Cystic FibrosisPhase 4NCT03632525
Intravenous Iron in Adults With Cystic Fibrosis
A Phase 4 study testing Ferric carboxymaltose in people with Cystic Fibrosis and Iron-deficiency, run by University of Oxford at 1 site in United Kingdom.
Trial summary
This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, Iron-deficiency
- What is being tested?
- Ferric carboxymaltose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 22 Feb 2019to 26 Oct 2021
- Phase
- Phase 4
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years with established diagnosis of cystic fibrosis
- Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)
Who cannot join
- Urgent (<6 weeks) need for iron supplementation
- Active infection (currently requiring IV antibiotics)
- Previous intravenous iron supplementation (within last 4 months)
- Current oral iron supplementation
- Hypersensitivity to ferric carboxymaltose
- Active non-tuberculous mycobacterial pulmonary disease (as defined by ATS-IDSA criteria)
Show all 11Show fewer
- Liver failure
- Ferritin >300 μg/l or transferrin saturation >45%
- Pregnancy or breast feeding
- Previous transplantation
- Judged by member of trial team to be unlikely to comply with safety aspects of trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic Fibrosis
This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency.
Read the detailed description
Iron deficiency is common in adults with cystic fibrosis, and is associated with adverse outcomes. Oral iron supplementation is poorly tolerated and may be ineffective. In some centres, intravenous iron is used to correct iron deficiency, but concerns have been raised about the safety of this treatment in the setting of chronic airways infection. The investigators are therefore planning a pilot interventional cohort study examining the effects of intravenous iron in a group of adults with cystic fibrosis. Patients will be recruited in Oxford and studied prospectively over 16 weeks, with iron given at week 4. The primary focus of this single-centre pilot/feasibility study is safety, specifically in relation to infection.
- Conditions
- Cystic Fibrosis, Iron-deficiency
- Being tested
- Ferric carboxymaltose
- Medical subject headings
- Cystic Fibrosis, Anemia, Iron-Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ferric carboxymaltoseDrug
Also called Ferrinject
Single dose of 20 mg/kg ferric carboxymaltose (maximum 1000 mg for patients with haemoglobin <14 g/dL or 500 mg for patients with haemoglobin ≥14 g/dL).
| Study group | Role | What they receive |
|---|---|---|
| Intravenous iron | Experimental | All participants will receive a single dose of intravenous ferric carboxymaltose Receives: Drug: Ferric carboxymaltose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
New infective events are defined as any of:
- New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study)
- Clinical infection requiring IV antibiotics (as determined by clinical team)
- Admission to hospital for infection-related reason (as determined by clinical team)
- Significant deterioration in lung function (>10% fall in FEV1), not otherwise explained (as determined by clinical team)
Measured: 8 weeks
Secondary outcomes (13)
Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron
Infective events are defined as per primary outcome. Data relating to the 8 weeks prior to the 16-week prospective study period will be obtained from the medical records.
Measured: 16 weeks (plus 8 weeks of retrospective data collection from notes)
Change in number of antibiotic days
Assessed by review of clinical notes and patient self-reporting, to determine total number of days on which the patient was treated with antibiotics
Measured: 16 weeks
Change in abundance of sputum Pseudomonas
Assessed by quantitative PCR
Measured: 16 weeks
Change in sputum microbiological diversity
Assessed by microbiota analysis (16s rRNA gene sequencing)
Measured: 16 weeks
Change in exercise capacity (shuttle walk test)
Standardised and validated exercise test involving exercise at progressive intensity
Measured: 16 weeks
Change in lung function (FEV1)
Assessed by spirometry
Measured: 16 weeks
Change in arterial oxygen saturation
Assessed by non-invasive pulse oximetry
Measured: 16 weeks
Change in body mass index
Calculated by standard formula: BMI=weight/(height squared)
Measured: 16 weeks
Change in quality of life (CFQ-R questionnaire)
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a 48-item questionnaire that provides scores in twelve quality of life domains (physical functioning, vitality, emotional state, social limitations, role limitations, embarrassment, body image, eating disturbance, treatment constraints) and three symptom domains (respiratory, digestive, weight). Scores ranging from 0 to 100 are calculated for each quality of life domain, using a published method, where a higher score indicates a more favourable health status.
Measured: 16 weeks
Change in quality of life (SF-36 questionnaire)
The 36-item short form questionnaire (SF-36) provides scores in eight major domains of health (physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions). Each is scored on a scale from 0-100, where a higher value represents a more favourable health status. The domains may be combined to provide two summary scores, namely the 'physical component summary' and the 'mental component summary', each of which is also scored from 0-100. In calculating the respective summary scores, subscales related to physical or psychological health (as appropriate) are positively weighted, according to a published method.
