ABTL0812 in Combination With FOLFIRINOX for First-line Treatment of Metastatic Pancreatic Study

NCT ID: NCT04431258

Last Updated: 2024-03-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-05-06

Study Completion Date

2024-01-10

Brief Summary

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A Phase I open label followed by a Phase II randomized, controlled study to assess the efficacy and safety of ABTL0812 in combination with FOLFIRINOX for first-line treatment of metastatic pancreatic. Funded by: FDA OOPD (Grant #FD-R-006817-01), H2020 EIC Accelerator (Grant #954825) and Ability Pharmaceuticals SL.

Detailed Description

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Phase I: This is an open label Phase I to determine the RP2D of ABTL0812 in combination with FOLFIRINOX. All patients will receive ABTL0812 in combination with FOLFIRINOX.

A dose de-escalation phase will be performed in which up to 3 different ABTL0812 dose levels will be tested in combination with FOLFIRINOX. ABTL0812 doses are: 1300 mg tid (starting dose), followed (if necessary) by 975 mg tid and 650 mg tid. Patient intra-escalation is not allowed.

Phase II: This is a double blind, randomized, placebo-controlled Phase II multicenter study to evaluate ABTL0812 in combination with FOLFIRINOX for first-line treatment of metastatic pancreatic cancer. Patients will be randomized to one of two groups: arm A) receiving ABTL0812 in addition to FOLFIRINOX and arm B) receiving FOLFIRINOX plus placebo.

Arm A) ABTL0812 + FOLFIRINOX Arm B) PLACEBO + FOLFIRINOX

Conditions

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Pancreatic Cancer

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double blind, randomized, placebo-controlled, multicenter study
Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Caregivers

Study Groups

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Arm A) ABTL0812 + FOLFIRINOX

FOLFIRINOX will be dosed according to the standard following regimen:

* oxaliplatin 85 mg/m2, administered as 2-hour iv infusion
* leucovorin 400 mg/m2, administered as 2-hour iv infusion
* irinotecan 180 mg/m2, administered as 1.5-hour iv infusion
* fluorouracil 2400 mg/m2, administered as 46-hour iv infusion every 2 weeks (=1 cycle) until disease progression or unacceptable toxicities.

ABTL0812 will be administered daily at its RP2D. ABTL0812 will be administered as single agent during a run-in period of one week before starting the first cycle of FOLFIRINOX, then daily during chemotherapy cycles. Also, ABTL0812 will be maintained once chemotherapy is discontinued, if ABTL0812 is tolerated and if the patient is in response or stable disease.

Group Type EXPERIMENTAL

ABTL0812

Intervention Type DRUG

ABTL0812 will be administered daily at its RP2D. ABTL0812 will be administered as single agent during a run-in period of one week before starting the first cycle of FOLFIRINOX, then daily during chemotherapy cycles. Also, ABTL0812 will be maintained once chemotherapy is discontinued, if ABTL0812 is tolerated and if the patient is in response or stable disease.

Folfirinox

Intervention Type DRUG

FOLFIRINOX will be dosed according to the standard following regimen:

* oxaliplatin 85 mg/m2, administered as 2-hour iv infusion
* leucovorin 400 mg/m2, administered as 2-hour iv infusion
* irinotecan 180 mg/m2, administered as 1.5-hour iv infusion
* fluorouracil 2400 mg/m2, administered as 46-hour iv infusionevery 2 weeks (=1 cycle) until disease progression or unacceptable toxicities.

Arm B) PLACEBO + FOLFIRINOX

FOLFIRINOX will be dosed according to the standard following regimen:

* oxaliplatin 85 mg/m2, administered as 2-hour iv infusion
* leucovorin 400 mg/m2, administered as 2-hour iv infusion
* irinotecan 180 mg/m2, administered as 1.5-hour iv infusion
* fluorouracil 2400 mg/m2, administered as 46-hour iv infusion every 2 weeks (=1 cycle) until disease progression or unacceptable toxicities.

Placebo will be administered at the same volume than ABTL0812 in arm A) FOLFIRINOX, then daily during chemotherapy cycles. Also, placebo will be maintained once chemotherapy is discontinued.

Group Type EXPERIMENTAL

ABTL0812

Intervention Type DRUG

ABTL0812 will be administered daily at its RP2D. ABTL0812 will be administered as single agent during a run-in period of one week before starting the first cycle of FOLFIRINOX, then daily during chemotherapy cycles. Also, ABTL0812 will be maintained once chemotherapy is discontinued, if ABTL0812 is tolerated and if the patient is in response or stable disease.

Placebo

Intervention Type DRUG

Placebo will be administered daily at the same regim as ABTL0812. Placebo will be administered as single agent during a run-in period of one week before starting the first cycle of FOLFIRINOX, then daily during chemotherapy cycles. Also, placebo will be maintained once chemotherapy is discontinued, if the patient is in response or stable disease.

Interventions

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ABTL0812

ABTL0812 will be administered daily at its RP2D. ABTL0812 will be administered as single agent during a run-in period of one week before starting the first cycle of FOLFIRINOX, then daily during chemotherapy cycles. Also, ABTL0812 will be maintained once chemotherapy is discontinued, if ABTL0812 is tolerated and if the patient is in response or stable disease.

Intervention Type DRUG

Folfirinox

FOLFIRINOX will be dosed according to the standard following regimen:

* oxaliplatin 85 mg/m2, administered as 2-hour iv infusion
* leucovorin 400 mg/m2, administered as 2-hour iv infusion
* irinotecan 180 mg/m2, administered as 1.5-hour iv infusion
* fluorouracil 2400 mg/m2, administered as 46-hour iv infusionevery 2 weeks (=1 cycle) until disease progression or unacceptable toxicities.

Intervention Type DRUG

Placebo

Placebo will be administered daily at the same regim as ABTL0812. Placebo will be administered as single agent during a run-in period of one week before starting the first cycle of FOLFIRINOX, then daily during chemotherapy cycles. Also, placebo will be maintained once chemotherapy is discontinued, if the patient is in response or stable disease.

Intervention Type DRUG

Other Intervention Names

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Chemotherapy

Eligibility Criteria

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Inclusion Criteria

1. Histologically or cytologically confirmed carcinoma, adenocarcinoma or ductal adenocarcinoma of the pancreas.
2. Confirmed metastatic disease
3. Measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 guidelines with at least one "target lesion" to be used to assess response. Tumors within a previously irradiated field will be designated as "non-target" lesions unless progression is documented.
4. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1.
5. Age, older than 18 years old
6. Adequate hematologic function, measured as:

* absolute neutrophil count ≥ 1.5x109/L
* platelet count ≥ 100x109/L without transfusion support
* hemoglobin ≥ 10 g/dL
7. Total bilirubin ≤ 1.5 x ULN
8. Albumin ≥ 3.3 g/dL
9. AST (SGOT) and ALT (SGPT) ≤ 2.5 times x upper limit of normal (≤ 5 times the ULN in patients with evidence of liver metastases)
10. Alkaline phosphatase ≤ 2.5 times ULN (≤5 times the ULN in patients with evidence of liver metastases)
11. Glomerular filtration rate (GFR) ≥ 60 mL/min/1.73 m2
12. Only for Phase II patients. If available, a sample of tumor tissue or cytology (either archival or new tumor biopsy) for biomarker analyses. The most recently collected tumor tissue sample should be provided.
13. Contraception: All premenopausal female patients must use contraception. Male patients and their female partners (if fertile), must use contraception as well. In both cases, contraception means two forms of highly effective contraception during the study and for a period of 6 months following the last administration of the study drug.
14. Willing and able to provide informed consent
15. Ability and willingness to comply with study visits, treatment, testing, and to comply with the protocol.

