Cystic Fibrosis, Cystic Fibrosis-related Diabetes
Conditions
Keywords
CFRD
Brief summary
This open label, single arm pilot study, will examine the safety and tolerability of GLP-1RA semaglutide as an add-on therapy to insulin for overweight/obese adult patients with cystic fibrosis related diabetes (CFRD).
Detailed description
The overall goal of this proposal is to collect pilot data for safety and feasibility metrics to support a future larger randomized controlled trial. In this study, we will examine the safety and tolerability of GLP-1RA semaglutide as an add-on therapy to insulin for overweight/obese adult patients with CFRD. We hypothesize that weekly administration of the long-acting GLP-1RA semaglutide to overweight/obese CFRD patients will be safe and well tolerated. Specific Aim 1: Collect pilot data on the safety and feasibility of weekly semaglutide therapy in overweight and obese patients with CFRD to support a future larger randomized controlled trial. Hypothesis 1: Weekly therapy with GLP-1RA semaglutide will be safe and well tolerated in overweight/obese adults with CFRD. Specific Aim 2: Collect preliminary data to examine the impact of semaglutide therapy on insulin secretion, glucagon and glucose levels as measured by oral glucose tolerance test (OGTT), and on glycemic outcomes as measured by continuous glucose monitoring (CGM) and HbA1c. Hypothesis 2a: Treatment with semaglutide will lower glucose levels, and increase insulin and C-peptide area under the curve (AUC) during the OGTT as compared to baseline. Hypothesis 2b: Treatment with semaglutide will improve glycemic control as indicated by time in range on CGM and HbA1c
Interventions
DRUGSemaglutide
glucagon-like peptide 1 (GLP-1) receptor agonist
Sponsors
University of Minnesota
Cystic Fibrosis Foundation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This open label, single arm pilot study, will examine the safety and tolerability of GLP-1RA semaglutide as an add-on therapy to insulin for overweight/obese adult patients with CFRD.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult subjects 18 years or older with CFRD and on insulin treatment * BMI \>26 kg/m2 * Diagnosis of pancreatic insufficiency (based on treatment with pancreatic enzyme replacement therapy) * A participant who is capable of becoming pregnant must agree to take precautions that are effective in preventing pregnancy throughout this study. These methods could include one of the following 1. Complete abstinence from sexual intercourse; 2. Oral, injectable, or implanted hormonal contraceptives 3. Intrauterine device 4. Tubal ligation
Exclusion criteria
* personal or family history of medullary thyroid cancer or multiple endocrine neoplasia syndrome type 2 (MEN2) * acute pulmonary exacerbation requiring IV antibiotics or systemic glucocorticoids within 4 weeks prior to baseline study procedures * gastrointestinal(GI) symptom exacerbation defined by current nausea/vomiting or diarrhea at the baseline assessment * history of chronic GI problems requiring hospitalization in the 1 year prior to baseline * history of clinically symptomatic pancreatitis * history of clinically significant gastroparesis * history of eating disorders * less than 24 weeks since start of a new CFTR corrector/modulator therapy * pregnancy or lactation * severe CF liver disease * chronic kidney disease * history of suicide attempts or active suicidal ideation * Non-English speakers and those unable to read in English
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Weight | 12 weeks | Change in weight before and 12 weeks post treatment |
| BMI (kg/m2) | 12 weeks | Change in BMI before and 12 weeks post treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| CGM % Time in Target TIR) | 12 weeks | Change in CGM TIR before and 12 weeks post treatment. Outcome reflects the change in continuous glucose monitoring (CGM) time in range (TIR), measured at baseline and again 12 weeks after treatment initiation. TIR is defined as the percentage of time glucose values are between 70 and 180 mg/dL |
| A1c | 12 weeks | change in the percent of HbA1c before and 12 weeks post treatment |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORAmir Moheet, MBBS
University of Minnesota
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 8 Participants |
| Age, Continuous | 40.4 years STANDARD_DEVIATION 5.2 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 8 Participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 7 / 8 |
| serious Total, serious adverse events | 0 / 8 |
Outcome results
None listed