Measured: 16 weeks
Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)
Assessed via changes in tricuspid regurgitant jet velocity
Measured: 16 weeks
Percentage of eligible patients entering and completing the study
Calculated based on number of eligible patients that enter and/or complete the study.
Measured: 16 weeks
Percentage of patient in whom each outcome is successfully measured
Calculated based on number of participating patients in whom each outcome is measured.
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2018
- Study start22 Feb 2019
- Primary completion27 Mar 2020
- Study completion26 Oct 2021
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 of OxfordThe 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Oxford
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Nick P Talbot, BMBCh DPhil, Principal investigator, University of Oxford
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03632525
- Sponsor's protocol ID
- PID12800
- Status last verified
- Sep 2022
- First submitted
- 21 Jun 2018
- Last updated
- 7 Sep 2022
- 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 7 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03632525
- Acronym / sponsor ID
- PID12800
- Conditions
- Cystic Fibrosis, Iron-deficiency
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of OxfordUniversity, hospital or other
- Study start
- 22 Feb 2019 (actual)
- Primary completion
- 27 Mar 2020 (actual)
- Study completion
- 26 Oct 2021 (actual)
- First posted
- 15 Aug 2018 (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.
This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency.
Detailed description
Iron deficiency is common in adults with cystic fibrosis, and is associated with adverse outcomes. Oral iron supplementation is poorly tolerated and may be ineffective. In some centres, intravenous iron is used to correct iron deficiency, but concerns have been raised about the safety of this treatment in the setting of chronic airways infection. The investigators are therefore planning a pilot interventional cohort study examining the effects of intravenous iron in a group of adults with cystic fibrosis. Patients will be recruited in Oxford and studied prospectively over 16 weeks, with iron given at week 4. The primary focus of this single-centre pilot/feasibility study is safety, specifically in relation to infection.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intravenous iron | Experimental | Drug: Ferric carboxymaltoseAll participants will receive a single dose of intravenous ferric carboxymaltose |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Age ≥18 years with established diagnosis of cystic fibrosis
- Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)
Key exclusion criteria (11 listed)
- Urgent (<6 weeks) need for iron supplementation
- Active infection (currently requiring IV antibiotics)
- Previous intravenous iron supplementation (within last 4 months)
- Current oral iron supplementation
- Hypersensitivity to ferric carboxymaltose
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 |
|---|---|---|
| Ferric carboxymaltoseAlso: FerrinjectSingle dose of 20 mg/kg ferric carboxymaltose (maximum 1000 mg for patients with haemoglobin <14 g/dL or 500 mg for patients with haemoglobin ≥14 g/dL). | Drug | Intravenous iron |
Primary outcome
Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
Time frame: 8 weeks
New infective events are defined as any of: 1. New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study) 2. Clinical infection requiring IV antibiotics (as determined by clinical team) 3. Admission to hospital for infection-related reason (as determined by clinical team) 4. Significant deterioration in lung function (>10% fall in FEV1), not otherwise explained (as determined by clinical team)
13 secondary outcomes
Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron
Time frame: 16 weeks (plus 8 weeks of retrospective data collection from notes)
Change in number of antibiotic days
Time frame: 16 weeks
Change in abundance of sputum Pseudomonas
Time frame: 16 weeks
Change in sputum microbiological diversity
Time frame: 16 weeks
Change in exercise capacity (shuttle walk test)
Time frame: 16 weeks
Change in lung function (FEV1)
Time frame: 16 weeks
Change in arterial oxygen saturation
Time frame: 16 weeks
Change in body mass index
Time frame: 16 weeks
Change in quality of life (CFQ-R questionnaire)
Time frame: 16 weeks
Change in quality of life (SF-36 questionnaire)
Time frame: 16 weeks
Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)
Time frame: 16 weeks
Percentage of eligible patients entering and completing the study
Time frame: 16 weeks
Percentage of patient in whom each outcome is successfully measured
Time frame: 16 weeks
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 7 Sep 2022.
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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.
Nick P Talbot, BMBCh DPhil principal investigator
University of Oxford
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Ferric carboxymaltose in people with Cystic Fibrosis and Iron-deficiency, run by University of Oxford at 1 site in United Kingdom.
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 2 inclusion and 11 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 7 Sep 2022. 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 United Kingdom. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron (time frame: 8 weeks). 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 7 Sep 2022.