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Prevention of Cystic Fibrosis Diabetes (Prevent)

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ClinicalTrials.gov Identifier: NCT00967798
Recruitment Status : Terminated (Could not achieve target enrollment)
First Posted : August 28, 2009
Results First Posted : June 25, 2018
Last Update Posted : June 25, 2018
Sponsor:
Information provided by (Responsible Party):
Arlene Stecenko, Emory University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Prevention
Conditions Cystic Fibrosis
Prediabetes
Intervention Drug: Sitagliptin
Enrollment 33
Recruitment Details Participants were recruited between May 2010 and August 2016.
Pre-assignment Details  
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description Participants randomized to receive Sitagliptin Participants randomized to receive a placebo
Period Title: Overall Study
Started 17 16
Completed 8 13
Not Completed 9 3
Reason Not Completed
Withdrawal by Subject             5             2
Lost to Follow-up             4             1
Arm/Group Title Sitagliptin Placebo Total
Hide Arm/Group Description Participants randomized to receive Sitagliptin Participants randomized to receive a placebo Total of all reporting groups
Overall Number of Baseline Participants 17 16 33
Hide Baseline Analysis Population Description
All participants are included in the baseline analysis.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 16 participants 33 participants
<=18 years
7
  41.2%
5
  31.3%
12
  36.4%
Between 18 and 65 years
10
  58.8%
11
  68.8%
21
  63.6%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 16 participants 33 participants
Female
7
  41.2%
6
  37.5%
13
  39.4%
Male
10
  58.8%
10
  62.5%
20
  60.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 16 participants 33 participants
Hispanic or Latino
0
   0.0%
1
   6.3%
1
   3.0%
Not Hispanic or Latino
17
 100.0%
15
  93.8%
32
  97.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 16 participants 33 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   5.9%
0
   0.0%
1
   3.0%
White
16
  94.1%
15
  93.8%
31
  93.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   6.3%
1
   3.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 17 participants 16 participants 33 participants
17
 100.0%
16
 100.0%
33
 100.0%
1.Primary Outcome
Title Number of Participants With Conversion to Cystic Fibrosis Related Diabetes
Hide Description The number of participants with conversion to cystic fibrosis related diabetes was determined.
Time Frame Month 15
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description:
Participants randomized to receive Sitagliptin
Participants randomized to receive a placebo
Overall Number of Participants Analyzed 17 16
Measure Type: Count of Participants
Unit of Measure: Participants
2
  11.8%
1
   6.3%
2.Secondary Outcome
Title Change in Beta-cell Disposition Index
Hide Description Preservation of beta-cell function was to be assessed with the disposition index, which is a measurement of beta-cell function adjusted for insensitivity to insulin.
Time Frame Baseline through Month 15
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Samples for the serum assays were destroyed when a freezer broke over a weekend and the samples thawed.
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description:
Participants randomized to receive Sitagliptin
Participants randomized to receive a placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Change in Redox Couples Glutathione/Glutathione Disulfide and Cysteine/Cystine
Hide Description Cysteine (Cys)/cystine (CySS) and glutathione (GSH)/glutathione disulfide (GSSG) redox couples are biomarkers of oxidative stress.
Time Frame Baseline through Month 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Samples for the serum assays were destroyed when a freezer broke over a weekend and the samples thawed.
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description:
Participants randomized to receive Sitagliptin
Participants randomized to receive a placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Change in Inflammatory Cytokines
Hide Description Hyperglycemia causes release of pro-inflammatory cytokines which can further compromise beta-cell function by increasing insulin resistance and by inducing beta-cell apoptosis. Inflammatory cytokines that have been shown to contribute to destruction of beta-cells include interleukin 1 beta (IL-1b), tumor necrosis factor alpha (TNFa), and interleukin 6 (IL-6).
Time Frame Baseline through Month 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Samples for the serum assays were destroyed when a freezer broke over a weekend and the samples thawed.
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description:
Participants randomized to receive Sitagliptin
Participants randomized to receive a placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Change in Percent Predicted FEV1
Hide Description The decline of lung function was assessed with forced expiratory volume (FEV1), which measures how much air is exhaled during one second of a forced exhale. Change is described as the difference in FEV1 at baseline subtracted from FEV1 at the end of treatment study visit. A protocol change during the study reduced the treatment time from 24 months to 12 months. The end of treatment study visit for participants in the early part of the study occurred at 24 months, while the end of treatment visit was a 12 months for participants enrolling later. Negative values indicate a decline in lung function over the course of the study.
Time Frame Baseline, end of treatment (Month 12 or Month 24)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The participants included in this analysis are limited to those who have a FEV value for the baseline and end of treatment visits.
