Exocrine Pancreatic Insufficiency (EPI)
Conditions
Keywords
Exocrine Pancreatic Insufficiency (EPI), Pancreatic Cancer, Creon, Pancrelipase
Brief summary
This is a study in participants with Exocrine Pancreatic Insufficiency (EPI) due to pancreatic cancer. This study will include resected participants who are post pancreatic cancer surgery, and an additional cohort in non-resected participants.
Interventions
DRUGPancrelipase
Pancrelipase is administered orally as capsules with a meal or snack
DRUGPlacebo
Placebo is administered orally as capsules with a meal or snack
Sponsors
AbbVie
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
21 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant has diagnosed cancer of pancreas with biopsy and/or radiography, with a life expectancy of at least 5 months at screening * Participant's pancreatic cancer must involve the head and/or neck of the pancreas * Confirmed exocrine pancreatic insufficiency (EPI) as evidenced by fecal elastase-1 (FE-1) ≤ 150 µg/g stool at screening * A positive Sudan stain for participants without history of fat malabsorption (fat malabsorption is defined as clinical steatorrhea, or measured stool fat \> 7 g/day, or positive stool results by Sudan stain) within 1 week of screening -- Positive stool results are defined as increased level of neutral OR total fats
Exclusion criteria
* Participant has neuroendocrine pancreatic cancer * Participant has fibrosing colonopathy * Participant has any other malignancy within 1 year of screening * Participant has uncontrolled gout, including those with a recent flare within 60 days of screening * Participant has other significant organ or bone marrow abnormality within 60 days of screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Stool Fat From Baseline (Day 1) to Week 1 (Day 8) Among Participants With Resected Pancreatic Cancer | Baseline (Day 1), Week 1 (Day 8) | Stool samples were collected during the 48 hours prior to the Day 1 and Week 1 visits and analyzed for fat content. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Average Daily Stool Frequency From Baseline (Day 1) to Week 1 (Day 8) Among Participants With Resected Pancreatic Cancer | Baseline (Day 1), Week 1 (Day 8) | Participants recorded stool frequency using an electronic diary (eDiary). The average daily stool frequency was calculated from the last 3 days prior to the Baseline and Week 1 visits. |
| Change in Stool Consistency From Baseline to Week 1 Among Participants With Resected Pancreatic Cancer | Baseline (Day 1), Week 1 (Day 8) | Participants recorded stool consistency using an electronic diary (eDiary). The change from Baseline to Week 1 is the proportion of days having watery stool consistency in the last 7 days prior to each of Baseline and Week 1 visits. Negative changes from Baseline indicate less frequent watery stools. |
| Change in the Total EPI Symptoms Score From Baseline to Week 1 Among Participants With Resected Pancreatic Cancer | Baseline (Day 1), Week 1 (Day 8) | The EPI Symptoms Questionnaire consists of 12 questions. The response scores range from 0 to 4 for each question (0 corresponding to None to 4 corresponding to Very Severe), with the total score ranging from 0 to 48. Positive changes indicate worsening from Baseline. |
Countries
United States
Participant flow
Pre-assignment details
Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Neither the Resected Participants Receiving Low Dose Pancrelipase group nor the Non-Resected Participants Receiving High Dose Pancrelipase group enrolled any participants.
