Once-daily insulin detemir for Cystic Fibrosis

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
John Hunter Children's Hospital, New Lambton, Australia
Cystic Fibrosis+2 More
Once-daily insulin detemir - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This study is evaluating whether insulin treatment can improve lung function and weight in children with cystic fibrosis.

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Eligible Conditions

  • Cystic Fibrosis
  • Diabetes

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

This trial is evaluating whether Once-daily insulin detemir will improve 2 primary outcomes and 8 secondary outcomes in patients with Cystic Fibrosis. Measurement will happen over the course of 12 months.

12 months
Bacterial colonisation of sputum
Body composition by DEXA. Patients at CHW will also have pQCT.
Change in Grip-strength
Change in Weight SDS (Standard Deviation Score)
Change in effort-dependent lung function: MIP, MEP, SnIP
Change in glycaemic status assessed by HbA1c and CGM
Change in lung function (FEV1, FVC)
Improved quality of life, measured by a validated CF QOL questionnaire
Reduced frequency of hospitalisation for acute respiratory illness
Reduced rate of decline in glycaemic category, comparing OGTT at baseline and 12 months.

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

Control group
1 of 2
Once-daily insulin detemir
1 of 2
Active Control
Experimental Treatment

This trial requires 100 total participants across 2 different treatment groups

This trial involves 2 different treatments. Once-daily Insulin Detemir is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

Once-daily insulin detemir
Drug
Once-daily insulin detemir
Control groupObservation only. Does not receive once-daily insulin detemir.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 12 months for reporting.

Who is running the study

Principal Investigator
D. C. V.
Dr Charles Verge, Head of Endocrinology, Sydney Children's Hospital, Randwick
Sydney Children's Hospitals Network

Closest Location

Children's Hospital Colorado - Denver, CO

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 2 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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).
Patients with CF aged >=5 yrs attending one of the study sites.

Patient Q&A Section

What causes cystic fibrosis?

"It is now widely accepted that there are numerous genetic and environmental factors which trigger CF. The environmental factors may include exposure to environmental toxins, such as tobacco smoke, air pollution and occupational hazards. There is also accumulating evidence which implicates a pathophysiological role for defective neutrophils in the causation of CF. Patients with more severe forms of the disorder have a higher chance of developing lung infections. In patients with milder forms of the disease, there is a risk of chronic lung disease and chronic rhinosinusitis. However, in a large survey of patients in the United Kingdom, a link was found between the prevalence of CF and certain environmental factors that included cigarette smoking, male sex, poor education, and living on damp, clayey soils." - Anonymous Online Contributor

Unverified Answer

What are the signs of cystic fibrosis?

"The signs that are present in patients with cystic fibrosis are similar to those that are present in patients with other chronic lung diseases. This is partly due to the fact that cystic fibrosis is caused by a mutation affecting a protein that is similar to the protein coded for by the "KCNE1" gene. The disease is probably preceded by a progressive decline in lung function.\n" - Anonymous Online Contributor

Unverified Answer

What are common treatments for cystic fibrosis?

"There is a substantial and growing list of commonly prescribed medications for both adults and children, though many have been shown to have no proven beneficial effects. Because of the complexity involved in designing a clinical research protocol, we can only give guidelines. When possible, it is important to conduct a comprehensive review of current evidence, along with careful analysis of cost-effectiveness considerations, before making any treatment decision." - Anonymous Online Contributor

Unverified Answer

What is cystic fibrosis?

"CF is a genetic condition that affects a protein in the body. People who have cystic fibrosis have a faulty protein that affects the body’s cells, tissues, and the glands that make mucus and sweat. Mucus is normally slippery and protects the linings of the airways, digestive tract, and other organs and tissues. People with cystic fibrosis make thick, sticky mucus that can build up and lead to blockages, damage, or infections in the affected organs. Inflammation also causes damage to organs like the lungs and pancreas, and can be lethal if left untreated and untreated." - Anonymous Online Contributor

Unverified Answer

How many people get cystic fibrosis a year in the United States?

"Approximately 40,000 new cases of cystic fibrosis were diagnosed each year in the United States. The 5-year survival rate was about 50%, comparable to the 10-year survival rate in the general population." - Anonymous Online Contributor

Unverified Answer

Can cystic fibrosis be cured?

"The two CFTR gene mutation combinations in the two families do not appear to be sufficient for a cure. We can no longer say that there is no cure for CF. We know a cure is not present. We still need further research in cystic fibrosis. We are making progress by understanding how the disease progresses and how we may better treat the disease. But a cure for cystic fibrosis is still distant when you look at the long-term forecast. The development of a cure will depend largely on research in cystic fibrosis and new drugs and clinical advances in cystic fibrosis." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of cystic fibrosis?

"The primary cause of cystic fibrosis is unknown, but recent models suggest that the mechanism causing disease involves several gene defects. The mutations in CFTR are thought to prevent the normal secretion of fluid by the pancreas, by disrupting protein function in a multitude of ways. The abnormalities of organs such as the lungs, heart, and liver contribute to the respiratory symptoms. Some of the symptoms are the result of the buildup of mucus in the lungs, which make it harder to breathe. Complications also arise from infection, which are the result of thick mucus that collect in the lungs, making it inhospitable to bacterial growth." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving once-daily insulin detemir?

"In this pilot trial, there were no clinically significant differences between once- and thrice-daily insulin detemir at comparable safety and tolerability, suggesting that once-daily is a rational dosing strategy. The longer duration of action for once-daily insulin detemir may facilitate improved diabetes control, adherence, and patient satisfaction. The design of a larger, longer-term study that includes both once- and thrice-daily administration is warranted." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for cystic fibrosis?

"Clinical trials as an approach to defining and prioritizing treatments in CF is only viable if it is considered before clinical trial data become available." - Anonymous Online Contributor

Unverified Answer

What is the latest research for cystic fibrosis?

"At present, several gene therapies exist. The first one was registered in the Swiss market in 2000. In the UK, the NAGA gene therapy was registered and will be in the market in 2010. Finally, an intravenous drug delivery, the AAV-ILV-SNAP-system was registered in November 2008 and should be in the market in 2009. Also, clinical trials are being performed on gene therapy of Cystic Fibrosis.\nAt present, no cure for cystic fibrosis exists and the standard life expectancy is only 44 years. Although there is no treatment for the mutation, treatment is available for the symptoms." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating cystic fibrosis?

"There has unfortunately been little progress on how to treat cystic fibrosis. New therapies are being investigated and even though most of them have significant problems and only a few treatments have been tested in a clinical trial, they are still in various stages of development. For example, there is potential that a new type of gene therapy, the use of an AAV viral vector that carries an artificial gene of interest into human cells, may be an effective treatment for cystic fibrosis. Another method is to make genetic modifications to the CFTR gene and in this way to modify the effect of the defective CFTR gene. One problem with this method is that only certain forms of the CF gene have been explored thus far." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets cystic fibrosis?

"It will be important to develop therapies that target specific genetic mutations or gene expressions (such as genes involved in the development of certain types of lung disease) to treat cystic fibrosis. Current therapies generally target symptoms of cystic fibrosis in patients with specific mutations or gene expression profiles. However, treatments that target more specific gene expression profiles, which may be found in only a certain portion of patients with particular mutations and/or gene expression profiles may be more promising. One way to pursue such research is to use whole genome gene expression measurements to identify specific patterns of gene expression that characterize certain patient populations." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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