Open label randomized, multicentre, controlled trial of pancreatic enzyme replacement therapy (PERT) for pancreatic exocrine insufficiency (PEI) in patients with unresectable pancreatic cancer.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

14 Feb 2024 → ongoing

Decision date (initial)

2024-09-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Viatris

External identifiers

EU CT number

2023-507367-18-00

EudraCT number

2021-005874-24

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

As hypothesis, PERT in patients with unresectable pancreatic cancer is associated with weight gain and improved nutritional status, which, in turn, improves the performance status of patients, quality of life and tolerance to chemotherapy. This approach could have therefore a benefit on survival.
To test this hypothesis, the primary objective of the study is to evaluate the impact of PERT on body weight in patients with unresectable pancreatic cancer.

Secondary objectives 2

1. To compare the impact of PERT on the nutritional status of patients with unresectable pancreatic cancer in terms of: o Patient-Generated Subjective Global Assessment (PG-SGA). o Nutritional and other biochemical markers: haemoglobin, lymphocyte count, Protocolo PERTseverance (PEI004/2021) Versión 3.0 de 25 de mayo de 2023 España Página 17 de 45 serum total protein, albumin, prealbumin, retinol binding protein, magnesium, selenium, zinc, fat-soluble vitamins (vitamin E and D), total and HDL cholesterol, triglycerides, and haemoglobin A1c (HbA1c).
2. To compare the impact of PERT in patients with unresectable pancreatic cancer on: o Karnofsky and ECOG performance status. o Clinical symptoms related to malabsorption (i.e., abdominal pain, stool consistency, stool frequency, abdominal distention and cramps, flatulence, PEI-Q). o Quality of life (EORTC QLQ-PAN26). o Tolerance to chemotherapy in terms of percentage of dose administered in relation to the optimal full dose.

Conditions and MedDRA coding

Pancreatic exocrine insufficiency

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Patients older than 18 years, any gender, fulfilling all the following criteria will be considered for enrolment into the study
2. Pathologically confirmed unresectable, locally advanced or metastatic, pancreatic cancer.
3. Tumour located in the head of the pancreas
4. Dilated main pancreatic duct confirmed by imaging methods (CT scan, MRI and/or EUS)
5. Significant weight loss (≥5% of the usual body weight, over the last 6 months) at screening
6. Life expectancy of at least six months at screening
7. Signed informed consent to the study
8. Patients with Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2.

Exclusion criteria 13

1. Hypersensitivity to pancreatin of porcine origin or to any of the excipients
2. Inability to comply with the study visits and study protocol, whatever the reason
3. Patients on neoadjuvant therapy, or in whom neoadjuvant therapy is planned
4. Patients already on PERT
5. Prior history of upper gastrointestinal or pancreatic surgery
6. Short life expectancy (shorter than 6 months)
7. Patients on second line or beyond chemotherapy (those who failed with first line chemotherapy therapy)
8. Patients in whom a pancreatic stent has been placed
9. Unsolved gastric outlet obstruction
10. Unwillingness to participate in the study
11. Patients with body weight <46 kg
12. Patients with pancreatitis in the acute phase.
13. Patients with contraindications to PPI concomitant treatment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is defined as the change in body weight (kg and percentage) from baseline to the end of the controlled phase, 3 months from inclusion, as well as to the end of the study, 6 months from inclusion, in the two groups of patients.

Secondary endpoints 6

1. Change in haemoglobin, lymphocytes, serum concentration of nutritional markers (albumin, prealbumin, retinol binding protein, transferrin, fat-soluble vitamins, total and HDL cholesterol, triglycerides, cholinesterase, magnesium, selenium, and zinc), HbA1C and C-reactive protein (CRP) from baseline to the end of the controlled phase (3 months) and the end of the study (6 months) in the two groups of patients
2. Change in Karnofsky performance status from baseline to the end of the controlled phase and the end of the study in the two groups of patients
3. Change in malabsorption-related symptoms (diarrhoea, flatulence, abdominal distention, meteorism, abdominal cramps) and Patient-Generated Subjective Global Assessment (PG-SGA) from baseline to the end of the controlled phase and the end of the study in the two groups of patients
4. Change in quality of life (EORTC QLQ-PAN26) from baseline to the end of the controlled phase and the end of the study in the two groups of patients
5. Tolerance to chemotherapy defined as the percentage of the optimal dose of chemotherapeutic agents administered at months 3 and 6 from inclusion
6. Survival over 3 and 6 months from inclusion

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela

Sponsor organisation

Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela

Address

Travesia Da Choupana S/n

City

Santiago De Compostela

Postcode

15706

Country

Spain

Scientific contact point

Organisation

Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela

Contact name

Juan Enrique Domínguez-Muñoz

Public contact point

Organisation

Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela

Contact name

Juan Enrique Domínguez-Muñoz

Third parties 1

Locations

3 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 17 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

11 submissions — initial application plus substantial / non-substantial modifications