Participants by arm
| Arm | Count |
|---|---|
| Resected Participants Receiving Low Dose (12,000/6,000 Units Lipase) Pancrelipase Resected participants (surgery to remove pancreatic cancer) will first be given low dose pancrelipase. Low dose is defined as 12,000 USP units (lipase) with meals and 6000 U with snacks. At Weeks 1, 5, or 9, participants will be evaluated, and those who meet criteria for dose increase will be given high dose pancrelipase. High dose is defined as 72,000 U with meals and 36,000 U with snacks. All participants will receive placebo as needed to keep the number of pills the same in each group and for blinding. | 0 |
| Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase Resected participants (surgery to remove pancreatic cancer) will receive and continue on high dose pancrelipase throughout the study. High dose is defined as 72,000 USP units (lipase) with meals and 36,000 U with snacks. All participants will receive placebo as needed to keep the number of pills the same in each group and for blinding. | 1 |
| Non-Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase Non-resected participants (those who did not have surgery to remove pancreatic cancer) will receive high dose pancrelipase throughout the study in an open-label cohort. High dose is defined as 72,000 USP units (lipase) with meals and 36,000 U with snacks. | 0 |
| Total | 1 |
Baseline characteristics
| Characteristic | Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase | Non-Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase | Total | Resected Participants Receiving Low Dose (12,000/6,000 Units Lipase) Pancrelipase |
|---|---|---|---|---|
| Age, Customized Age 21 - 90 years old | 1 Participants | 0 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | — | 0 Participants | — |
| Race (NIH/OMB) Asian | 0 Participants | — | 0 Participants | — |
| Race (NIH/OMB) Black or African American | 0 Participants | — | 0 Participants | — |
| Race (NIH/OMB) More than one race | 0 Participants | — | 0 Participants | — |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | — | 0 Participants | — |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | — | 0 Participants | — |
| Race (NIH/OMB) White | 1 Participants | — | 1 Participants | — |
| Sex: Female, Male Female | 0 Participants | — | 0 Participants | — |
| Sex: Female, Male Male | 1 Participants | — | 1 Participants | — |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 0 | 0 / 1 | 0 / 0 |
| other Total, other adverse events | 0 / 0 | 1 / 1 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 1 | 0 / 0 |
Outcome results
Primary
Change in Stool Fat From Baseline (Day 1) to Week 1 (Day 8) Among Participants With Resected Pancreatic Cancer
Stool samples were collected during the 48 hours prior to the Day 1 and Week 1 visits and analyzed for fat content.
Time frame: Baseline (Day 1), Week 1 (Day 8)
Population: Participants who received at least one dose of study drug and who had evaluable stool fat at both Baseline and Week 1 visits
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase | Change in Stool Fat From Baseline (Day 1) to Week 1 (Day 8) Among Participants With Resected Pancreatic Cancer | 3.1 g/24 hours |
Secondary
Change in Average Daily Stool Frequency From Baseline (Day 1) to Week 1 (Day 8) Among Participants With Resected Pancreatic Cancer
Participants recorded stool frequency using an electronic diary (eDiary). The average daily stool frequency was calculated from the last 3 days prior to the Baseline and Week 1 visits.
Time frame: Baseline (Day 1), Week 1 (Day 8)
Population: Participants who received at least one dose of study drug
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase | Change in Average Daily Stool Frequency From Baseline (Day 1) to Week 1 (Day 8) Among Participants With Resected Pancreatic Cancer | NA number of stools/day |
Secondary
Change in Stool Consistency From Baseline to Week 1 Among Participants With Resected Pancreatic Cancer
Participants recorded stool consistency using an electronic diary (eDiary). The change from Baseline to Week 1 is the proportion of days having watery stool consistency in the last 7 days prior to each of Baseline and Week 1 visits. Negative changes from Baseline indicate less frequent watery stools.
Time frame: Baseline (Day 1), Week 1 (Day 8)
Population: Participants who received at least one dose of study drug
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase | Change in Stool Consistency From Baseline to Week 1 Among Participants With Resected Pancreatic Cancer | -0.29 proportion of days w/ watery consistency |
Secondary
Change in the Total EPI Symptoms Score From Baseline to Week 1 Among Participants With Resected Pancreatic Cancer
The EPI Symptoms Questionnaire consists of 12 questions. The response scores range from 0 to 4 for each question (0 corresponding to None to 4 corresponding to Very Severe), with the total score ranging from 0 to 48. Positive changes indicate worsening from Baseline.
Time frame: Baseline (Day 1), Week 1 (Day 8)
Population: Participants who received at least one dose of study drug
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Resected Participants Receiving High Dose (72,000/36,000 Units Lipase) Pancrelipase | Change in the Total EPI Symptoms Score From Baseline to Week 1 Among Participants With Resected Pancreatic Cancer | 3 units on a scale |