DiabetesLIGHT-CFNCT02653027
Effect of Lumacaftor-ivacaftor on Glucose Handling and Tolerance in Cystic Fibrosis Phe508del
A study testing Lumacaftor-ivacaftor and OGTT in people with Diabetes and Cystic Fibrosis, run by Massachusetts General Hospital.
Trial summary
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor effects how people with cystic fibrosis respond to an oral glucose tolerance test, a test for diabetes. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Diabetes, Cystic Fibrosis
- What is being tested?
- Lumacaftor-ivacaftor and OGTT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2018to 1 May 2018
- Phase
- Interventional
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 years old or greater
- Patients diagnosed with CF, genotype homozygous PheDel508
- Subject is planning on starting lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug* OR subject is taking combination drug and had an OGTT done at a partners facility in the 6 months prior to initiating the drug.
- Contraindications for taking drug include abnormal liver enzyme tests, renal dysfunction, pregnancy or nursing mothers
Who cannot join
- Currently taking any medications for diabetes (including oral or injectable antihyperglycemic agents and/or insulin).
- Had an admission for CF exacerbation less than 2 weeks prior to staring the medication. This will be defined as requiring new IV or PO antibiotics different than those used in maintenance therapy.
- Is currently taking oral glucocorticoids or has been on oral or IV glucocorticoids in the past 2 weeks.
About this study
Official title: A Study of the Effect of Combination Lumacaftor and Ivacaftor on Glucose Tolerance in Persons With Cystic Fibrosis Who Are Homozygous for the Phe508del Cystic Fibrosis Transmembrane Conductance Regulator Mutation.
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor effects how people with cystic fibrosis respond to an oral glucose tolerance test, a test for diabetes.
Read the detailed description
This is a single center, open label study with crossover in patients with Cystic Fibrosis (CF). Patients will have 2-3 visits at the Diabetes Center at Massachusetts General Hospital (MGH).
The participants will have been previously screened clinically to make sure they are candidates for starting the combination drug, lumacaftor-ivacaftor. These patients will be contacted prior to their first visit to discuss enrollment in the study.
At the first study visit (Visit 1), the participant will come to the Diabetes Center after an overnight fast of at least 8 hours. The following will occur at this study visit: informed consent; brief medical history; weight and height; vital signs and blood pressure; blood draw for DNA extraction, and an extra research tube for storage; administration of 75g Glucola load as per a standard oral glucose tolerance test (OGTT) protocol; and blood work for glucose and insulin at 30, 60, 90 and 120 minutes after the glucose load. This will be scheduled at a time that is convenient to the patient, with an attempt to coordinate with the patient's visit to the CF clinic prior to starting lumacaftor-ivacaftor combination drug.
At the 2nd study visit (Visit 2), which will take place 3 months after starting the combination drug, the participant will again come to the Diabetes Center after an overnight fast of at least 8 hours. The participant will undergo a second OGTT as in the first visit. The 3rd study visit (Visit 3) will be 6 months after initiation of the drug, and will have a repeat administration of an OGTT. If a participant starts the combination drug before enrolling in the study, he/she can still participate in the study as long as he or she has had a clinical OGTT performed within 6 months of starting the combination drug. In these cases, the informed consent, brief medical history, weight and height and vital signs and blood pressure, as well as blood draw for DNA will occur on the Visit 2, which will be the first study visit for these participants. If a clinical OGTT had been performed prior to but within 6 months of starting the combination therapy, this OGTT can be used in analysis of the data, although will not have the full amount of data as the study OGTT.
- Conditions
- Diabetes, Cystic Fibrosis
- Being tested
- Lumacaftor-ivacaftor, OGTT
- Keywords
- Genetics
- Medical subject headings
- Diabetes Mellitus, Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lumacaftor-ivacaftorDrug
Also called Orkambi
Subjects who are planning on starting the combination therapy (lumacaftor-ivacaftor) will participate in OGTTs before and after starting the medication.
OGTTOther
Also called Oral Glucose tolerance test
A subject is given an oral glucose load and insulin and glucose measurements are taken at specified time periods.
| Study group | Role | What they receive |
|---|---|---|
| Lumacaftor Ivacaftor | Experimental | Subjects will get an OGTT before and after starting the combination therapy lumacaftor-ivacaftor. Receives: Drug: Lumacaftor-ivacaftor; Other: OGTT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Fasting Glucose
This will be compared from baseline to 3 months after starting the medication
Measured: 3 months
Change in Fasting Glucose
This will compare baseline to 6 months after starting the medication
Measured: 6 months
Secondary outcomes (4)
Genetic risk score
We will examine how the OGTT data is dependent on genotype at variants associated with type 2 diabetes using a genetic risk score
Measured: 6 months
Pulmonary function test (PFT) FEV1 measurements
We will compare how PFT measurement of FEV1 are related to changes in OGTT
Measured: 6 months
Change in Fasting Insulin
This will be compared from baseline to 3 months after starting the medication
Measured: 3 months
Change in Fasting Insulin
This will be compared from baseline to 6 months after starting the medication
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2016 (estimated)
- Study start1 Jan 2018
- Primary completion1 Feb 2018 (estimated)
- Study completion1 May 2018
Why it stopped: Potential subjects were already on the combination therapy.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for DiabetesStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jose C. Florez, MD, PhD, Massachusetts General Hospital
- Investigators
- Jose Florez, MD, PhD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02653027
- Sponsor's protocol ID
- 2015P001811
- Acronym
- LIGHT-CF
- Status last verified
- Jul 2019
- First submitted
- 7 Jan 2016
- Last updated
- 31 Jul 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17.
