Cystic Fibrosis Homozygous for Phe 508 Del CFTRGLUCORRECTORNCT03512119
Observational Study of Glucose Tolerance Abnormalities in Patient With Cystic Fibrosis Homozygous for Phe 508 Del CFTR Treated by Lumacaftor-Ivacaftor
An observational study of Cystic Fibrosis Homozygous for Phe 508 Del CFTR and Glucose Intolerance or Newly Diagnosis Diabetes, run by University Hospital, Strasbourg, France at 13 sites in France.
Trial summary
Cystic Fibrosis related diabetes (CFRD), a major factor of morbid-mortality in CF, is characterized by a preclinical phase of glucose intolerance particularly long reaching up to 10 years. At the physiopathology level, insulin secretion is determinant in the glucose tolerance abnormalities in CF. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis Homozygous for Phe 508 Del CFTR, Glucose Intolerance or Newly Diagnosis Diabetes
- What is being tested?
- Lumacaftor-Ivacaftor treatment
- How many people
- 55 participants
- Where
- 13 sitesFrance
- Study dates
- 11 Feb 2016to 10 Apr 2019
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patient with cystic fibrosis homozygous for Phe 508 del CFTR having a glucose intolerance or newly diagnosis diabetes
Who may be able to join
- patients with CF homozygous for the Phe508del CFTR mutation aged 12 years and over
- Combined Lumacaftor-Ivacaftor treatment scheduled or already started
- glucose intolerance in OGTT (ADA criteria) or newly diabetes diagnosed at the OGTT (ADA criteria) or diabetic patients with insulin requirement ≤ 0.3 unit / kg / day or without insulin treatment
- signed informed consent of patient and of one parent OR legal representative for minor subject
Who cannot join
- hypersensitivity to the active substances or to any of the excipients of Lumactfor -Ivacaftor
- lung and/or liver transplant patient
- Known diabetes with insulin treatment > 0.3 unit / kg / day
- patient pregnant or wishing to pregnancy
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
France13 sites
Centre Hospitalier Universitaire d'Angers
Angers, 49033
Hôpital Renée Sabran
Giens, 83406
Centre Hospitalier Lyon Sud
Lyon, 69000
Hôpital NORD - Assistance Publique Hôpitaux de Marseille
Marseille, 13385
Hôpital Arnaud de Villeneuve
Montpellier, 34295
Hôpital Robert Debré
Paris, 75019
American Memorial Hospital
Reims, 51092
Clinique "Mucoviscidose" Presqu'île de Perharidy
Roscoff, 29684
Hôpital Charles Nicolle
Rouen, 76031
Hôpitaux Universitaires de Strasbourg
Strasbourg, Alsace, 67000
Hôpital FOCH
Suresnes, 92151
Hôpital Bretonneau - CHRU de Tours
Tours, 37044
Hôpital de Clocheville - CHRU de Tours
Tours, 37044
About this study
Cystic Fibrosis related diabetes (CFRD), a major factor of morbid-mortality in CF, is characterized by a preclinical phase of glucose intolerance particularly long reaching up to 10 years.
At the physiopathology level, insulin secretion is determinant in the glucose tolerance abnormalities in CF. Indeed insulin secretion is dependent of the CFTR activity at the beta cell surface and inhibition of CFTR leads to a decrease in insulin secretion.
Read more
Recently, the combination of the lumacaftor, a CFTR corrector, with Ivacaftor, a CFTR potentiator, was studied in patient with CF homozygous for the Phe508 del CFTR mutation patients and showed an improvement of the respiratory state in comparison with the placebo group.
These data suggests that lumacaftor in combination with ivacaftor in targeting CFTR action may have an early impact on the insulin-secretion and consequently on the glucose tolerance.
