Trial Outcomes & Findings for Mouse Cancer Cell-containing Macrobeads in the Treatment of Human Cancer (NCT NCT00283075)

NCT ID: NCT00283075

Last Updated: 2019-01-16

Results Overview

Dose limiting toxicity (DLT) was defined as: * any Grade 2 allergic reaction of generalized urticaria or any other Grade ≥ 3 allergic reaction; * any Grade ≥ 3 infection and * any Grade ≥ 3 local (intraperitoneal) reaction and any Grade ≥ 3 hematologic or non- hematologic reaction. This definition of DLT is in accord with the NCI CTCAE v3.0. Maximum tolerated dose (MTD) was to be identified if, within a cohort, \> 1 subject out of the first 3, or 2 subjects out of 5 experienced DLT. In such a case, the MTD will have been exceeded, and the administration of the study agent was to cease. MTD would not be considered to have been reached if no DLTs were observed.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

56 participants

Primary outcome timeframe

6 months

Results posted on

2019-01-16

Participant Flow

Fifty-six subjects provided informed consent to participate in this study; of these 31 subjects underwent the implantation of macrobeads that contain mouse renal adenocarcinoma cells (RENCA macrobeads). The first subject was implanted on 6 April 2005 and the last subject was implanted on 1 November 2011.

Participant milestones

Participant milestones
Measure
8 RENCA Macrobeads/kg Body Weight Implantation
RENCA macrobeads placement in abdominal cavity at 8 RENCA macrobeads/kg body weight.
16 RENCA Macrobeads/kg Body Weight Implantation
RENCA macrobeads placement in abdominal cavity at 16 RENCA macrobeads/kg body weight.
Overall Study
STARTED
25
6
Overall Study
COMPLETED
25
6
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Mouse Cancer Cell-containing Macrobeads in the Treatment of Human Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
All Participants
n=31 Participants
All participants who had at least one implantation of RENCA macrobeads, either at 8 RENCA macrobeads/kg body weight or 16 RENCA macrobeads/kg body weight.
Age, Continuous
59.1 years
STANDARD_DEVIATION 8.53 • n=93 Participants
Sex: Female, Male
Female
16 Participants
n=93 Participants
Sex: Female, Male
Male
15 Participants
n=93 Participants
Race/Ethnicity, Customized
White
12 participants
n=93 Participants
Race/Ethnicity, Customized
Hispanic
2 participants
n=93 Participants
Race/Ethnicity, Customized
Asian
1 participants
n=93 Participants
Race/Ethnicity, Customized
Not Available
16 participants
n=93 Participants

PRIMARY outcome

Timeframe: 6 months

Dose limiting toxicity (DLT) was defined as: * any Grade 2 allergic reaction of generalized urticaria or any other Grade ≥ 3 allergic reaction; * any Grade ≥ 3 infection and * any Grade ≥ 3 local (intraperitoneal) reaction and any Grade ≥ 3 hematologic or non- hematologic reaction. This definition of DLT is in accord with the NCI CTCAE v3.0. Maximum tolerated dose (MTD) was to be identified if, within a cohort, \> 1 subject out of the first 3, or 2 subjects out of 5 experienced DLT. In such a case, the MTD will have been exceeded, and the administration of the study agent was to cease. MTD would not be considered to have been reached if no DLTs were observed.

Outcome measures

Outcome measures
Measure
All Participants
n=31 Participants
All participants who had at least one implantation of RENCA macrobeads, either at 8 RENCA macrobeads/kg body weight or 16 RENCA macrobeads/kg body weight.
Day 14
Tumor marker response at Day 14 after the first implantation.
Day 21
Tumor marker response at Day 21 after the first implantation.
Day 28
Tumor marker response at Day 28 after the first implantation.
Maximum Tolerated Dose (MTD) of RENCA Macrobeads
NA RENCA Macrobeads/kg
MTD of of RENCA macrobeads has not been reached because no DLTs were observed.

PRIMARY outcome

Timeframe: 6 months

Dose limiting toxicity (DLT) was defined as: * any Grade 2 allergic reaction of generalized urticaria or any other Grade ≥ 3 allergic reaction; * any Grade ≥ 3 infection and * any Grade ≥ 3 local (intraperitoneal) reaction and any Grade ≥ 3 hematologic or non- hematologic reaction. This definition of DLT is in accord with the NCI CTCAE v3.0.

