Cystic FibrosisPhase 3CF-IDEANCT01100892
Cystic Fibrosis - Insulin Deficiency, Early Action
A Phase 3 study testing Once-daily insulin detemir in people with Cystic Fibrosis and Diabetes, run by Sydney Children's Hospitals Network at 6 sites in 2 countries.
Trial summary
Cystic Fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and eventually diabetes. Read more about this study
- Who is it for?
- Ages 5 to 19 years
- Conditions the study is for
- Cystic Fibrosis, Diabetes
- What is being tested?
- Once-daily insulin detemir
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 6 sitesin 2 countries
- Study dates
- Dec 2010to Feb 2023
- 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 5 to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with CF aged >=5 yrs attending one of the study sites.
- CFID1 or CFID2 (defined as BGmax >=8.2 and BG120 <11.1mmol/l on OGTT performed within the last 6 months, when respiratory function stable as judged by the treating respiratory team, not taking fluoroquinolone antibiotics, and not taking systemic glucocorticoids).
Who cannot join
- Cystic Fibrosis Related Diabetes, defined as CFID3 (BG120 >11.1mmol/L) or CFID4 (fasting BG >7mmol/L). Such patients will be offered insulin treatment as standard clinical care.
- Unstable respiratory disease (hospital admission for treatment of respiratory exacerbation within the last month).
- Treatment with systemic glucocorticoids of more than 1 month duration, within the last 12 months.
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
Australia5 sites
Women's and Children's Hospital
Adelaide, South Australia, 5006
Lady Cilento Children's Hospital
Brisbane, Queensland, 4101
John Hunter Children's Hospital
New Lambton, New South Wales, 2310
Sydney Children's Hospital
Randwick, New South Wales, 2031
Children's Hospital at Westmead
Westmead, New South Wales, 2145
United States1 site
Children's Hospital Colorado
Denver, Colorado, 80045
About this study
Cystic Fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and eventually diabetes. The CF-IDEA trial (Cystic Fibrosis - Insulin Deficiency, Early Action) will determine whether starting insulin treatment before the onset of diabetes (earlier than current practice) will improve the health of children with CF by improving body weight and lung function.
Read the detailed description
As well as progressive lung disease, patients with Cystic Fibrosis (CF) suffer pancreatic destruction, leading to slow but progressive insulin deficiency. Deficiency of insulin, a powerful anabolic hormone, causes accelerated decline of weight and lung function (important predictors of early mortality in CF).
We analysed Oral Glucose Tolerance Tests sampled every 30 mins and defined stages of CF Insulin Deficiency (CFID) as early glucose abnormalities, CFID1 (BGmax >=8.2 and <11.1mmol/L) and CFID2 (BGmax >=11.1 and BG120min <11.1), progressing to diabetes without fasting hyperglycaemia (CFID3), and finally to diabetes with fasting hyperglycaemia (CFID4). Currently insulin treatment is standard only for CFID3 and 4, but we have data showing that the earlier stages (CFID1 and 2) are also associated with declining weight and lung function.
In the CF-IDEA Trial, subjects with CF aged >=5 years with early glucose abnormalities (CFID1 or 2) will be randomised to once-daily insulin detemir (Levemir) for 12 months, or to observation only. We aim to determine whether starting insulin earlier than current practice will prevent decline in weight and lung function, reduce frequency of hospitalisation, improve quality of life, and slow progression through CFID categories.
Our pilot studies using once-daily Levemir in children with CFID1 and 2 found that this simple insulin regimen (rather than multiple daily injections) was well accepted by patients, with minimal hypoglycaemia, and resulted in significant weight gain and improved lung function (compared with 12 months prior to insulin). Sample size calculations for the CF-IDEA Trial are based on our pilot studies. When 70-80% of patients have completed the protocol, the study statistician will perform an interim analysis (blinded to the other investigators) to check the original power calculations.
Stages of CF Insulin Deficiency:
CFID1 Peak BG on OGTT >=8.2mmol/L and <11.1mmol/l.
