Chronic Pancreatitis, Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, Chronic Pancreatitis, Exocrine Pancreatic Insufficiency, Creon, Pancrelipase
Brief summary
Exocrine pancreatic insufficiency (EPI) is a condition that is caused by the inadequate pancreatic enzymes needed for normal digestion and is commonly associated with a wide range of chronic diseases, including cystic fibrosis (CF), chronic pancreatitis (CP), and pancreatic cancer. This study will assess clinical symptoms when participants with CF or CP are treated with Creon with alternate source of active drug. Creon is an approved drug for the treatment of EPI. This study is subject-blinded which means participants will not know the source of the study drug they are given. Approximately 30 adult participants with CF or CP will be enrolled at approximately 15 sites across the Unites States. Participants will receive oral capsules of CREON for 113 days and will be followed for 30 days. Participants will attend regular visits during the study at a hospital or clinic or via telemedicine. The effect of the treatment will be checked by medical assessments, checking for side effects and completing questionnaires.
Interventions
DRUGCREON
Capsule; Oral
Sponsors
AbbVie
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Previous diagnosis of cystic fibrosis (CF) or chronic pancreatitis (CP). * Previous diagnosis of exocrine pancreatic insufficiency (EPI) that is currently clinically controlled. * Total Symptom Score (TSS) \< 1.8 on Pancreatic Exocrine Insufficiency Questionnaire (PEI-Q) at Screening.
Exclusion criteria
\- Malignancy involving the digestive tract in the last 5 years, or other significant disease or medical condition that may interfere with EPI symptom assessment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Day 1 (baseline), 8, 15, 29, and 85 | The Pancreatic Exocrine Insufficiency Questionnaire (PEI-Q) is an 18-item Patient-Reported Outcome (PRO) instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. TSS is calculated from the average of the mean abdominal domain score and the mean bowel movement symptom score. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms. |
| Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Day 1 (Baseline), 8, 15, 29, and 85 | The PEI-Q is an 18-item PRO instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. ASDS is the mean abdominal symptom domain score measured by the PEI-Q. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms. |
| Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Day 1 (Baseline), 8, 15, 29, and 85 | The PEI-Q is an 18-item PRO instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. BMSS is the mean bowel movement symptom domain score measured by the PEI-Q. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms. |
Countries
United States
Participant flow
Recruitment details
Thirty participants were enrolled at 13 sites throughout the United States. During the run-in period, 30 participants were treated and completed 28 days of treatment with Creon-ABT.
Pre-assignment details
One participant failed continuation criteria, with the remaining 29 participants continuing on to the treatment period with Creon-AAPIS. The Per Protocol Population included all enrolled subjects who received at least 1 dose of Creon-AAPIS, had Day 1 (Baseline) PEI-Q scores collected, and had at least one post-baseline PEI-Q scores collected during the Treatment Period (Days 8, 15, 29, and 85).
Participants by arm
| Arm | Count |
|---|---|
| Treatment: Creon-AAPIS Participants who completed the Run-in Period were assessed for the continuation criteria at Day 1. Blinded Creon-ABT study drug from the Run-In Period was returned, and the participant was dispensed blinded Creon-AAPIS to use at the same stable individual dose per LU of Creon he/she was on before screening and during the Run-in Period. This assured that the treatment assessments after Day 1 reflect the symptoms on Creon-AAPIS. | 29 |
| Total | 29 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Treatment Period (Day 1 to Day 85) | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | Treatment: Creon-AAPIS |
|---|---|
| Age, Continuous | 44.6 Years STANDARD_DEVIATION 16.77 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 28 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| 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 | 29 Participants |
| Region of Enrollment United States | 29 participants |
| Sex: Female, Male Female | 14 Participants |
| Sex: Female, Male Male | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 30 | 0 / 29 |
| other Total, other adverse events | 1 / 30 | 9 / 29 |
| serious Total, serious adverse events | 0 / 30 | 2 / 29 |
Outcome results
Primary
Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
The PEI-Q is an 18-item PRO instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. ASDS is the mean abdominal symptom domain score measured by the PEI-Q. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms.
Time frame: Day 1 (Baseline), 8, 15, 29, and 85
Population: The Per Protocol Population included all enrolled subjects who received at least 1 dose of study drug of Creon-AAPIS, had Day 1 (Baseline) PEI-Q scores collected, and had at least one post-baseline PEI-Q scores collected during the Treatment Period (Days 8, 15, 29, and 85). The pharmacodynamic endpoints were analyzed in the Per Protocol Population. Number analyzed indicates the number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Treatment: Creon-AAPIS | Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from baseline to Day 8 | 0.0582 score on a scale | Standard Deviation 0.3722 |
| Treatment: Creon-AAPIS | Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from baseline to Day 15 | 0.0530 score on a scale | Standard Deviation 0.4605 |
| Treatment: Creon-AAPIS | Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from baseline to Day 29 | 0.1905 score on a scale | Standard Deviation 0.61 |
| Treatment: Creon-AAPIS | Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from baseline to Day 85 | 0.0893 score on a scale | Standard Deviation 0.519 |
Primary
Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
The PEI-Q is an 18-item PRO instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. BMSS is the mean bowel movement symptom domain score measured by the PEI-Q. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms.
Time frame: Day 1 (Baseline), 8, 15, 29, and 85
Population: The Per Protocol Population included all enrolled subjects who received at least 1 dose of study drug of Creon-AAPIS, had Day 1 (Baseline) PEI-Q scores collected, and had at least one post-baseline PEI-Q scores collected during the Treatment Period (Days 8, 15, 29, and 85). The pharmacodynamic endpoints were analyzed in the Per Protocol Population. Number analyzed indicates the number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Treatment: Creon-AAPIS | Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 8 | -0.0555 score on a scale | Standard Deviation 0.3235 |
| Treatment: Creon-AAPIS | Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 15 | 0.0432 score on a scale | Standard Deviation 0.524 |
| Treatment: Creon-AAPIS | Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 29 | 0.1111 score on a scale | Standard Deviation 0.5565 |
| Treatment: Creon-AAPIS | Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 85 | 0.0695 score on a scale | Standard Deviation 0.7 |
Primary
Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
The Pancreatic Exocrine Insufficiency Questionnaire (PEI-Q) is an 18-item Patient-Reported Outcome (PRO) instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. TSS is calculated from the average of the mean abdominal domain score and the mean bowel movement symptom score. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms.
Time frame: Day 1 (baseline), 8, 15, 29, and 85
Population: The Per Protocol Population included all enrolled subjects who received at least 1 dose of study drug of Creon-AAPIS, had Day 1 (Baseline) PEI-Q scores collected, and had at least one post-baseline PEI-Q scores collected during the Treatment Period (Days 8, 15, 29, and 85). The pharmacodynamic endpoints were analyzed in the Per Protocol Population. Number analyzed indicates the number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Treatment: Creon-AAPIS | Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 8 | 0.0012 score on a scale | Standard Deviation 0.2953 |
| Treatment: Creon-AAPIS | Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 15 | 0.0480 score on a scale | Standard Deviation 0.4354 |
| Treatment: Creon-AAPIS | Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 29 | 0.1506 score on a scale | Standard Deviation 0.5396 |
| Treatment: Creon-AAPIS | Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Change from Baseline to Day 85 | 0.0791 score on a scale | Standard Deviation 0.5298 |