Cystic Fibrosis in ChildrenNCT06284577
Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
A study testing Multistrain Probiotic in people with Cystic Fibrosis in Children, run by Oslo University Hospital at 1 site in Norway.
Trial summary
The aim of the present study is to investigate the effect of probiotic supplementation on GI related quality of life, through a randomised placebo-controlled clinical trial. Moreover, the invetigators wish to study CF microbiota and intestinal inflammation in the setting of probiotic supplementation and newly started… Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Cystic Fibrosis in Children
- What is being tested?
- Multistrain Probiotic
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteNorway
- Study dates
- 2 Jan 2024to 1 Jul 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- CFTR mutations eligible for treatment with ETI
- Age 2-18 years. Majority of patients will be 2-6 years of age as ETI was approved from 6 years of age in 2022, and will be available for children above 2 years from 2024.
- Included in the Norwegian CF Register and consented to participation in CF general research biobank
- Age 3-18 years
- CFTR modulator treatment naïve or treated with CFTR modulator for at least 6 months
- Included in the Norwegian CF Register and consented to participation in CF general research biobank
Who cannot join
- Other CFTR modulators commenced the last 6 months before inclusion
- Use of probiotics or prebiotics last 2 months
- Current pulmonary exacerbation
- CFTR modulators commenced the last 6 months before inclusion
- Use of probiotics or prebiotics last 2 months
- Current pulmonary exacerbation
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Norway1 site, 1 recruiting
- Recruiting
Oslo University Hospital
Oslo
Magnhild P Kolsgaard, PhD, 0047 41514045, uxpoma@ous-hf.no
Camilla Styffe Sæland, Master, 004793257798, casael@ous-hf.no
About this study
Official title: Probiotic Supplementation, Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
The aim of the present study is to investigate the effect of probiotic supplementation on GI related quality of life, through a randomised placebo-controlled clinical trial. Moreover, the invetigators wish to study CF microbiota and intestinal inflammation in the setting of probiotic supplementation and newly started treatment with a highly effective CF-specific treatment, elexacaftor-tezacaftor-ivacaftor (ETI). The proposed project has the potential to increase QoL and decrease GI morbidity in children with CF. If successful, the results of this study can contribute to alter the care of CF patients by including supplementation of probiotics in routine CF care. Morever, the study can provide much needed insights to GI microbiota and inflammation in pediatric CF patients.
Read the detailed description
The project targets pediatric patients with cystic fibrosis (CF), and has to work packages (WP). WP1 is an observational study, and WP2 is a randomised placebo-controlled clinical trial. The goal of the study is to investigate the effect of probiotics on pediatric CF patients' quality of life (QoL). Moreover the investigators wish to explore effects of both a highly effective CFTR modulator and probiotics on gut microbiota and intestinal inflammation.
The primary question it aims to answer are:
- Can probiotics improve GI related QoL in children with CF?
Secondary aims are to:
- Investigate GI microbiota and GI inflammation before and after commencement of the highly effective triple-combination elexacaftor-tezacaftor-ivacaftor (ETI)
- Explore GI microbiota before and after treatment with probiotics vs. placebo
- Study intestinal inflammation before and after treatment with probiotics vs. placebo
- Examine body composition and its relation to lung function
In WP1 participants will during routine examination before starting treatment with ETI be asked to deliver stool samples, and fill in QoL questionnaires. In WP2 participants will be randomized to intervention with probiotics or placebo, and the same parameters as in WP1 will also be collected.
- Conditions
- Cystic Fibrosis in Children
- Being tested
- Multistrain Probiotic, Placebo - maltodextrin
- Keywords
- Cystic Fibrosis, Children, Microbiota, Quality of life, Probiotic supplement, CFTR modulators, elexacaftor-tezacaftor-ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Multistrain ProbioticDietary supplement
Participants will recieve a multi-strain probiotic daily for 6 months
Placebo - maltodextrinDietary supplement
Participants will recieve placebo/ maltodextri daily for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Probiotics | Experimental | Participants will recieve a multi-strain probiotic daily for 6 months Receives: Dietary Supplement: Multistrain Probiotic |
| Placebo | Sham comparator | Participants will recieve maltodextrin daily for 6 months Receives: Dietary Supplement: Placebo - maltodextrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in GI related QoL using the questionnaire PedsQL GI
PEDsQL GI has been validated for use in the CF population. Participants will be scored before and 6 months after treatment start with ETI (WP2), and before and after 6 months treatment with probiotics vs placebo (WP2).
Measured: 0-6 months
Secondary outcomes (2)
Changes in microbiota
Stool samples for microbiota will be collected using a collection kit provided to the participants at recruitment, and then after 6 month of ETI treamtment (WP1), or probiotic/placebo (WP2). Microbiota analysis will be done using 16s rRNA amplicon sequencing and reduced metagenome sequencing.
Measured: 0-6 months
Changes in intestinal inflammation
Stool samples for gut inflammation markers will be collected using a collection kit provided to the participants at recruitment and after 6 months of ETI treamtment (WP1), or probiotic/placebo (WP2. Levels of calprotectin, neopterin and myeloperoxidase will be measured.
