Ignite Creation Date:
2025-12-24 @ 3:56 PM
Ignite Modification Date:
2025-12-27 @ 6:36 AM
Study NCT ID:
NCT01100892
Status:
COMPLETED
Last Update Posted:
2023-05-31
First Post:
2010-03-31
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Cystic Fibrosis - Insulin Deficiency, Early Action
Sponsor:
Sydney Children's Hospitals Network
Organization:
Study Overview
Official Title:
Cystic Fibrosis - Insulin Deficiency, Early Action
Status:
COMPLETED
Status Verified Date:
2023-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CF-IDEA
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.
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).
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 Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: