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Pancreatic Adenocarcinoma Non-resectablePhase 2MacrobeadNCT01053013

Open-Label Phase 2 Efficacy Trial of Cancer Macrobeads in Patients With Treatment-Resistant Pancreatic/Colorectal Cancer

A Phase 2 study testing RENCA macrobeads in people with Pancreatic Adenocarcinoma Non-resectable and Pancreatic Adenocarcinoma Metastatic, run by The Rogosin Institute at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a clinical research study of an investigational (FDA BB-IND 10091) treatment for patients with pancreatic cancer (all stages) and advanced colorectal cancer that no longer responds to standard therapies. The treatment is being evaluated for its effect on tumor growth. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
RENCA macrobeads
Could I receive a placebo?
No, everyone receives an active treatment
How many people
58 participants
Where
1 siteUnited States
Study dates
15 Apr 2010to 16 Apr 2016
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically confirmed adenocarcinoma of the pancreas, colon or rectum.
  • Radiographic evidence of metastatic cancer of the colon or rectum.
  • Pancreatic cancer that is unresectable or already metastatic, or colorectal cancer that has failed available approved treatment modalities.
  • For pancreatic cancer patients, prior chemotherapy is not required; for colon and rectal cancer patients must have failed available chemotherapy/targeted regimens. There were no limits to the number of prior chemotherapeutic regimens.
  • The patient had evidence of progressive disease defined as at least one of the following:
  • Progressive measurable disease using conventional solid tumor criteria.
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  • Increasing tumor markers and/or activity on positron emission tomography / standard uptake value (PET/SUV) measurement.
  • All clinically significant toxic effects (excluding alopecia) of prior surgery, radiotherapy, or hormonal therapy were resolved to ≤ Grade 1 based on the National Cancer Institute Common Terminology Criteria for Adverse Events Version 3.0 (NCI CTCAE v.3.0) with the exception of neuropathy, which was resolved to ≤ Grade 2.
  • Performance status (ECOG PS) 0-2.
  • Adequate hematologic function, minimum requirements:
  • absolute neutrophil count (ANC) ≥ 1500/mL
  • hemoglobin ≥ 9 g/dL
  • platelets ≥ 100,000/mL
  • Adequate hepatic function, defined as follows:
  • bilirubin ≤ 1.5 times the upper limit of normal (ULN)
  • aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 3 times the ULN, or ≤ 5 times the ULN if liver metastases are present
  • Adequate renal function, defined as serum creatinine ≤ 2.0 mg/dL.
  • Adequate coagulation function, defined as follows:
  • an international normalized ratio (INR) ≤ 1.5
  • a partial thromboplastin time (PTT) ≤ 5 seconds above the ULN, unless on anti-coagulant therapy
  • patients receiving full-dose anticoagulation therapy were eligible provided they met all other criteria, were on a stable dose or anticoagulant therapy or low molecular weight heparin (and if on warfarin had a therapeutic INR between 2-3)
  • Life expectancy of at least 6 weeks.
  • For females of childbearing potential, a negative pregnancy test.
  • Agrees to contraceptive use (barrier method) while on study, if sexually active.
  • Provided signed informed consent.

Who cannot join

  • Any condition presenting an unacceptably high anesthetic or surgical risk, based on current anesthesia/general surgery standards.
  • Positive test for HIV, hepatitis B, C, or E.
  • Cognitive impairment such as to preclude informed consent.
  • Hypersensitivity reaction that, in the opinion of the investigators posed an increased risk of an allergy to the macrobeads, particularly any known allergy to murine antigens or body tissues.
  • Surgical treatment or chemotherapy within three weeks of scheduled macrobead implantation or within four weeks of bevacizumab (or similar drugs), or radiation therapy within four weeks of scheduled macrobead implantation.
  • Investigational medication(s)/therapies for respective tumor within one month of baseline evaluation.
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  • Inadequate hematologic function, defined as follows:
  • ANC < 1500/mL
  • hemoglobin < 9 g/dL
  • platelets < 100,000/mL
  • Inadequate hepatic function, defined as follows:
  • bilirubin > 1.5 times the ULN
  • AST and ALT > 3 times the ULN or > 5 times the ULN, if liver metastases present
  • Inadequate renal function, defined as serum creatinine > 2.0 mg/dL.
  • Inadequate coagulation function, defined as follows:
  • INR > 1.5
  • PTT > 5 seconds (unless on anti-coagulant therapy)
  • Hepatic blood flow abnormalities, portal vein hypertension and thrombosis, and/or large-volume ascites.
  • Concurrent cancer of any other type except skin cancer (excluding melanoma).
  • Ongoing or active infection, congestive heart failure, unstable angina, serious cardiac arrhythmias, psychiatric illness, difficult social situations not permitting reliable participation, active bleeding.
  • As a result of the medical history, examination, or blood testing, the investigator considers the patient unfit for the study.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Weill Cornell Medical Center / The Rogosin Institute