- Conditions
- Cystic Fibrosis Homozygous for Phe 508 Del CFTR, Glucose Intolerance or Newly Diagnosis Diabetes
- Being tested
- Lumacaftor-Ivacaftor treatment
- Keywords
- Cystic fibrosis, Glucose tolerance abnormalities, CFTR corrector and potentiator
- Medical subject headings
- Glucose Intolerance, Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lumacaftor-Ivacaftor treatmentDrug
Lumacaftor-Ivacaftor treatment during one year
| Study group | Role | What they receive |
|---|---|---|
| Patient | Patient with cystic fibrosis homozygous for Phe 508 del CFTR having a glucose intolerance or newly diagnosis diabetes Receives: Drug: Lumacaftor-Ivacaftor treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure of 2 hours plasma glucose value (mmol/l) of OGTT, change from baseline at one year of Lumacaftor-Ivacaftor treatment
Measured: Day 0 (traitement beginning) and year 1
Secondary outcomes (17)
Fasting and one hour glucose value of OGTT (mmol/l)
Measured: Day 0 (traitement beginning) and year 1
C peptide and insulin values at T0, 1 , 2 hours of OGTT (µg/l)
Measured: Day 0 (traitement beginning) and year 1
Glucose, insulin and C peptide AUC of OGTT (µU/L)
Measured: Day 0 (traitement beginning) and year 1
HOMA -R , HOMA-S
Measured: Day 0 (traitement beginning) and year 1
Mean glucose value per day and 2 h after meal (mg/dl)
Measured: Day 0 (traitement beginning) and year 1
Duration in hypoglycemic area [hypo CGM = 2 consecutive values below 3.3 mmol/l - % of time spent]
Measured: Day 0 (traitement beginning) and year 1
Duration in hyperglycemic area [for glucose value higher than 7.7 mmol/l, % time /24h]
Number hypoglycaemic events (below 3.3mmol/L, from midnight to 6 am) Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)
Measured: Day 0 (traitement beginning) and year 1
Number hypoglycaemic events (below 3.3mmol/L, from midnight to 6 am)
Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)
Measured: Day 0 (traitement beginning) and year 1
Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)
Measured: Day 0 (traitement beginning) and year 1
HbA1c (mmol/l and %)
Measured: Day 0 (traitement beginning) and year 1
Daily insulin doses (UI/day)
Measured: Day 0 (traitement beginning) and year 1
(BMI) body mass index
Measured: Day 0 (traitement beginning) and year 1
Weight (Kg) maximum weight never reached
Measured: Day 0 (traitement beginning) and year 1
Albumin and Pre albumin (g/l)
Measured: Day 0 (traitement beginning) and year 1
FEV1, Vital Capacity (VC) (L and %)
Measured: Day 0 (traitement beginning) and year 1
O2 saturation (%)
Measured: Day 0 (traitement beginning) and year 1
Number of cures of antibiotics IV and per os /year and interval between 2 cures (week)
Measured: Day 0 (traitement beginning) and year 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2018
- Study start11 Feb 2016
- Primary completion10 Apr 2019
- Study completion10 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis Homozygous for Phe 508 Del CFTRStudies of the same condition, open and under way.
- More trials run by University Hospital, Strasbourg, FranceThe 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
- Other
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Strasbourg, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03512119
- Sponsor's protocol ID
- 6403
- Acronym
- GLUCORRECTOR
- Status last verified
- Nov 2021
- First submitted
- 15 Jan 2018
- Last updated
- 24 Nov 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study of Glucose Tolerance Abnormalities in Patient With Cystic Fibrosis Homozygous for Phe 508 Del CFTR Treated by Lumacaftor-Ivacaftor
- ClinicalTrials.gov ID
- NCT03512119
- Acronym / sponsor ID
- GLUCORRECTOR / 6403
- Conditions
- Cystic Fibrosis Homozygous for Phe 508 Del CFTR, Glucose Intolerance or Newly Diagnosis Diabetes
- Keywords
- Cystic fibrosis, Glucose tolerance abnormalities, CFTR corrector and potentiator
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 55 participants (actual)
- Lead sponsor
- University Hospital, Strasbourg, FranceUniversity, hospital or other
- Study start
- 11 Feb 2016 (actual)
- Primary completion
- 10 Apr 2019 (actual)
- Study completion
- 10 Apr 2019 (actual)
- First posted
- 30 Apr 2018 (actual)
- 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.
Cystic Fibrosis related diabetes (CFRD), a major factor of morbid-mortality in CF, is characterized by a preclinical phase of glucose intolerance particularly long reaching up to 10 years.
At the physiopathology level, insulin secretion is determinant in the glucose tolerance abnormalities in CF. Indeed insulin secretion is dependent of the CFTR activity at the beta cell surface and inhibition of CFTR leads to a decrease in insulin secretion.
