial flora (on MiSeq, from Illumina®)
Measured: Change from Baseline measurement at 3 months
Evolution of the composition of the fungal intestinal microbiota
The composition of the fungal intestinal microbiota will be assessed by high-throughput sequencing and phylogenetic allocation of the fungal flora (on MiSeq, from Illumina®)
Measured: Change from Baseline measurement at 3 months
Evolution of the pulmonary inflammation, assessed by the dosage of calprotectin in the sputum
Evolution of the Pulmonary inflammation will be assessed by measurement of salivary calprotectin (µg / g) by an ELISA biological test between the initiation of treatment (D0) and 3 months of treatment with polyethylene glycol (M3)
Measured: Change from Baseline measurement at 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2020
- Study start29 Sep 2020
- Primary completion8 Apr 2022
- Study completion8 Jul 2022
Why it stopped: Target number of inclusion not reached
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University Hospital, BordeauxThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Bi-centric, non-comparative, prospective, Phase II trial according to a Fleming scheme.
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Marie M MITTAINE, MD, Principal investigator, CHU de Toulouse - Hôpital des Enfants - Centre de Ressources et de Compétences de la Mucoviscidose (CRCM) pédiatrique
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04458129
- Sponsor's protocol ID
- CHUBX 2019/25
- Other IDs
- 2019-004958-29, 2019
- Acronym
- MUCOLAX
- Status last verified
- Jun 2020
- First submitted
- 30 Jun 2020
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Polyethylene Glycol Treatment on Intestinal Inflammation Associated With Cystic Fibrosis Inchildren
- ClinicalTrials.gov ID
- NCT04458129
- Acronym / sponsor ID
- MUCOLAX / CHUBX 2019/25
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, intestinal inflammation, fecal calprotectin, microbiota, children, polyethylene glycol
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Target number of inclusion not reached
- Enrolment
- 23 participants (actual)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Study start
- 29 Sep 2020 (actual)
- Primary completion
- 8 Apr 2022 (actual)
- Study completion
- 8 Jul 2022 (actual)
- First posted
- 7 Jul 2020 (actual)
- 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.
The main objective of the study is to evaluate the effectiveness of polyethylene glycol treatment on intestinal inflammation in children with cystic fibrosis. In this test, a method adapted from the Fleming one-step scheme will be used. The success rate is measured by the proportion of patients with fecal calprotectin levels < 250 µg/g at 3 months after treatment initiation.
Detailed description
Cystic fibrosis is one of the most frequent serious genetic diseases in France (7000 patients). It is the consequence of mutations in the CFTR gene, encoding a protein involved in the hydro-electrolytic balance of secretions. Beyond the well-known lung damage in these patients, intestinal inflammation is present in the majority of patients.
While advances in the management of cystic fibrosis are increasing patient life expectancy, other issues are emerging, including the impact of this chronic intestinal inflammation on the nutritional status and high risk of digestive cancers.
Currently, no management is proposed to treat this intestinal inflammation. The use of laxatives to fluidize digestive secretions and restore a digestive ecosystem close to the healthy subject could constitute a new therapeutic approach to this intestinal inflammation, as previously shown in the mouse model of cystic fibrosis. However, to date, to the investigator's knowledge, no studies have evaluated the effect of laxative treatment on intestinal inflammation of cystic fibrosis in humans.
This study is a bi-centric, non-comparative, prospective study for a phase II trial according to a Fleming scheme.