PubMed 25981758Bellin MD, Laguna T, Leschyshyn J, Regelmann W, Dunitz J, Billings J, Moran A. Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study. Pediatr Diabetes. 2013 Sep;14(6):417-21. doi: 10.1111/pedi.12026. Epub 2013 Mar 13.
PubMed 23952705
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of the Effect of Combination Lumacaftor and Ivacaftor on Glucose Tolerance in Persons With Cystic Fibrosis Who Are Homozygous for the Phe508del Cystic Fibrosis Transmembrane Conductance Regulator Mutation.
- ClinicalTrials.gov ID
- NCT02653027
- Acronym / sponsor ID
- LIGHT-CF / 2015P001811
- Conditions
- Diabetes, Cystic Fibrosis
- Keywords
- Genetics
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Potential subjects were already on the combination therapy.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 1 Feb 2018 (estimated)
- Study completion
- 1 May 2018 (actual)
- First posted
- 12 Jan 2016 (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.
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor effects how people with cystic fibrosis respond to an oral glucose tolerance test, a test for diabetes.
Detailed description
This is a single center, open label study with crossover in patients with Cystic Fibrosis (CF). Patients will have 2-3 visits at the Diabetes Center at Massachusetts General Hospital (MGH).
The participants will have been previously screened clinically to make sure they are candidates for starting the combination drug, lumacaftor-ivacaftor. These patients will be contacted prior to their first visit to discuss enrollment in the study.
At the first study visit (Visit 1), the participant will come to the Diabetes Center after an overnight fast of at least 8 hours. The following will occur at this study visit: informed consent; brief medical history; weight and height; vital signs and blood pressure; blood draw for DNA extraction, and an extra research tube for storage; administration of 75g Glucola load as per a standard oral glucose tolerance test (OGTT) protocol; and blood work for glucose and insulin at 30, 60, 90 and 120 minutes after the glucose load. This will be scheduled at a time that is convenient to the patient, with an attempt to coordinate with the patient's visit to the CF clinic prior to starting lumacaftor-ivacaftor combination drug.
At the 2nd study visit (Visit 2), which will take place 3 months after starting the combination drug, the participant will again come to the Diabetes Center after an overnight fast of at least 8 hours. The participant will undergo a second OGTT as in the first visit. The 3rd study visit (Visit 3) will be 6 months after initiation of the drug, and will have a repeat administration of an OGTT. If a participant starts the combination drug before enrolling in the study, he/she can still participate in the study as long as he or she has had a clinical OGTT performed within 6 months of starting the combination drug. In these cases, the informed consent, brief medical history, weight and height and vital signs and blood pressure, as well as blood draw for DNA will occur on the Visit 2, which will be the first study visit for these participants. If a clinical OGTT had been performed prior to but within 6 months of starting the combination therapy, this OGTT can be used in analysis of the data, although will not have the full amount of data as the study OGTT.
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lumacaftor Ivacaftor | Experimental | Drug: Lumacaftor-ivacaftor, Other: OGTTSubjects will get an OGTT before and after starting the combination therapy lumacaftor-ivacaftor. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age 18 years old or greater
- Patients diagnosed with CF, genotype homozygous PheDel508
- Subject is planning on starting lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug* OR subject is taking combination drug and had an OGTT done at a partners facility in the 6 months prior to initiating the drug.
Key exclusion criteria (3 listed)
- Currently taking any medications for diabetes (including oral or injectable antihyperglycemic agents and/or insulin).
- Had an admission for CF exacerbation less than 2 weeks prior to staring the medication. This will be defined as requiring new IV or PO antibiotics different than those used in maintenance therapy.
- Is currently taking oral glucocorticoids or has been on oral or IV glucocorticoids in the past 2 weeks.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lumacaftor-ivacaftorAlso: OrkambiSubjects who are planning on starting the combination therapy (lumacaftor-ivacaftor) will participate in OGTTs before and after starting the medication. | Drug | Lumacaftor Ivacaftor |
| OGTTAlso: Oral Glucose tolerance testA subject is given an oral glucose load and insulin and glucose measurements are taken at specified time periods. | Other | Lumacaftor Ivacaftor |
Primary outcome
Change in Fasting Glucose
Time frame: 3 months
This will be compared from baseline to 3 months after starting the medication
Change in Fasting Glucose
Time frame: 6 months
This will compare baseline to 6 months after starting the medication
4 secondary outcomes
Genetic risk score
Time frame: 6 months
Pulmonary function test (PFT) FEV1 measurements
Time frame: 6 months
Change in Fasting Insulin
Time frame: 3 months
Change in Fasting Insulin
Time frame: 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2019.
- Background: PubMed 25981758Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17.
- Background: PubMed 23952705Bellin MD, Laguna T, Leschyshyn J, Regelmann W, Dunitz J, Billings J, Moran A. Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study. Pediatr Diabetes. 2013 Sep;14(6):417-21. doi: 10.1111/pedi.12026. Epub 2013 Mar 13.
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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.
Jose Florez, MD, PhD principal investigator
Massachusetts General Hospital
Questions about this trial
What is this clinical trial studying?
A study testing Lumacaftor-ivacaftor and OGTT in people with Diabetes and Cystic Fibrosis, run by Massachusetts General Hospital.
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 3 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: withdrawn, as of the sponsor's last update on 31 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
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 was withdrawn before enrolling participants, so there are no results.
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 in Fasting Glucose (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02653027, which also lists 2 linked publications.
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 31 Jul 2019.