CFID2 Peak BG on OGTT >=11.1mmol/L and 120 minute BG <11.1.
CFID3 120 minute BG on OGTT >=11.1mmol/L.
CFID4 Fasting hyperglycemia (Fasting BG >=7mmol/L).
- Conditions
- Cystic Fibrosis, Diabetes
- Being tested
- Once-daily insulin detemir
- Keywords
- Pediatrics, Pancreas
- Medical subject headings
- Cystic Fibrosis, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Once-daily insulin detemirDrug
Also called Levemir
Insulin detemir is a long-acting insulin analog. Starting dose 0.1 units/kg/day (titrated according to the results of home blood glucose monitoring).
| Study group | Role | What they receive |
|---|---|---|
| Control group | No intervention | Observation only. Does not receive once-daily insulin detemir. |
| Once-daily insulin detemir | Experimental | Once-daily insulin detemir Receives: Drug: Once-daily insulin detemir |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Weight SDS (Standard Deviation Score)
Measured: 12 months
Change in lung function (FEV1, FVC)
Measured: 12 months
Secondary outcomes (8)
Reduced rate of decline in glycaemic category, comparing OGTT at baseline and 12 months.
OGTT = Oral Glucose Tolerance Test
Measured: 12 months
Reduced frequency of hospitalisation for acute respiratory illness
Measured: 12 months
Change in glycaemic status assessed by HbA1c and CGM
CGM = Continuous Glucose Monitoring
Measured: 12 months
Body composition by DEXA. Patients at CHW will also have pQCT.
DEXA = Dual Energy X-ray Absorptiometry
pQCT = peripheral Quantitative Computed Tomography
Measured: 12 months
Change in Grip-strength
Measured: 12 months
Improved quality of life, measured by a validated CF QOL questionnaire
Measured: 12 months
Bacterial colonisation of sputum
Measured: 12 months
Change in effort-dependent lung function: MIP, MEP, SnIP
MIP = Mouth Inspiratory Pressure
MEP = Mouth Expiratory Pressure
SnIP = Sniff Nasal Inspiratory Pressure
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2010 (estimated)
- Study startDec 2010
- Primary completionFeb 2023
- Study completionFeb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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 (estimated)
Sponsor and investigators
- Lead sponsor
- Sydney Children's Hospitals Network
- Sponsor type
- University, hospital or other
- Collaborators
- John Hunter Children's Hospital, Lady Cilento Children's Hospital, Brisbane, Women's and Children's Hospital, Adelaide, Children's Hospital Colorado
- Responsible party
- Principal investigator, Dr Charles Verge, Sydney Children's Hospitals Network
- Investigators
- Charles Verge, MBBS PhD, Principal investigator, Endocrinology, Sydney Children's Hospital Randwick; School of Women's and Children's Health, University of NSW
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01100892
- Sponsor's protocol ID
- CF-IDEA
- Acronym
- CF-IDEA
- Status last verified
- May 2023
- First submitted
- 31 Mar 2010
- Last updated
- 31 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Hameed S, Morton JR, Jaffe A, Field PI, Belessis Y, Yoong T, Katz T, Verge CF. Early glucose abnormalities in cystic fibrosis are preceded by poor weight gain. Diabetes Care. 2010 Feb;33(2):221-6. doi: 10.2337/dc09-1492. Epub 2009 Nov 12.
PubMed 19910502Hameed S, Morton JR, Field PI, Belessis Y, Yoong T, Katz T, Woodhead HJ, Walker JL, Neville KA, Campbell TA, Jaffe A, Verge CF. Once daily insulin detemir in cystic fibrosis with insulin deficiency. Arch Dis Child. 2012 May;97(5):464-7. doi: 10.1136/adc.2010.204636. Epub 2011 Apr 14.
PubMed 21493664Hameed S, Jaffe A, Verge CF. Cystic fibrosis related diabetes (CFRD)--the end stage of progressive insulin deficiency. Pediatr Pulmonol. 2011 Aug;46(8):747-60. doi: 10.1002/ppul.21495. Epub 2011 May 27.