Study participants will take a 3-month laxative treatment with polyethylene glycol for 3 months. In addition to the inclusion visit, a follow-up visit will take place at 3 months and 3 intermediate telephone calls will be made to ensure efficacy, tolerance and compliance.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Bi-centric, non-comparative, prospective, Phase II trial according to a Fleming scheme.
| Arm | Type | Receives |
|---|---|---|
| Polyethylene glycol treatment | Experimental | Drug: Treatment with polyethylene glycol (Macrogol 4000)Study participants will take a 3-month laxative treatment with polyethylene glycol for 3 months. |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Age ≥ 4 years old and <18 years old ;
- Patient with cystic fibrosis (sweat test > 60 mmol/l and/or molecular biology identifying mutations in the CFTR gene) with associated pancreatic insufficiency (fecal elastase <100 µg/g);
- With a rapid calprotectin assay result via the IBDoc test (Bühlmann®) superior or equal to 250 µg/g;
- Person affiliated or benefiting from a social security scheme;
- Free, informed and written consent signed by the holders of parental authority and the investigator before any examination required by the research and oral and/or written consent by the participant (depending on his/her age).
Key exclusion criteria (13 listed)
- Ongoing processing that can modulate the functionality of the CFTR (such as lumacaftor-ivacaftor protein therapy);
- Patient already on polyethylene glycol or other laxative within 3 months before the inclusion visit;
- Patient with diarrhea at inclusion (diarrhea will be defined as the presence of 3 or more stools / day in the 7 days prior to the inclusion visit);
- Acute viral or bacterial diarrhea in the month prior to the inclusion visit (associated with fever);
- Cure of oral or intravenous antibiotics or antifungals in the month preceding the collection of samples;
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 |
|---|---|---|
| Treatment with polyethylene glycol (Macrogol 4000)3-month treatment with polyethylene glycol (Macrogol 4000), powder for oral solution, in 4g and 10g sachets. Dosage of 0.7 g/kg/day, with a maximum dose of 20 g/day. | Drug | Polyethylene glycol treatment |
Primary outcome
Impact of a 3-month polyethylene glycol treatment on intestinal inflammation assessed by measurement of fecal calprotectin (µg / g) by an ELISA biological test
Time frame: 3 months after the inclusion visit i.e. 3 months after the initiation of treatment with polyethylene glycol
proportion of patients with fecal calprotectin <250 µg / g measured by an ELISA test 3 months after initiation of treatment with polyethylene glycol, testifying to the absence of intestinal inflammation or slight inflammation.
7 secondary outcomes
Evolution of the digestive inflammatory response, assessed by measurement of fecal calprotectin (µg / g)
Time frame: Change from Baseline fecal calprotectin measurement at 3 months
Evolution of the digestive inflammatory response, assessed by Analysis of the expression of genes involved in the inflammatory and pro-oncogenic response
Time frame: Change from Baseline measurement at 3 months
Evolution of the digestive symptoms, assessed by the JenAbdomen CF-score
Time frame: Change from Baseline measurement at 3 months
Evolution of the quality of life scores, assessed by the CFQ-R questionnaire
Time frame: Change from Baseline measurement at 3 months
Evolution of the composition of the bacterial intestinal microbiota
Time frame: Change from Baseline measurement at 3 months
Evolution of the composition of the fungal intestinal microbiota
Time frame: Change from Baseline measurement at 3 months
Evolution of the pulmonary inflammation, assessed by the dosage of calprotectin in the sputum
Time frame: Change from Baseline measurement at 3 months
Results and safety
The study was stopped early (reason given: Target number of inclusion not reached). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
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.
Marie M MITTAINE, MD principal investigator
CHU de Toulouse - Hôpital des Enfants - Centre de Ressources et de Compétences de la Mucoviscidose (CRCM) pédiatrique
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Treatment with polyethylene glycol (Macrogol 4000) in people with Cystic Fibrosis, run by University Hospital, Bordeaux at 1 site in France.
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 5 inclusion and 13 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: stopped early, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: Target number of inclusion not reached). 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?
Impact of a 3-month polyethylene glycol treatment on intestinal inflammation assessed by measurement of fecal calprotectin (µg / g) by an ELISA biological test (time frame: 3 months after the inclusion visit i.e. 3 months after the initiation of treatment with polyethylene glycol). 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 22 Jun 2026.