Cystic Fibrosis - Insulin Deficiency, Early Action

NCT01100892 | Completed Phase 3 DMC

• 1 other identifier: CF-IDEA
• Study Type: interventional
• Target: 100
• Locations: 2 countries
• Sites: 6

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.

Conditions

Cystic Fibrosis; Diabetes

Keywords

Pediatrics; Pancreas

Outcome Measures

Primary Outcomes (2)

• Change in Weight SDS (Standard Deviation Score)

  12 months

• Change in lung function (FEV1, FVC)

  12 months

Secondary Outcomes (8)

• Reduced rate of decline in glycaemic category, comparing OGTT at baseline and 12 months.

  12 months

• Reduced frequency of hospitalisation for acute respiratory illness

  12 months

• Change in glycaemic status assessed by HbA1c and CGM

  12 months

• Body composition by DEXA. Patients at CHW will also have pQCT.

  12 months

• Change in Grip-strength

  12 months

Study Arms (2)

Control group

NO INTERVENTION

Observation only. Does not receive once-daily insulin detemir.

Once-daily insulin detemir

EXPERIMENTAL

Once-daily insulin detemir

Drug: Once-daily insulin detemir

Interventions

Once-daily insulin detemir DRUG

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).

Also known as: Levemir

Once-daily insulin detemir

Eligibility Criteria

• Age: 5 Years - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sydney Children's Hospitals Network: lead
• John Hunter Children's Hospital: collaborator
• Lady Cilento Children's Hospital, Brisbane: collaborator
• Women's and Children's Hospital, Adelaide: collaborator
• Children's Hospital Colorado: collaborator

Study Sites (6)

Children's Hospital Colorado

Denver, Colorado, 80045, United States

Location

John Hunter Children's Hospital

New Lambton, New South Wales, 2310, Australia

Location

Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia

Location

Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

Lady Cilento Children's Hospital

Brisbane, Queensland, 4101, Australia

Location

Women's and Children's Hospital

Adelaide, South Australia, 5006, Australia

Location

Related Publications (4)

• 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.

  PMID: 19910502 BACKGROUND

• Hameed 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.

  PMID: 21493664 BACKGROUND

• Hameed 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.

  PMID: 21626717 BACKGROUND

• Hameed 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.

  PMID: 40379429 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis; Diabetes Mellitus

Interventions

Insulin Detemir

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Insulin, Long-Acting; Insulins; Pancreatic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Charles Verge, MBBS PhD

  Endocrinology, Sydney Children's Hospital Randwick; School of Women's and Children's Health, University of NSW

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Endocrinology, Sydney Children's Hospital, Randwick

Study Record Dates

• First Submitted: March 31, 2010
• First Posted: April 9, 2010
• Study Start: December 1, 2010
• Primary Completion: February 1, 2023
• Study Completion: February 1, 2023
• Last Updated: May 31, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (5); US (1)