PubMed 21626717Hameed S, Barnes EH, Briody J, Wainwright CE, Hilton J, Field PI, Tai A, Belessis Y, Chan CL, Selvadurai H, Prentice B, Katz T, McMahon SK, Neylan M, Pena A, Jaffe A, Verge CF. Insulin for early glycaemic abnormality in children with cystic fibrosis without cystic fibrosis-related diabetes (CF-IDEA): a randomised controlled trial. Lancet Child Adolesc Health. 2025 Jun;9(6):371-382. doi: 10.1016/S2352-4642(25)00099-9.
PubMed 40379429
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cystic Fibrosis - Insulin Deficiency, Early Action
- ClinicalTrials.gov ID
- NCT01100892
- Acronym / sponsor ID
- CF-IDEA / CF-IDEA
- Conditions
- Cystic Fibrosis, Diabetes
- Keywords
- Pediatrics, Pancreas
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Sydney Children's Hospitals NetworkUniversity, hospital or other
- Collaborators
- John Hunter Children's Hospital, Lady Cilento Children's Hospital, Brisbane, Women's and Children's Hospital, Adelaide, Children's Hospital Colorado
- Study start
- Dec 2010 (actual)
- Primary completion
- Feb 2023 (actual)
- Study completion
- Feb 2023 (actual)
- First posted
- 9 Apr 2010 (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.
Cystic Fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and eventually diabetes. The CF-IDEA trial (Cystic Fibrosis - Insulin Deficiency, Early Action) will determine whether starting insulin treatment before the onset of diabetes (earlier than current practice) will improve the health of children with CF by improving body weight and lung function.
Detailed description
As well as progressive lung disease, patients with Cystic Fibrosis (CF) suffer pancreatic destruction, leading to slow but progressive insulin deficiency. Deficiency of insulin, a powerful anabolic hormone, causes accelerated decline of weight and lung function (important predictors of early mortality in CF).
We analysed Oral Glucose Tolerance Tests sampled every 30 mins and defined stages of CF Insulin Deficiency (CFID) as early glucose abnormalities, CFID1 (BGmax >=8.2 and <11.1mmol/L) and CFID2 (BGmax >=11.1 and BG120min <11.1), progressing to diabetes without fasting hyperglycaemia (CFID3), and finally to diabetes with fasting hyperglycaemia (CFID4). Currently insulin treatment is standard only for CFID3 and 4, but we have data showing that the earlier stages (CFID1 and 2) are also associated with declining weight and lung function.
In the CF-IDEA Trial, subjects with CF aged >=5 years with early glucose abnormalities (CFID1 or 2) will be randomised to once-daily insulin detemir (Levemir) for 12 months, or to observation only. We aim to determine whether starting insulin earlier than current practice will prevent decline in weight and lung function, reduce frequency of hospitalisation, improve quality of life, and slow progression through CFID categories.
Our pilot studies using once-daily Levemir in children with CFID1 and 2 found that this simple insulin regimen (rather than multiple daily injections) was well accepted by patients, with minimal hypoglycaemia, and resulted in significant weight gain and improved lung function (compared with 12 months prior to insulin). Sample size calculations for the CF-IDEA Trial are based on our pilot studies. When 70-80% of patients have completed the protocol, the study statistician will perform an interim analysis (blinded to the other investigators) to check the original power calculations.
Stages of CF Insulin Deficiency:
CFID1 Peak BG on OGTT >=8.2mmol/L and <11.1mmol/l.
CFID2 Peak BG on OGTT >=11.1mmol/L and 120 minute BG <11.1.
CFID3 120 minute BG on OGTT >=11.1mmol/L.
CFID4 Fasting hyperglycemia (Fasting BG >=7mmol/L).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control group | No intervention | –Observation only. Does not receive once-daily insulin detemir. |
| Once-daily insulin detemir | Experimental | Drug: Once-daily insulin detemirOnce-daily insulin detemir |
Who may qualify
- Ages
- 5 to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patients with CF aged >=5 yrs attending one of the study sites.
