A Phase 4 interventional study of Pancreatic Enzyme Replacement Therapy and Placebo in Chronic Pancreatitis, Recurrent Acute Pancreatitis and Exocrine Pancreatic Insufficiency, sponsored by Anna Evans Phillips. Recruiting at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by Anna Evans Phillips · Phase 4, Interventional, and Treatment
This project uses the Malabsorption Blood Test (MBT) to identify patients with recurrent acute or chronic pancreatitis who have mild to moderate exocrine pancreatic insufficiency. A subgroup of patients who have response to pancreatic enzyme replacement therapy will enter a randomized, placebo-controlled pilot clinical trial for 8 weeks to identify improvements in quality of life (QOL).
This proposal uses a blood test detection method for EPI specifically designed to detect fat malabsorption in the setting of inadequate release of pancreatic digestive enzymes. The purpose of using it in this study is to identify mild and moderate EPI, for which to date no reliable test exists. The MBT evaluates the absorption of heptadecanoic acid (HA), a fatty acid dependent on lipase to release it from more complex form before it can be absorbed. Steatorrhea (fatty diarrhea) is a symptom present in over half of subjects with severe EPI, but is frequently not present in mild or moderate forms of EPI. In the absence of overt steatorrhea, there exists no reliable test to detect mild or moderate EPI in subjects with RAP or CP or track response to treatment. Without detection, RAP and CP subjects are at risk for malnutrition if they cannot properly absorb dietary fat and nutrients, and simultaneously significant weight loss, sarcopenia, osteopathy, nutritional deficiencies, GI symptoms and QOL. There is a clear medical need to identify subjects with pancreatic fat malabsorption who will benefit from treatment for EPI - pancreatic enzyme replacement therapy (PERT). In the proposed work, the investigators will enroll 80 subjects with RAP or CP who do not have steatorrhea or known severe EPI, and perform the MBT before and after 5 days of PERT therapy to identify subjects with fat malabsorption responsive to PERT. The investigators will assess clinical factors that correlate with PERT-responsive fat malabsorption. The primary outcome for assessment of the MBT results will be prevalence of PERT-responsive fat malabsorption. It is anticipated that 33% of subjects will have PERT-responsive fat malabsorption. The investigators will then sequentially enroll 24 PERT-responders to an 8-week pilot randomized placebo-controlled clinical trial of PERT supplementation (144,000 lipase units per day) versus placebo to determine the effects on QOL. The hypothesis is that PERT will result in improvement of QOL defined by a positive change from baseline in the PROMIS 29+2 in PERT-responders. PROMIS Gastrointestinal Scales will be assessed as secondary outcomes. Change in the results of a short physical performance battery and changes in body morphology (weight, BMI) from beginning of the study will be assessed in an exploratory fashion for correlation with PERT administration versus placebo.
Exclusion Criteria
Participants will undergo the MBT off PERT followed by the MBT on PERT. They will not be enrolled in the randomized trial.
Diagnostic Test: MBT1 · Diagnostic Test: MBT 2
Participants will undergo MBT on PERT followed by MBT off PERT. Participants will not be enrolled in the randomized clinical trial.
Diagnostic Test: MBT1 · Diagnostic Test: MBT 2
Participants will undergo MBT off PERT followed by MBT on PERT, and subsequently be randomized to the clinical trial arm treated with PERT.
Drug: Pancreatic Enzyme Replacement Therapy · Diagnostic Test: MBT1 · Diagnostic Test: MBT 2
Participants will undergo MBT off PERT followed by MBT on PERT, and subsequently be randomized to the clinical trial arm treated with placebo.
Drug: Placebo · Diagnostic Test: MBT1 · Diagnostic Test: MBT 2
Participants will undergo MBT on PERT followed by MBT off PERT, and subsequently be randomized to the clinical trial arm treated with PERT.
Drug: Pancreatic Enzyme Replacement Therapy · Diagnostic Test: MBT1 · Diagnostic Test: MBT 2
Participants will undergo MBT on PERT followed by MBT off PERT, and subsequently be randomized to the clinical trial arm treated with placebo.
Drug: Placebo · Diagnostic Test: MBT1 · Diagnostic Test: MBT 2
12 participants who are PERT responders in the MBT will be randomized to receive 8 weeks of PERT (144,000 lipase units daily)
12 participants who are PERT responders in the MBT will be assigned to receive 8 weeks of placebo therapy
MBT off PERT
MBT on PERT
Primary Outcome (Aim 1)
The primary outcome of the first phase of the study will be the prevalence of responders to PERT by assessing Heptadecanoic Acid absorption following 5-days of PERT therapy compared to baseline (no PERT therapy). This will be measured by a positive area under the curve of the difference between absorption curves for Triheptadecanoic Acid and Pentadecanoic Acid.
Time frame: 2 weeks
Primary Outcome (Aim 2)
The primary outcome of the second phase of the study will be change in the Overall Quality of Life Score of the Patient Reported Outcomes Measurement Systems (PROMIS) 29 + 2 questionnaire for subjects receiving PERT compared to placebo.
Time frame: Baseline (at time of entry to RCT) to completion of RCT, 8 weeks
Secondary Outcome: PROMIS Gastrointestinal Symptom Score for Belly Pain
The PROMIS gastrointestinal symptoms short form for belly pain will be used to track symptoms of EPI over the course of the 8 week trial and change in this will be assessed comparing the cohorts receiving PERT and placebo. Range 0-25. Higher score equals worse belly pain.
Time frame: 8 weeks
Secondary Outcome: PROMIS Gastrointestinal Scale for Bowel Incontinence
The PROMIS gastrointestinal symptoms short form for bowel incontinence will be used to track symptoms of EPI over the course of the 8 week trial. This will be assessed as a secondary outcomes, comparing change between the cohorts receiving PERT and placebo. Range 4-20. Higher score equals more bowel incontinence.
Time frame: 8 weeks
Secondary Outcome: PROMIS Gastrointestinal Scale for Constipation
The PROMIS gastrointestinal symptoms short form for constipation will be used to track symptoms of EPI over the course of the 8 week trial. This will be assessed as a secondary outcome, comparing change between the cohorts receiving PERT and placebo. Ragne 8-45. Higher score equals worse constipation.
Time frame: 8 weeks
Secondary Outcome: PROMIS Gastrointestinal Scale for Diarrhea
The PROMIS gastrointestinal symptoms short form for diarrhea will be used to track symptoms of EPI over the course of the 8 week trial. This will be assessed as a secondary outcome, comparing change between the cohorts receiving PERT and placebo. Range 5-30. Higher score equals worse diarrhea.
Time frame: 8 weeks
Secondary Outcome: PROMIS Gastrointestinal Scale for Nausea and Vomiting
The PROMIS gastrointestinal symptoms short form for nausea and vomiting will be used to track symptoms of EPI over the course of the 8 week trial. This will be assessed as a secondary outcome, comparing change between the cohorts receiving PERT and placebo. Range 3-20. Higher score equals worse nausea and vomiting.
Time frame: 8 weeks
Secondary Outcome: PROMIS Gastrointestinal Scale for Gas and Bloating
The PROMIS gastrointestinal symptoms short form for gas and bloating will be used to track symptoms of EPI over the course of the 8 week trial. This will be assessed as a secondary outcome, comparing change between the cohorts receiving PERT and placebo. Ragne 4-65. Higher score equals worse gas and bloating.
Time frame: 8 weeks
Plan to share: No — Individual participant data is not planning to be shared except in conjunction with any NIH or NIDDK policies that include this requirement, in which case all active policies will be followed.
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Anna Evans Phillips