Exclusion Criteria

1. Patients with any histology other than carcinoma, adenocarcinoma or ductal adenocarcinoma (such as squamous cell, acinar cell, medullary, colloid, neuroendocrine, etc)
2. Patients has only locally advanced disease, resectable or borderline resectable.
3. The patient has received chemotherapy as adjuvant therapy for locally advanced disease, resectable or borderline resectable.
4. Patient has received previous abdominal radiotherapy, (with the exception of analgesic radiotherapy that was not performed on target lesions).
5. Patients previously treated with an inhibitor of the PI3K/Akt/mTOR pathway by a systemic route.
6. History of chronic diarrhea or inflammatory disease of the colon or rectum, or occlusion or sub-occlusion not resolved under symptomatic treatment
7. Patient is pregnant or in lactation period. High sensitivity pregnancy test (urine or serum) to be performed within 7 days before study treatment starts.
8. Patient had myocardial infarction within ≤ 6 months prior to study entry, LVEF \<50%, symptomatic congestive heart failure (New York Heart Association \> class II), unstable angina pectoris, or unstable cardiac arrhythmia requiring medication.
9. 12-lead ECG with clinically relevant abnormality or showing a QTcF \>450 ms, PR \>210 ms, or QRS \>120 ms at screening.
10. Patients with any other medical conditions (such as psychiatric illness, cardiovascular disease, infectious diseases, abnormal physical examination or laboratory findings) that in the opinion of the investigator may interfere with the planned treatment, affect patient compliance or place the patient at high risk from treatment-related complications.
11. Patient has active Hepatitis B or C, human immunodeficiency virus (HIV) or Covid-19 infection with non-controlled disease according to the treating physician.
12. Patients unable to provide informed consent like those under administrative or legal supervision
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ability Pharmaceuticals SL

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc Cortal

Role: STUDY_DIRECTOR

Ability Pharmaceuticals SL

Locations

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Cedars Sinai

Los Angeles, California, United States

Site Status

University of Kansas Cancer Center

Westwood, Kansas, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

CGFL Dijon

Dijon, , France

Site Status

Institute Paoli-Calmettes

Marseille, , France

Site Status

Institut Gustave Roussy

Villejuif, , France

Site Status

Rabam MC

Haifa, , Israel

Site Status

Shaare Zedek MC

Jerusalem, , Israel

Site Status

Sheba MC

Ramat Gan, , Israel

Site Status

ICO Badalona

Badalona, Barcelona, Spain

Site Status

Centro Oncológico de Galicia

A Coruña, Galicia, Spain

Site Status

Hospital General Universitario Dr. Balmis

Alicante, , Spain

Site Status

Hospital Quiron Salud

Barcelona, , Spain

Site Status

Vall d'Hebron University Hospital

Barcelona, , Spain

Site Status

ICO Girona

Girona, , Spain

Site Status

Hospital Universitari Arnau de Vilanova

Lleida, , Spain

Site Status

Hospital Gregorio Marañón

Madrid, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital General Universitario Morales Meseguer

Murcia, , Spain

Site Status

Hospital Universitario Virgen del Rocío

Seville, , Spain

Site Status

Hospital Universitario de Toledo

Toledo, , Spain

Site Status

Hospital Universitario de Valencia

Valencia, , Spain

Site Status

Hospital Universitario Miguel Servet

Zaragoza, , Spain

Site Status

Countries

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United States France Israel Spain

References

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Erazo T, Lorente M, Lopez-Plana A, Munoz-Guardiola P, Fernandez-Nogueira P, Garcia-Martinez JA, Bragado P, Fuster G, Salazar M, Espadaler J, Hernandez-Losa J, Bayascas JR, Cortal M, Vidal L, Gascon P, Gomez-Ferreria M, Alfon J, Velasco G, Domenech C, Lizcano JM. The New Antitumor Drug ABTL0812 Inhibits the Akt/mTORC1 Axis by Upregulating Tribbles-3 Pseudokinase. Clin Cancer Res. 2016 May 15;22(10):2508-19. doi: 10.1158/1078-0432.CCR-15-1808. Epub 2015 Dec 15.

Reference Type BACKGROUND
PMID: 26671995 (View on PubMed)

Munoz-Guardiola P, Casas J, Megias-Roda E, Sole S, Perez-Montoyo H, Yeste-Velasco M, Erazo T, Dieguez-Martinez N, Espinosa-Gil S, Munoz-Pinedo C, Yoldi G, Abad JL, Segura MF, Moran T, Romeo M, Bosch-Barrera J, Oaknin A, Alfon J, Domenech C, Fabrias G, Velasco G, Lizcano JM. The anti-cancer drug ABTL0812 induces ER stress-mediated cytotoxic autophagy by increasing dihydroceramide levels in cancer cells. Autophagy. 2021 Jun;17(6):1349-1366. doi: 10.1080/15548627.2020.1761651. Epub 2020 May 25.