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description:
Participants randomized to receive Sitagliptin
Participants randomized to receive a placebo
Overall Number of Participants Analyzed 8 13
Mean (Standard Deviation)
Unit of Measure: percent predicted
-9.7  (29.4) -8.5  (14.3)
Time Frame For this study, the reporting period for adverse events is from the time the subject signs the informed consent form to 90 days after taking the last dose of the study medication, up to 15 months.
Adverse Event Reporting Description Over 90% of the morbidity in cystic fibrosis (CF) is due to respiratory dysfunction and to readily identify an increase in the frequency of respiratory exacerbations, adverse events will be classified as respiratory or non-respiratory events. Respiratory events are defined as any increase in respiratory symptoms and/or signs (increase sputum production, increase in cough, runny or stuffy nose, a cold, decrease in lung function, and/or change in physical findings of the respiratory system).
 
Arm/Group Title Sitagliptin Placebo
Hide Arm/Group Description Participants randomized to receive Sitagliptin Participants randomized to receive a placebo
All-Cause Mortality
Sitagliptin Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/17 (0.00%)      0/16 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Sitagliptin Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/17 (64.71%)      9/16 (56.25%)    
General disorders     
Hospitalization due to fever and body aches *  0/17 (0.00%)  0 1/16 (6.25%)  1
Musculoskeletal and connective tissue disorders     
Hospitalization due to osteochondroplasty of femoral neck due to previous motor vehical accident *  0/17 (0.00%)  0 1/16 (6.25%)  1
Renal and urinary disorders     
Lithotripsy with stent placement *  1/17 (5.88%)  1 1/16 (6.25%)  1
Respiratory, thoracic and mediastinal disorders     
Hospitalization due to Respiratory Event * [1]  11/17 (64.71%)  30 9/16 (56.25%)  27
Skin and subcutaneous tissue disorders     
Hospitalization due to erythematous maculopapualr rash *  1/17 (5.88%)  1 0/16 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
[1]
Respiratory events are defined as any increase in respiratory symptoms (increase sputum production, increase in cough, runny or stuffy nose, a cold, decrease in lung function, and/or change in physical findings of the respiratory system).
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Sitagliptin Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   16/17 (94.12%)      16/16 (100.00%)    
Gastrointestinal disorders     
Constipation *  1/17 (5.88%)  2 0/16 (0.00%)  0
Diarrhea *  1/17 (5.88%)  1 3/16 (18.75%)  3
Gastritis *  0/17 (0.00%)  0 1/16 (6.25%)  1
Heartburn *  0/17 (0.00%)  0 1/16 (6.25%)  1
Intestinal blockage *  0/17 (0.00%)  0 1/16 (6.25%)  1
Stomach blockage with abdominal pain *  1/17 (5.88%)  1 0/16 (0.00%)  0
Nausea/vomiting *  2/17 (11.76%)  2 0/16 (0.00%)  0
Vomiting and diarrhea *  2/17 (11.76%)  2 0/16 (0.00%)  0
General disorders     
Abdominal pain *  2/17 (11.76%)  2 2/16 (12.50%)  3
Abnormal lab results *  0/17 (0.00%)  0 1/16 (6.25%)  1
Decreased appetite *  1/17 (5.88%)  1 0/16 (0.00%)  0
Difficulty sleeping *  1/17 (5.88%)  2 0/16 (0.00%)  0
Elevated bilirubin *  1/17 (5.88%)  1 0/16 (0.00%)  0
Elevated CO2 *  0/17 (0.00%)  0 1/16 (6.25%)  1
Elevated eosinophils *  1/17 (5.88%)  1 0/16 (0.00%)  0
Elevated lipid panel/triglycerides *  0/17 (0.00%)  0 1/16 (6.25%)  2
Elevated prothrombin induced by vitamin K absence-II (PIVKA-II) *  1/17 (5.88%)  1 0/16 (0.00%)  0
Fatigue *  0/17 (0.00%)  0 2/16 (12.50%)  2
Fever *  2/17 (11.76%)  2 1/16 (6.25%)  1
Head tingling *  1/17 (5.88%)  1 0/16 (0.00%)  0
