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Cystic Fibrosis - Insulin Deficiency, Early Action (CF-IDEA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01100892
Recruitment Status : Recruiting
First Posted : April 9, 2010
Last Update Posted : September 16, 2021
John Hunter Children's Hospital
Lady Cilento Children's Hospital, Brisbane
Women's and Children's Hospital, Adelaide
Children's Hospital Colorado
Information provided by (Responsible Party):
Dr Charles Verge, Sydney Children's Hospitals Network

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Diabetes Drug: Once-daily insulin detemir Phase 3

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).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Cystic Fibrosis - Insulin Deficiency, Early Action
Actual Study Start Date : December 2010
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
No Intervention: Control group
Observation only. Does not receive once-daily insulin detemir.
Experimental: Once-daily insulin detemir
Once-daily insulin detemir
Drug: Once-daily insulin detemir
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).
Other Name: Levemir

Primary Outcome Measures :
  1. Change in Weight SDS (Standard Deviation Score) [ Time Frame: 12 months ]
  2. Change in lung function (FEV1, FVC) [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. Reduced rate of decline in glycaemic category, comparing OGTT at baseline and 12 months. [ Time Frame: 12 months ]
    OGTT = Oral Glucose Tolerance Test

  2. Reduced frequency of hospitalisation for acute respiratory illness [ Time Frame: 12 months ]
  3. Change in glycaemic status assessed by HbA1c and CGM [ Time Frame: 12 months ]
    CGM = Continuous Glucose Monitoring

  4. Body composition by DEXA. Patients at CHW will also have pQCT. [ Time Frame: 12 months ]

    DEXA = Dual Energy X-ray Absorptiometry

    pQCT = peripheral Quantitative Computed Tomography

  5. Change in Grip-strength [ Time Frame: 12 months ]
  6. Improved quality of life, measured by a validated CF QOL questionnaire [ Time Frame: 12 months ]
  7. Bacterial colonisation of sputum [ Time Frame: 12 months ]
  8. Change in effort-dependent lung function: MIP, MEP, SnIP [ Time Frame: 12 months ]

    MIP = Mouth Inspiratory Pressure

    MEP = Mouth Expiratory Pressure

    SnIP = Sniff Nasal Inspiratory Pressure

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   5 Years to 19 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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).

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01100892

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Contact: Shihab Hameed, BSc(Med)MBBS +61 2 9382 1456
Contact: Charles Verge, MBBS PhD +61 2 9382 1456

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United States, Colorado
Children's Hospital Colorado Recruiting
Denver, Colorado, United States, 80045
Contact: Christine L Chan, MD    720-777-1234   
Australia, New South Wales
John Hunter Children's Hospital Recruiting
New Lambton, New South Wales, Australia, 2310
Contact: Jodi Hilton         
Contact: Patricia Crock         
Sydney Children's Hospital Recruiting
Randwick, New South Wales, Australia, 2031
Contact: Charles Verge    93821456   
Contact: Shihab Hameed    93821456   
Children's Hospital at Westmead Recruiting
Westmead, New South Wales, Australia, 2145
Contact: Hiran Selvadurai         
Contact: Peter Cooper         
Australia, Queensland
Lady Cilento Children's Hospital Recruiting
Brisbane, Queensland, Australia, 4101
Contact: Claire Wainwright         
Contact: Joyce Cheney         
Australia, South Australia
Women's and Children's Hospital Recruiting
Adelaide, South Australia, Australia, 5006
Contact: Andrew Tai         
Contact: Alexia Pena         
Sponsors and Collaborators
Sydney Children's Hospitals Network
John Hunter Children's Hospital
Lady Cilento Children's Hospital, Brisbane
Women's and Children's Hospital, Adelaide
Children's Hospital Colorado
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Principal Investigator: Charles Verge, MBBS PhD Endocrinology, Sydney Children's Hospital Randwick; School of Women's and Children's Health, University of NSW
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Responsible Party: Dr Charles Verge, Head of Endocrinology, Sydney Children's Hospital, Randwick, Sydney Children's Hospitals Network Identifier: NCT01100892    
Other Study ID Numbers: CF-IDEA
First Posted: April 9, 2010    Key Record Dates
Last Update Posted: September 16, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Dr Charles Verge, Sydney Children's Hospitals Network:
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Insulin Detemir
Hypoglycemic Agents
Physiological Effects of Drugs