Trial Outcomes & Findings for Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon (NCT NCT05069597)

Last Updated: 2024-07-23

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

30 participants

Primary outcome timeframe

Day 1 (baseline), 8, 15, 29, and 85

Results posted on

2024-07-23

Participant Flow

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.

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).

Participant milestones

Participant milestones
Measure	Run-in: Creon-ABT Participants received blinded Creon-ABT for the duration of the Run-in Period (28 days) at the same stable individual dose of Creon he/she was on before screening. This assured that the Day 1 assessment reflected the symptoms on stable Creon-ABT use.	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.
Run-in Period (Day -28 to Day 0) STARTED	30	0
Run-in Period (Day -28 to Day 0) COMPLETED	30	0
Run-in Period (Day -28 to Day 0) NOT COMPLETED	0	0
Treatment Period (Day 1 to Day 85) STARTED	0	29
Treatment Period (Day 1 to Day 85) COMPLETED	0	28
Treatment Period (Day 1 to Day 85) NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Run-in: Creon-ABT Participants received blinded Creon-ABT for the duration of the Run-in Period (28 days) at the same stable individual dose of Creon he/she was on before screening. This assured that the Day 1 assessment reflected the symptoms on stable Creon-ABT use.	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.
Treatment Period (Day 1 to Day 85) Withdrawal by Subject	0	1

Baseline Characteristics

Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment: Creon-AAPIS n=29 Participants 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.
Age, Continuous	44.6 Years STANDARD_DEVIATION 16.77 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	29 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	29 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment: Creon-AAPIS n=29 Participants 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.
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
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
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
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

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment: Creon-AAPIS n=29 Participants 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.
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
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
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 0.6100
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 0.5190

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment: Creon-AAPIS n=29 Participants 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.
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
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 0.5240
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
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 0.7000

Adverse Events

Run-in: Creon-ABT

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Treatment: Creon-AAPIS

Serious events: 2 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Run-in: Creon-ABT n=30 participants at risk Participants received blinded Creon-ABT for the duration of the Run-in Period (28 days) at the same stable individual dose of Creon he/she was on before screening. This assured that the Day 1 assessment reflected the symptoms on stable Creon-ABT use.	Treatment: Creon-AAPIS n=29 participants at risk 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.
Gastrointestinal disorders INTESTINAL OBSTRUCTION	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	3.4% 1/29 • Number of events 1 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Injury, poisoning and procedural complications ALCOHOL POISONING	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	3.4% 1/29 • Number of events 2 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Injury, poisoning and procedural complications FEMUR FRACTURE	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	3.4% 1/29 • Number of events 1 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Psychiatric disorders ALCOHOL ABUSE	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	3.4% 1/29 • Number of events 1 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.

Other adverse events

Other adverse events
Measure	Run-in: Creon-ABT n=30 participants at risk Participants received blinded Creon-ABT for the duration of the Run-in Period (28 days) at the same stable individual dose of Creon he/she was on before screening. This assured that the Day 1 assessment reflected the symptoms on stable Creon-ABT use.	Treatment: Creon-AAPIS n=29 participants at risk 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.
Gastrointestinal disorders ABDOMINAL DISTENSION	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	13.8% 4/29 • Number of events 7 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Gastrointestinal disorders ABDOMINAL PAIN	3.3% 1/30 • Number of events 1 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	17.2% 5/29 • Number of events 5 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Gastrointestinal disorders DIARRHOEA	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	20.7% 6/29 • Number of events 9 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Gastrointestinal disorders FLATULENCE	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	6.9% 2/29 • Number of events 5 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.
Infections and infestations VIRAL UPPER RESPIRATORY TRACT INFECTION	0.00% 0/30 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.	6.9% 2/29 • Number of events 3 • All-cause mortality and adverse event tables include events reported from enrollment to end of study. The median time participants were followed was 28 days for CREON-ABT and 116 days for CREON-AAPIS.

Additional Information

Global Medical Services

AbbVie

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Email: [email protected]

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Principal investigator is a sponsor employee AbbVie requests that any investigator or institution that plans on presenting/publishing results disclosure, provide written notification of their request 60 days prior to their presentation/publication. AbbVie requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if AbbVie needs to secure patent or proprietary protection.
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