    New York, New York, 10021

Other trials in United States

About this study

Official title: An Open-Label Phase 2 Efficacy Trial of the Implantation of Mouse Renal Adenocarcinoma Cell-Containing Agarose-Agarose Macrobeads in the Treatment of Patients With Treatment-Resistant, Metastatic Pancreatic or Colorectal Cancer

This is a clinical research study of an investigational (FDA BB-IND 10091) treatment for patients with pancreatic cancer (all stages) and advanced colorectal cancer that no longer responds to standard therapies.

The treatment is being evaluated for its effect on tumor growth. It consists of the placement (implantation) of small beads that contain mouse renal adenocarcinoma cells (RENCA macrobeads). The cells in the macrobeads produce substances that have been shown to slow or stop the growth of tumors in experimental animals and veterinary patients. It has been tested in 31 human subjects with different types of cancers in a Phase I safety trial. Phase II studies in patients with colorectal, pancreatic or prostate cancers are in progress.

Read the detailed description

This is an open-label Phase 2 clinical trial. The study will have a duration of 12 months and involve a potential total of four macrobead implants for each enrolled patient, with the implants being no less than three months apart.

The RENCA macrobeads are implanted intraperitoneally, using laparoscopic surgical procedures. The macrobeads remain permanently within the peritoneal cavity, even if patients withdraw from the study and/or begin new therapy (e.g. chemotherapy). After the formal phase of the trial is completed, subjects will be followed for life.

The intent of this Phase 2 trial is to assess efficacy, safety, and tolerability of RENCA macrobeads. Screening occurs within 28 days prior to first implantation. Enrolled patients are treated with a dose of 8 RENCA macrobeads per kilogram of body weight and observed for a minimum of 3 months prior to the next implantation. A maximum of 4 implantation procedures is possible. Day 0 is the day of implantation.

Prior to the start of the study, the Investigators ensured that the protocol and any attendant documentation were approved, in writing, by the Institutional Review Board. The study was conducted in compliance with the International Conference on Harmonization (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use Good Clinical Practice Guidelines (E6 [R1]) for conducting, recording, and reporting studies, as well as for archiving essential documents.

The Investigators, the Medical Monitor of the sponsor, and the Data Safety Monitoring Board were responsible for monitoring safety parameters collected in the study. They, collectively, provided oversight and monitoring of the safety of the participants in the study.

Being tested
RENCA macrobeads
Keywords
macrobead, biological, mouse cells

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RENCA macrobeadsBiological

    Also called mouse renal adenocarcinoma (RENCA) macrobeads, macrobead, cancer macrobead

Study groups in NCT01053013
Study groupRoleWhat they receive
Macrobead ImplantationExperimentalpatients will undergo up to 4 implantations of RENCA macrobeads (no less than 3 months apart), at an amount of 8 RENCA macrobeads per kilogram of body weight
Receives: Biological: RENCA macrobeads

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Survival - Based on Most Recent Scan

    The primary efficacy outcome is post-implantation all-cause mortality, where time to death is defined as the time from the most recent scan prior to first implantation (time of origin, To) to death from any cause.

    Measured: From date of the most recent scan (disease progression) prior to the first macrobead implantation; assessed up to 32 months.

Secondary outcomes (2)
  1. Overall Survival - Based on 1st Implant

    The secondary efficacy objective was to evaluate overall survival calculated using the time from first RENCA macrobead implantation to death from any cause.

    Measured: From date of the first RENCA macrobead implant; assessed up to 32 months.

  2. Overall Survival - Based on Stage IV Disease Diagnosis

    The secondary efficacy objective was to evaluate overall survival calculated using the time from Stage IV disease diagnosis to death from any cause.

