Cystic FibrosisNCT02858843
The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia
A study testing lumacaftor-ivacaftor in people with Cystic Fibrosis and Diabetes, run by Massachusetts General Hospital.
Trial summary
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Cystic Fibrosis, Diabetes
- What is being tested?
- lumacaftor-ivacaftor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- 1 Aug 2016to 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 cystic fibrosis (CF), genotype homozygous Phe508del
- Subject has been started on lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug* * Contraindications for taking drug include abnormal liver enzyme tests, renal dysfunction, pregnancy or nursing mothers
Who cannot join
- Does not have a HgbA1c within 1 year prior to starting medication.
- Has not been on the combination therapy for at least 2 months
About this study
Official title: A Study of the Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia in Persons With Cystic Fibrosis
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.
Read the detailed description
This is a single center, open label study. Patients will have 1 visits at the Diabetes Research Center (DRC), or Clinical Research Center (CRC).
The participants will have been previously screened to make sure they are candidates for the study. These patients will be contacted prior to their first visit to discuss enrollment in the study.
At the study visit the participant will come to the CRC or DRC for a research visit. 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, A1c and an extra research tube for storage. This will be scheduled at a time that is convenient to the patient.
- Conditions
- Cystic Fibrosis, Diabetes
- Being tested
- lumacaftor-ivacaftor
- Keywords
- genetics
- Medical subject headings
- Cystic Fibrosis, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
lumacaftor-ivacaftorDrug
Also called Orkambi
Drug given for cystic fibrosis
| Study group | Role | What they receive |
|---|---|---|
| Lumacaftor-ivacaftor | Experimental | Subjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor. Receives: Drug: lumacaftor-ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in glycated hemoglobin (hemoglobin A1C)
A blood test will be used to determine the hemoglobin A1c change while on the medication.
Measured: 1 year
Change in units of insulin used over a period of 6 months to 1 year.
Using chart review, the change in insulin units used per day will be calculated
Measured: 1 year
Secondary outcomes (2)
Change in glycemia contingent on genetic risk score
The investigators will examine how change in glycemia is dependent on genotype at variants associated with type 2 diabetes and insulin secretion using genetic risk scores.
Measured: 1 year
Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements
The investigators will compare how PFT measurement of FEV1 are related to changes in glycemia
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2016 (estimated)
- Study start1 Aug 2016
- Primary completion1 May 2018
- Study completion1 May 2018
Why it stopped: We were unable to enroll eligible subjects for the study.
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 Cystic FibrosisStudies 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
- 1 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02858843
- Sponsor's protocol ID
- 2016P000959
- Status last verified
- May 2018
- First submitted
- 5 Jul 2016
- Last updated
- 21 May 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
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 21 May 2018. 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 Markers of Hyperglycemia in Persons With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT02858843
- Acronym / sponsor ID
- 2016P000959
- Conditions
- Cystic Fibrosis, Diabetes
- Keywords
- genetics
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: We were unable to enroll eligible subjects for the study.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- 1 Aug 2016
- Primary completion
- 1 May 2018 (actual)
- Study completion
- 1 May 2018 (actual)
- First posted
- 8 Aug 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 affects glycemia in patient with cystic fibrosis.
Detailed description
This is a single center, open label study. Patients will have 1 visits at the Diabetes Research Center (DRC), or Clinical Research Center (CRC).
The participants will have been previously screened to make sure they are candidates for the study. These patients will be contacted prior to their first visit to discuss enrollment in the study.
At the study visit the participant will come to the CRC or DRC for a research visit. 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, A1c and an extra research tube for storage. This will be scheduled at a time that is convenient to the patient.
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-ivacaftorSubjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Age 18 years old or greater
- Patients diagnosed with cystic fibrosis (CF), genotype homozygous Phe508del
- Subject has been started on lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug* * Contraindications for taking drug include abnormal liver enzyme tests, renal dysfunction, pregnancy or nursing mothers
Key exclusion criteria (2 listed)
- Does not have a HgbA1c within 1 year prior to starting medication.
- Has not been on the combination therapy for at least 2 months
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: OrkambiDrug given for cystic fibrosis | Drug | Lumacaftor-ivacaftor |
Primary outcome
Change in glycated hemoglobin (hemoglobin A1C)
Time frame: 1 year
A blood test will be used to determine the hemoglobin A1c change while on the medication.
Change in units of insulin used over a period of 6 months to 1 year.
Time frame: 1 year
Using chart review, the change in insulin units used per day will be calculated
2 secondary outcomes
Change in glycemia contingent on genetic risk score
Time frame: 1 year
Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements
Time frame: 1 year
Results and safety
The study was stopped early (reason given: We were unable to enroll eligible subjects for the study.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2018.
- 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.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing lumacaftor-ivacaftor in people with Cystic Fibrosis and Diabetes, 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 3 inclusion and 2 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: stopped early, as of the sponsor's last update on 21 May 2018. 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 stopped early (reason given: We were unable to enroll eligible subjects for the study.). Results are not available in the source record.
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 glycated hemoglobin (hemoglobin A1C) (time frame: 1 year). 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 NCT02858843, 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 21 May 2018.