- CFID1 or CFID2 (defined as BGmax >=8.2 and BG120 <11.1mmol/l on OGTT performed within the last 6 months, when respiratory function stable as judged by the treating respiratory team, not taking fluoroquinolone antibiotics, and not taking systemic glucocorticoids).
Key exclusion criteria (3 listed)
- Cystic Fibrosis Related Diabetes, defined as CFID3 (BG120 >11.1mmol/L) or CFID4 (fasting BG >7mmol/L). Such patients will be offered insulin treatment as standard clinical care.
- Unstable respiratory disease (hospital admission for treatment of respiratory exacerbation within the last month).
- Treatment with systemic glucocorticoids of more than 1 month duration, within the last 12 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 |
|---|---|---|
| Once-daily insulin detemirAlso: LevemirInsulin detemir is a long-acting insulin analog. Starting dose 0.1 units/kg/day (titrated according to the results of home blood glucose monitoring). | Drug | Once-daily insulin detemir |
Primary outcome
Change in Weight SDS (Standard Deviation Score)
Time frame: 12 months
Change in lung function (FEV1, FVC)
Time frame: 12 months
8 secondary outcomes
Reduced rate of decline in glycaemic category, comparing OGTT at baseline and 12 months.
Time frame: 12 months
Reduced frequency of hospitalisation for acute respiratory illness
Time frame: 12 months
Change in glycaemic status assessed by HbA1c and CGM
Time frame: 12 months
Body composition by DEXA. Patients at CHW will also have pQCT.
Time frame: 12 months
Change in Grip-strength
Time frame: 12 months
Improved quality of life, measured by a validated CF QOL questionnaire
Time frame: 12 months
Bacterial colonisation of sputum
Time frame: 12 months
Change in effort-dependent lung function: MIP, MEP, SnIP
Time frame: 12 months
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 31 May 2023.
- Background: PubMed 19910502Hameed S, Morton JR, Jaffe A, Field PI, Belessis Y, Yoong T, Katz T, Verge CF. Early glucose abnormalities in cystic fibrosis are preceded by poor weight gain. Diabetes Care. 2010 Feb;33(2):221-6. doi: 10.2337/dc09-1492. Epub 2009 Nov 12.
- Background: PubMed 21493664Hameed S, Morton JR, Field PI, Belessis Y, Yoong T, Katz T, Woodhead HJ, Walker JL, Neville KA, Campbell TA, Jaffe A, Verge CF. Once daily insulin detemir in cystic fibrosis with insulin deficiency. Arch Dis Child. 2012 May;97(5):464-7. doi: 10.1136/adc.2010.204636. Epub 2011 Apr 14.
- Background: PubMed 21626717Hameed S, Jaffe A, Verge CF. Cystic fibrosis related diabetes (CFRD)--the end stage of progressive insulin deficiency. Pediatr Pulmonol. 2011 Aug;46(8):747-60. doi: 10.1002/ppul.21495. Epub 2011 May 27.
- Derived from this study: PubMed 40379429Hameed S, Barnes EH, Briody J, Wainwright CE, Hilton J, Field PI, Tai A, Belessis Y, Chan CL, Selvadurai H, Prentice B, Katz T, McMahon SK, Neylan M, Pena A, Jaffe A, Verge CF. Insulin for early glycaemic abnormality in children with cystic fibrosis without cystic fibrosis-related diabetes (CF-IDEA): a randomised controlled trial. Lancet Child Adolesc Health. 2025 Jun;9(6):371-382. doi: 10.1016/S2352-4642(25)00099-9.
- 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. This study is not recruiting at the moment.
Charles Verge, MBBS PhD principal investigator
Endocrinology, Sydney Children's Hospital Randwick; School of Women's and Children's Health, University of NSW
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Once-daily insulin detemir in people with Cystic Fibrosis and Diabetes, run by Sydney Children's Hospitals Network at 6 sites in 2 countries.
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 3 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 31 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Australia, United States. 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 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?
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?
Change in Weight SDS (Standard Deviation Score) (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01100892, which also lists 4 linked publications.
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 31 May 2023.