Measured: 0-6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Feb 2024
- Study start2 Jan 2024
- Primary completion1 Jun 2027 (estimated)
- Study completion1 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis in ChildrenStudies of the same condition, open and under way.
- More trials run by Oslo University HospitalThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Oslo University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Norwegian National Adivisory Unit on Rare Disorders
- Responsible party
- Principal investigator, Magnhild L. P. Kolsgaard, Oslo University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06284577
- Sponsor's protocol ID
- 677186
- Status last verified
- Jun 2024
- First submitted
- 12 Feb 2024
- Last updated
- 13 Jun 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Probiotic Supplementation, Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT06284577
- Acronym / sponsor ID
- 677186
- Conditions
- Cystic Fibrosis in Children
- Keywords
- Cystic Fibrosis, Children, Microbiota, Quality of life, Probiotic supplement, CFTR modulators, elexacaftor-tezacaftor-ivacaftor
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Oslo University HospitalUniversity, hospital or other
- Collaborators
- Norwegian National Adivisory Unit on Rare Disorders
- Study start
- 2 Jan 2024 (actual)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 1 Jul 2027 (estimated)
- First posted
- 29 Feb 2024 (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 aim of the present study is to investigate the effect of probiotic supplementation on GI related quality of life, through a randomised placebo-controlled clinical trial. Moreover, the invetigators wish to study CF microbiota and intestinal inflammation in the setting of probiotic supplementation and newly started treatment with a highly effective CF-specific treatment, elexacaftor-tezacaftor-ivacaftor (ETI). The proposed project has the potential to increase QoL and decrease GI morbidity in children with CF. If successful, the results of this study can contribute to alter the care of CF patients by including supplementation of probiotics in routine CF care. Morever, the study can provide much needed insights to GI microbiota and inflammation in pediatric CF patients.
Detailed description
The project targets pediatric patients with cystic fibrosis (CF), and has to work packages (WP). WP1 is an observational study, and WP2 is a randomised placebo-controlled clinical trial. The goal of the study is to investigate the effect of probiotics on pediatric CF patients' quality of life (QoL). Moreover the investigators wish to explore effects of both a highly effective CFTR modulator and probiotics on gut microbiota and intestinal inflammation.
The primary question it aims to answer are:
- Can probiotics improve GI related QoL in children with CF?
Secondary aims are to:
- Investigate GI microbiota and GI inflammation before and after commencement of the highly effective triple-combination elexacaftor-tezacaftor-ivacaftor (ETI)
- Explore GI microbiota before and after treatment with probiotics vs. placebo
- Study intestinal inflammation before and after treatment with probiotics vs. placebo
- Examine body composition and its relation to lung function
In WP1 participants will during routine examination before starting treatment with ETI be asked to deliver stool samples, and fill in QoL questionnaires. In WP2 participants will be randomized to intervention with probiotics or placebo, and the same parameters as in WP1 will also be collected.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Probiotics | Experimental | Dietary Supplement: Multistrain ProbioticParticipants will recieve a multi-strain probiotic daily for 6 months |
| Placebo | Sham comparator | Dietary Supplement: Placebo - maltodextrinParticipants will recieve maltodextrin daily for 6 months |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- CFTR mutations eligible for treatment with ETI
- Age 2-18 years. Majority of patients will be 2-6 years of age as ETI was approved from 6 years of age in 2022, and will be available for children above 2 years from 2024.
- Included in the Norwegian CF Register and consented to participation in CF general research biobank
- Age 3-18 years
- CFTR modulator treatment naïve or treated with CFTR modulator for at least 6 months
Key exclusion criteria (6 listed)
- Other CFTR modulators commenced the last 6 months before inclusion
- Use of probiotics or prebiotics last 2 months
- Current pulmonary exacerbation
- CFTR modulators commenced the last 6 months before inclusion
- Use of probiotics or prebiotics last 2 months
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 |
|---|---|---|
| Multistrain ProbioticParticipants will recieve a multi-strain probiotic daily for 6 months | Dietary supplement | Probiotics |
| Placebo - maltodextrinParticipants will recieve placebo/ maltodextri daily for 6 months | Dietary supplement | Placebo |
Primary outcome
Changes in GI related QoL using the questionnaire PedsQL GI
Time frame: 0-6 months
PEDsQL GI has been validated for use in the CF population. Participants will be scored before and 6 months after treatment start with ETI (WP2), and before and after 6 months treatment with probiotics vs placebo (WP2).
2 secondary outcomes
Changes in microbiota
Time frame: 0-6 months
Changes in intestinal inflammation
Time frame: 0-6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2024.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Multistrain Probiotic in people with Cystic Fibrosis in Children, run by Oslo University Hospital at 1 site in Norway.
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 6 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: looking for participants now, as of the sponsor's last update on 13 Jun 2024. 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 Norway. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in GI related QoL using the questionnaire PedsQL GI (time frame: 0-6 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 13 Jun 2024.