    Measured: From date of Stage IV disease diagnosis up to 126 months; up to 32 months from first RENCA macrobead implant.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Jan 2010 (estimated)
  2. Study start15 Apr 2010
  3. Primary completion16 Apr 2016
  4. Study completion16 Apr 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
58 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01053013
Sponsor's protocol ID
0911010739
Acronym
Macrobead
Status last verified
Apr 2021
First submitted
20 Jan 2010
Last updated
12 May 2021
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Smith BH, Gazda LS, Conn BL, Jain K, Asina S, Levine DM, Parker TS, Laramore MA, Martis PC, Vinerean HV, David EM, Qiu S, Cordon-Cardo C, Hall RD, Gordon BR, Diehl CH, Stenzel KH, Rubin AL. Three-dimensional culture of mouse renal carcinoma cells in agarose macrobeads selects for a subpopulation of cells with cancer stem cell or cancer progenitor properties. Cancer Res. 2011 Feb 1;71(3):716-24. doi: 10.1158/0008-5472.CAN-10-2254. Epub 2011 Jan 24.

    PubMed 21266363
  • Smith BH, Gazda LS, Conn BL, Jain K, Asina S, Levine DM, Parker TS, Laramore MA, Martis PC, Vinerean HV, David EM, Qiu S, North AJ, Couto CG, Post GS, Waters DJ, Cordon-Cardo C, Hall RD, Gordon BR, Diehl CH, Stenzel KH, Rubin AL. Hydrophilic agarose macrobead cultures select for outgrowth of carcinoma cell populations that can restrict tumor growth. Cancer Res. 2011 Feb 1;71(3):725-35. doi: 10.1158/0008-5472.CAN-10-2258. Epub 2011 Jan 24.

    PubMed 21266362
  • Smith BH, Gazda LS, Fahey TJ, Nazarian A, Laramore MA, Martis P, Andrada ZP, Thomas J, Parikh T, Sureshbabu S, Berman N, Ocean AJ, Hall RD, Wolf DJ. Clinical laboratory and imaging evidence for effectiveness of agarose-agarose macrobeads containing stem-like cells derived from a mouse renal adenocarcinoma cell population (RMBs) in treatment-resistant, advanced metastatic colorectal cancer: Evaluation of a biological-systems approach to cancer therapy (U.S. FDA IND-BB 10091; NCT 02046174, NCT 01053013). Chin J Cancer Res. 2018 Feb;30(1):72-83. doi: 10.21147/j.issn.1000-9604.2018.01.08.

    PubMed 29545721
  • Gazda LS, Martis PC, Laramore MA, Bautista MA, Dudley A, Vinerean HV, Smith BH. Treatment of agarose-agarose RENCA macrobeads with docetaxel selects for OCT4(+) cells with tumor-initiating capability. Cancer Biol Ther. 2013 Dec;14(12):1147-57. doi: 10.4161/cbt.26455. Epub 2013 Sep 12.

    PubMed 24025409
  • Martis PC, Dudley AT, Bemrose MA, Gazda HL, Smith BH, Gazda LS. MEF2 plays a significant role in the tumor inhibitory mechanism of encapsulated RENCA cells via EGF receptor signaling in target tumor cells. BMC Cancer. 2018 Dec 4;18(1):1217. doi: 10.1186/s12885-018-5128-5.

    PubMed 30514247
  • Smith BH, Parikh T, Andrada ZP, Fahey TJ, Berman N, Wiles M, Nazarian A, Thomas J, Arreglado A, Akahoho E, Wolf DJ, Levine DM, Parker TS, Gazda LS, Ocean AJ. First-in-Human Phase 1 Trial of Agarose Beads Containing Murine RENCA Cells in Advanced Solid Tumors. Cancer Growth Metastasis. 2016 Aug 2;9:9-20. doi: 10.4137/CGM.S39442. eCollection 2016.

    PubMed 27499645

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing RENCA macrobeads in people with Pancreatic Adenocarcinoma Non-resectable and Pancreatic Adenocarcinoma Metastatic, run by The Rogosin Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 12 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Survival - Based on Most Recent Scan (time frame: From date of the most recent scan (disease progression) prior to the first macrobead implantation; assessed up to 32 months.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01053013, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 May 2021.

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