Cystic Fibrosis Related DiabetesNCT02039986
Ivacaftor (Kalydeco) and Insulin in Cystic Fibrosis (CF)
An observational study of Cystic Fibrosis Related Diabetes and Cystic Fibrosis, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
This study is aimed at better understanding the impact of ivacaftor upon insulin and incretin secretion and glucose tolerance in patients with Cystic Fibrosis with a glycine (G551D) mutation. Investigators hypothesize that treatment with ivacaftor improves insulin secretion in individuals with CF. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis Related Diabetes, Cystic Fibrosis
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Jan 2014to 11 Oct 2016
- Phase
- Observational
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with a confirmed diagnosis of cystic fibrosis.
Who may be able to join
- 6 yrs or older with cystic fibrosis
- at least one G551D CFTR mutation or other non-G551D gating mutation, or residual function CFTR mutation such as, but not limited to, R117H mutation, for which ivacaftor is to be initiated.
- Plan to initiate ivacaftor treatment for FDA approved indications by clinical care team or as part of an ongoing study of ivacaftor for other CFTR mutations, including gating mutations, or residual function mutations.
- not pregnant
Who cannot join
- established diagnosis of non-CF related diabetes (ie., Type I diabetes)
- history of clinically symptomatic pancreatitis in past year
- prior lung or liver transplant
- severe CF liver disease
- fundoplication-related dumping syndrome
- medical co-morbidities that are not CF-related or are unstable per the Investigator opinion
Show all 12Show fewer
- acute CF pulmonary exacerbation within 4 weeks prior to study procedures
- treatment with oral or intravenous corticosteroids within 4 weeks of study
- hemoglobin <10g/dL within 90 days of GPA test or at Screening
- abnormal renal function within 90 days of GPA test or at Screening
- long-standing CFRD with fasting hyperglycemia, elevated HbA1C (>8) beyond time surrounding diagnosis of CFRD, significant basal insulin requirement
- inability to perform study specific procedures (MMTT, GPA).
Trial locations and contacts
1 site in 1 country.
United States1 site
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Effects of Ivacaftor (Kalydeco) Treatment Upon Insulin and Incretin Secretion in Patients With Cystic Fibrosis
This study is aimed at better understanding the impact of ivacaftor upon insulin and incretin secretion and glucose tolerance in patients with Cystic Fibrosis with a glycine (G551D) mutation. Investigators hypothesize that treatment with ivacaftor improves insulin secretion in individuals with CF.
Read the detailed description
Cystic Fibrosis Related Diabetes (CFRD) is associated with worse nutritional status, greater pulmonary function decline, and increased mortality, highlighting its relevance in Cystic Fibrosis (CF). CFRD arises primarily from compromised insulin secretion - traditionally considered a by-product of pancreatic exocrine tissue damage and fibrosis. Recent developments in the field of diabetes are propelling a re-examination of this basic explanation. The impact of the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, upon insulin secretion and glucose regulation has not been examined, but improved glucose tolerance has been appreciated anecdotally. This study aims to understand the impact of ivacaftor therapy upon blood glucose and insulin and incretin secretion.
- Conditions
- Cystic Fibrosis Related Diabetes, Cystic Fibrosis
- Keywords
- cystic fibrosis, CF, cystic fibrosis related diabetes, CFRD, kalydeco, ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| all subjects | all subjects enrolled in same cohort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in insulin secretion capacity at 16 weeks
To compare insulin secretion and maximal insulin secretory capacity prior to initiation of ivacaftor and after 16 weeks of ivacaftor treatment in subjects with CF and at least one G551D CFTR mutation, or other CFTR gating mutation, and to explore the impact of ivacaftor upon incretin secretion, incretin regulation of insulin secretion, and glucose excursion during a mixed meal tolerance test in CF.
Measured: baseline and 16 weeks
Secondary outcomes (1)
Composite change from baseline in relationships of insulin secretion and protein and interleukin levels at 16 weeks
To explore the composite relationships of insulin secretion, maximal insulin secretory capacity, and incretin secretion with secreted frizzled protein-4 levels and interleukin 1β levels.