Increased thirst and urination *  0/17 (0.00%)  0 1/16 (6.25%)  1
Migraine headache *  1/17 (5.88%)  1 0/16 (0.00%)  0
sleeplessness, restlessness, bruising, and muscle tightness *  0/17 (0.00%)  0 1/16 (6.25%)  1
Tongue numbness due to colistin *  1/17 (5.88%)  2 0/16 (0.00%)  0
Weight loss *  1/17 (5.88%)  1 3/16 (18.75%)  3
Infections and infestations     
Athletes foot *  1/17 (5.88%)  1 0/16 (0.00%)  0
Ehrlichiosis *  1/17 (5.88%)  1 0/16 (0.00%)  0
Tooth abcess *  0/17 (0.00%)  0 1/16 (6.25%)  1
Urinary tract infection *  1/17 (5.88%)  1 1/16 (6.25%)  1
Yeast infection *  1/17 (5.88%)  1 2/16 (12.50%)  2
Musculoskeletal and connective tissue disorders     
Ankle injury *  0/17 (0.00%)  0 1/16 (6.25%)  1
Back pain *  1/17 (5.88%)  1 0/16 (0.00%)  0
Broken/fractured hand bones *  0/17 (0.00%)  0 1/16 (6.25%)  2
Spinal compression fracture *  1/17 (5.88%)  1 0/16 (0.00%)  0
Foot pain *  1/17 (5.88%)  1 2/16 (12.50%)  2
Joint pain *  1/17 (5.88%)  1 0/16 (0.00%)  0
Joint stiffness in hands *  1/17 (5.88%)  1 0/16 (0.00%)  0
Knee pain *  1/17 (5.88%)  1 1/16 (6.25%)  1
Myositis *  1/17 (5.88%)  1 0/16 (0.00%)  0
Neck pain from pulled muscle *  1/17 (5.88%)  1 0/16 (0.00%)  0
Open wound to left forearm *  0/17 (0.00%)  0 1/16 (6.25%)  1
Right knee contusion with edema *  1/17 (5.88%)  1 0/16 (0.00%)  0
Shoulder pain *  1/17 (5.88%)  1 1/16 (6.25%)  1
Tendinitis due to levaquin *  1/17 (5.88%)  1 0/16 (0.00%)  0
Psychiatric disorders     
Anxiety *  2/17 (11.76%)  3 0/16 (0.00%)  0
Depression *  2/17 (11.76%)  2 1/16 (6.25%)  1
Renal and urinary disorders     
Acute kidney injury *  0/17 (0.00%)  0 1/16 (6.25%)  1
Elevated creatinine *  1/17 (5.88%)  1 2/16 (12.50%)  3
Elevated potassium *  1/17 (5.88%)  1 0/16 (0.00%)  0
Hematuria *  1/17 (5.88%)  1 0/16 (0.00%)  0
Kidney stones with colic *  0/17 (0.00%)  0 1/16 (6.25%)  1
Reproductive system and breast disorders     
Bacterial vaginosis *  1/17 (5.88%)  1 0/16 (0.00%)  0
hydroceles with scrotal pain *  0/17 (0.00%)  0 1/16 (6.25%)  1
Prostratitis with hematuria *  0/17 (0.00%)  0 1/16 (6.25%)  1
Respiratory, thoracic and mediastinal disorders     
Respiratory event * [1]  16/17 (94.12%)  63 16/16 (100.00%)  109
Skin and subcutaneous tissue disorders     
Psoriasis *  0/17 (0.00%)  0 1/16 (6.25%)  1
Rash *  1/17 (5.88%)  3 1/16 (6.25%)  1
Sebaceous cyst, carbuncle on back *  1/17 (5.88%)  1 0/16 (0.00%)  0
Skin irritation from colistin *  0/17 (0.00%)  0 1/16 (6.25%)  1
Surgical and medical procedures     
Complications with PICC line *  3/17 (17.65%)  3 0/16 (0.00%)  0
Complications with port *  1/17 (5.88%)  2 0/16 (0.00%)  0
Wisdom teeth extraction *  0/17 (0.00%)  0 2/16 (12.50%)  2
*
Indicates events were collected by non-systematic assessment
[1]
Respiratory events are defined as any increase in respiratory symptoms and/or signs (increase sputum production, increase in cough, runny or stuffy nose, a cold, decrease in lung function, and/or change in physical findings of the respiratory system)
Poor enrollment lead to early termination of the study and a small sample size for analyses. Equipment failure resulted in loss of serum samples for some of the secondary outcomes.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Arlene Stecenko, MD
Organization: Emory University
Phone: 404-712-2657
Responsible Party: Arlene Stecenko, Emory University
ClinicalTrials.gov Identifier: NCT00967798     History of Changes
Other Study ID Numbers: IRB00012724
1R01FD003527-01 ( U.S. FDA Grant/Contract )
First Submitted: August 27, 2009
First Posted: August 28, 2009
Results First Submitted: April 10, 2018
Results First Posted: June 25, 2018
Last Update Posted: June 25, 2018