Recently, the combination of the lumacaftor, a CFTR corrector, with Ivacaftor, a CFTR potentiator, was studied in patient with CF homozygous for the Phe508 del CFTR mutation patients and showed an improvement of the respiratory state in comparison with the placebo group.
These data suggests that lumacaftor in combination with ivacaftor in targeting CFTR action may have an early impact on the insulin-secretion and consequently on the glucose tolerance.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patient | – | Drug: Lumacaftor-Ivacaftor treatmentPatient with cystic fibrosis homozygous for Phe 508 del CFTR having a glucose intolerance or newly diagnosis diabetes |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patient with cystic fibrosis homozygous for Phe 508 del CFTR having a glucose intolerance or newly diagnosis diabetes
Key inclusion criteria (4 listed)
- patients with CF homozygous for the Phe508del CFTR mutation aged 12 years and over
- Combined Lumacaftor-Ivacaftor treatment scheduled or already started
- glucose intolerance in OGTT (ADA criteria) or newly diabetes diagnosed at the OGTT (ADA criteria) or diabetic patients with insulin requirement ≤ 0.3 unit / kg / day or without insulin treatment
- signed informed consent of patient and of one parent OR legal representative for minor subject
Key exclusion criteria (4 listed)
- hypersensitivity to the active substances or to any of the excipients of Lumactfor -Ivacaftor
- lung and/or liver transplant patient
- Known diabetes with insulin treatment > 0.3 unit / kg / day
- patient pregnant or wishing to pregnancy
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-Ivacaftor treatmentLumacaftor-Ivacaftor treatment during one year | Drug | Patient |
Primary outcome
Measure of 2 hours plasma glucose value (mmol/l) of OGTT, change from baseline at one year of Lumacaftor-Ivacaftor treatment
Time frame: Day 0 (traitement beginning) and year 1
17 secondary outcomes
Fasting and one hour glucose value of OGTT (mmol/l)
Time frame: Day 0 (traitement beginning) and year 1
C peptide and insulin values at T0, 1 , 2 hours of OGTT (µg/l)
Time frame: Day 0 (traitement beginning) and year 1
Glucose, insulin and C peptide AUC of OGTT (µU/L)
Time frame: Day 0 (traitement beginning) and year 1
HOMA -R , HOMA-S
Time frame: Day 0 (traitement beginning) and year 1
Mean glucose value per day and 2 h after meal (mg/dl)
Time frame: Day 0 (traitement beginning) and year 1
Duration in hypoglycemic area [hypo CGM = 2 consecutive values below 3.3 mmol/l - % of time spent]
Time frame: Day 0 (traitement beginning) and year 1
Duration in hyperglycemic area [for glucose value higher than 7.7 mmol/l, % time /24h]
Time frame: Day 0 (traitement beginning) and year 1
Number hypoglycaemic events (below 3.3mmol/L, from midnight to 6 am)
Time frame: Day 0 (traitement beginning) and year 1
Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)
Time frame: Day 0 (traitement beginning) and year 1
HbA1c (mmol/l and %)
Time frame: Day 0 (traitement beginning) and year 1
Daily insulin doses (UI/day)
Time frame: Day 0 (traitement beginning) and year 1
(BMI) body mass index
Time frame: Day 0 (traitement beginning) and year 1
Weight (Kg) maximum weight never reached
Time frame: Day 0 (traitement beginning) and year 1
Albumin and Pre albumin (g/l)
Time frame: Day 0 (traitement beginning) and year 1
FEV1, Vital Capacity (VC) (L and %)
Time frame: Day 0 (traitement beginning) and year 1
O2 saturation (%)
Time frame: Day 0 (traitement beginning) and year 1
Number of cures of antibiotics IV and per os /year and interval between 2 cures (week)
Time frame: Day 0 (traitement beginning) and year 1
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 24 Nov 2021.
- Individual participant dataSharing planned: No
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.
13 sites 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 Homozygous for Phe 508 Del CFTR and Glucose Intolerance or Newly Diagnosis Diabetes, run by University Hospital, Strasbourg, France at 13 sites in France.
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 4 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 24 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in France. 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?
Measure of 2 hours plasma glucose value (mmol/l) of OGTT, change from baseline at one year of Lumacaftor-Ivacaftor treatment (time frame: Day 0 (traitement beginning) and year 1). 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 24 Nov 2021.