Measured: baseline and 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2014 (estimated)
- Study start6 Jan 2014
- Primary completion11 Oct 2016
- Study completion11 Oct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis Related DiabetesStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andrea Kelly, MD, MSCE, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02039986
- Sponsor's protocol ID
- 13-010465
- Other IDs
- KELLY13A0
- Status last verified
- Dec 2018
- First submitted
- 16 Jan 2014
- Last updated
- 14 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Ivacaftor (Kalydeco) Treatment Upon Insulin and Incretin Secretion in Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT02039986
- Acronym / sponsor ID
- 13-010465
- Conditions
- Cystic Fibrosis Related Diabetes, Cystic Fibrosis
- Keywords
- cystic fibrosis, CF, cystic fibrosis related diabetes, CFRD, kalydeco, ivacaftor
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Study start
- 6 Jan 2014 (actual)
- Primary completion
- 11 Oct 2016 (actual)
- Study completion
- 11 Oct 2016 (actual)
- First posted
- 20 Jan 2014 (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.
This study is aimed at better understanding the impact of ivacaftor upon insulin and incretin secretion and glucose tolerance in patients with Cystic Fibrosis with a glycine (G551D) mutation. Investigators hypothesize that treatment with ivacaftor improves insulin secretion in individuals with CF.
Detailed description
Cystic Fibrosis Related Diabetes (CFRD) is associated with worse nutritional status, greater pulmonary function decline, and increased mortality, highlighting its relevance in Cystic Fibrosis (CF). CFRD arises primarily from compromised insulin secretion - traditionally considered a by-product of pancreatic exocrine tissue damage and fibrosis. Recent developments in the field of diabetes are propelling a re-examination of this basic explanation. The impact of the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, upon insulin secretion and glucose regulation has not been examined, but improved glucose tolerance has been appreciated anecdotally. This study aims to understand the impact of ivacaftor therapy upon blood glucose and insulin and incretin secretion.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| all subjects | – | –all subjects enrolled in same cohort |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with a confirmed diagnosis of cystic fibrosis.
Key inclusion criteria (4 listed)
- 6 yrs or older with cystic fibrosis
- at least one G551D CFTR mutation or other non-G551D gating mutation, or residual function CFTR mutation such as, but not limited to, R117H mutation, for which ivacaftor is to be initiated.
- Plan to initiate ivacaftor treatment for FDA approved indications by clinical care team or as part of an ongoing study of ivacaftor for other CFTR mutations, including gating mutations, or residual function mutations.
- not pregnant
Key exclusion criteria (12 listed)
- established diagnosis of non-CF related diabetes (ie., Type I diabetes)
- history of clinically symptomatic pancreatitis in past year
- prior lung or liver transplant
- severe CF liver disease
- fundoplication-related dumping syndrome
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change from baseline in insulin secretion capacity at 16 weeks
Time frame: baseline and 16 weeks
To compare insulin secretion and maximal insulin secretory capacity prior to initiation of ivacaftor and after 16 weeks of ivacaftor treatment in subjects with CF and at least one G551D CFTR mutation, or other CFTR gating mutation, and to explore the impact of ivacaftor upon incretin secretion, incretin regulation of insulin secretion, and glucose excursion during a mixed meal tolerance test in CF.
1 secondary outcome
Composite change from baseline in relationships of insulin secretion and protein and interleukin levels at 16 weeks
Time frame: baseline and 16 weeks
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 14 Dec 2018.
Related trials for Cystic Fibrosis Related Diabetes
Recruiting
ACCESS 2: AI for pediatriC diabetiC Eye examS Study 2
Type 1 Diabetes, Type 2 Diabetes
Active, not recruitingPhase 2/3
SGLT2 Inhibitor Therapy in Cystic Fibrosis-related Diabetes
Cystic Fibrosis-related Diabetes, Cystic Fibrosis
Active, not recruitingPhase 3
Bionic Pancreas in CFRD
Cystic Fibrosis-related Diabetes
RecruitingPhase 1
Dorzagliatin in Pancreatic Insufficient Cystic Fibrosis
Pancreatic Insufficiency, Cystic Fibrosis-related Diabetes
RecruitingObservational study
Hormonal Responses to a Mixed Meal in People With Cystic Fibrosis
Cystic Fibrosis
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.
Andrea Kelly, MD, MSCE principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis Related Diabetes and Cystic Fibrosis, run by Children's Hospital of Philadelphia at 1 site in United States.
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 4 inclusion and 12 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 14 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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.
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 from baseline in insulin secretion capacity at 16 weeks (time frame: baseline and 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 Dec 2018.