Reference Type BACKGROUND
PMID: 32397857 (View on PubMed)

Felip I, Moiola CP, Megino-Luque C, Lopez-Gil C, Cabrera S, Sole-Sanchez S, Munoz-Guardiola P, Megias-Roda E, Perez-Montoyo H, Alfon J, Yeste-Velasco M, Santacana M, Dolcet X, Reques A, Oaknin A, Rodriguez-Freixinos V, Lizcano JM, Domenech C, Gil-Moreno A, Matias-Guiu X, Colas E, Eritja N. Therapeutic potential of the new TRIB3-mediated cell autophagy anticancer drug ABTL0812 in endometrial cancer. Gynecol Oncol. 2019 May;153(2):425-435. doi: 10.1016/j.ygyno.2019.03.002. Epub 2019 Mar 7.

Reference Type BACKGROUND
PMID: 30853360 (View on PubMed)

Lopez-Plana A, Fernandez-Nogueira P, Munoz-Guardiola P, Sole-Sanchez S, Megias-Roda E, Perez-Montoyo H, Jauregui P, Yeste-Velasco M, Gomez-Ferreria M, Erazo T, Ametller E, Recalde-Percaz L, Moragas-Garcia N, Noguera-Castells A, Mancino M, Moran T, Nadal E, Alfon J, Domenech C, Gascon P, Lizcano JM, Fuster G, Bragado P. The novel proautophagy anticancer drug ABTL0812 potentiates chemotherapy in adenocarcinoma and squamous nonsmall cell lung cancer. Int J Cancer. 2020 Aug 15;147(4):1163-1179. doi: 10.1002/ijc.32865. Epub 2020 Feb 6.

Reference Type BACKGROUND
PMID: 31943158 (View on PubMed)

Vidal L, Victoria I, Gaba L, Martin MG, Brunet M, Colom H, Cortal M, Gomez-Ferreria M, Yeste-Velasco M, Perez A, Rodon J, Sohal DPS, Lizcano JM, Domenech C, Alfon J, Gascon P. A first-in-human phase I/Ib dose-escalation clinical trial of the autophagy inducer ABTL0812 in patients with advanced solid tumours. Eur J Cancer. 2021 Mar;146:87-94. doi: 10.1016/j.ejca.2020.12.019. Epub 2021 Feb 12.

Reference Type BACKGROUND
PMID: 33588149 (View on PubMed)

Paris-Coderch L, Soriano A, Jimenez C, Erazo T, Munoz-Guardiola P, Masanas M, Antonelli R, Boloix A, Alfon J, Perez-Montoyo H, Yeste-Velasco M, Domenech C, Roma J, Sanchez de Toledo J, Moreno L, Lizcano JM, Gallego S, Segura MF. The antitumour drug ABTL0812 impairs neuroblastoma growth through endoplasmic reticulum stress-mediated autophagy and apoptosis. Cell Death Dis. 2020 Sep 17;11(9):773. doi: 10.1038/s41419-020-02986-w.

Reference Type BACKGROUND
PMID: 32943619 (View on PubMed)

Mancini A, Colapietro A, Cristiano L, Rossetti A, Mattei V, Gravina GL, Perez-Montoyo H, Yeste-Velasco M, Alfon J, Domenech C, Festuccia C. Anticancer effects of ABTL0812, a clinical stage drug inducer of autophagy-mediated cancer cell death, in glioblastoma models. Front Oncol. 2022 Nov 2;12:943064. doi: 10.3389/fonc.2022.943064. eCollection 2022.

Reference Type BACKGROUND
PMID: 36408162 (View on PubMed)

Polonio-Alcala E, Sole-Sanchez S, Munoz-Guardiola P, Megias-Roda E, Perez-Montoyo H, Yeste-Velasco M, Alfon J, Lizcano JM, Domenech C, Ruiz-Martinez S, Puig T. ABTL0812 enhances antitumor effect of paclitaxel and reverts chemoresistance in triple-negative breast cancer models. Cancer Commun (Lond). 2022 Jun;42(6):567-571. doi: 10.1002/cac2.12282. Epub 2022 Mar 16. No abstract available.

Reference Type DERIVED
PMID: 35293148 (View on PubMed)

Other Identifiers

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2020-002791-13

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

FD-R-006817-01

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

ABT-C11-2020

Identifier Type: -

Identifier Source: org_study_id

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