Outcome measures

Outcome measures
Measure
All Participants
n=25 Participants
All participants who had at least one implantation of RENCA macrobeads, either at 8 RENCA macrobeads/kg body weight or 16 RENCA macrobeads/kg body weight.
Day 14
n=6 Participants
Tumor marker response at Day 14 after the first implantation.
Day 21
Tumor marker response at Day 21 after the first implantation.
Day 28
Tumor marker response at Day 28 after the first implantation.
Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs)
0 Participants who observed DLT
0 Participants who observed DLT

SECONDARY outcome

Timeframe: From date of RENCA macrobeads implantation until date of death from any cause

Overall Survival (OS) was measured as date of first implantation to date of death of any cause, and was analyzed using the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure
All Participants
n=31 Participants
All participants who had at least one implantation of RENCA macrobeads, either at 8 RENCA macrobeads/kg body weight or 16 RENCA macrobeads/kg body weight.
Day 14
Tumor marker response at Day 14 after the first implantation.
Day 21
Tumor marker response at Day 21 after the first implantation.
Day 28
Tumor marker response at Day 28 after the first implantation.
Overall Survival
All Cancer Type
5.4 months
Interval 2.2 to 7.0
Overall Survival
Colorectal Cancer
7.0 months
Interval 1.1 to 9.7
Overall Survival
Pancreatic Cancer
1.1 months
Interval 1.0 to 2.2
Overall Survival
Other Cancer
5.7 months
Interval 2.0 to 7.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Prior to Implantation and Day 7, Day 14, Day 21 and Day 28 after each implantation

Population: All participants who had at least one implantation of RENCA macrobeads, either at 8 macrobeads/kg body weight or 16 macrobeads/kg body weight.

Tumor marker response after the first implantation with RENCA macrobeads. Responders showed at least a 20% decrease from baseline in Cancer Antigen 19-9 (CA19-9) or Carcinoembryonic Antigen (CEA); Non-responders do not show at least a 20% decrease from baseline in CA19-9 or CEA.

Outcome measures

Outcome measures
Measure
All Participants
n=20 Participants
All participants who had at least one implantation of RENCA macrobeads, either at 8 RENCA macrobeads/kg body weight or 16 RENCA macrobeads/kg body weight.
Day 14
n=21 Participants
Tumor marker response at Day 14 after the first implantation.
Day 21
n=13 Participants
Tumor marker response at Day 21 after the first implantation.
Day 28
n=17 Participants
Tumor marker response at Day 28 after the first implantation.
Tumor Marker Response
Responder
5 participants
13 participants
9 participants
10 participants
Tumor Marker Response
Non-Responder
15 participants
8 participants
4 participants
7 participants

Adverse Events

All Participants

Serious events: 20 serious events
Other events: 27 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
All Participants
n=31 participants at risk
All participants who had at least one implantation of RENCA macrobeads, either at 8 macrobeads/kg body weight or 16 macrobeads/kg body weight.
Surgical and medical procedures
Implant site fluid collection
3.2%
1/31 • Number of events 1 • 6 months
Surgical and medical procedures
Tear from mesh s/p hernia repair
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Intermittent fever
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Disease progression
6.5%
2/31 • Number of events 2 • 6 months
Gastrointestinal disorders
Diarrhea
3.2%
1/31 • Number of events 1 • 6 months
Gastrointestinal disorders
Vomiting
6.5%
2/31 • Number of events 2 • 6 months
General disorders
Hypophagia
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Pleurodesis
3.2%
1/31 • Number of events 1 • 6 months
Cardiac disorders
Pericardial effusion
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Hypotension, poor nutrition
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Aspiration
3.2%
1/31 • Number of events 1 • 6 months
Gastrointestinal disorders
Jaundice
3.2%
1/31 • Number of events 1 • 6 months
Gastrointestinal disorders
Abdominal distension
6.5%
2/31 • Number of events 2 • 6 months
Gastrointestinal disorders
Abdominal pain
6.5%
2/31 • Number of events 2 • 6 months
General disorders
Transfusion
3.2%
1/31 • Number of events 2 • 6 months
General disorders
Dehydration
3.2%
1/31 • Number of events 1 • 6 months
Gastrointestinal disorders
Gastrointestinal obstruction
3.2%
1/31 • Number of events 4 • 6 months
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Asthenia
6.5%
2/31 • Number of events 2 • 6 months
General disorders
Blood creatinine increased
3.2%
1/31 • Number of events 1 • 6 months
Gastrointestinal disorders
Small intestinal obstruction
6.5%
2/31 • Number of events 2 • 6 months
Gastrointestinal disorders
Melena
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Hypotension
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Anemia
3.2%
1/31 • Number of events 1 • 6 months
Surgical and medical procedures
Incision site pain
3.2%
1/31 • Number of events 1 • 6 months
Surgical and medical procedures
Wound complication
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Hyponatremia
6.5%
2/31 • Number of events 2 • 6 months
General disorders
Pain
3.2%
1/31 • Number of events 1 • 6 months
General disorders
Hydronephrosis
3.2%
1/31 • Number of events 2 • 6 months

Other adverse events

Other adverse events
Measure
All Participants
n=31 participants at risk
All participants who had at least one implantation of RENCA macrobeads, either at 8 macrobeads/kg body weight or 16 macrobeads/kg body weight.
Gastrointestinal disorders
Abdominal distension
16.1%
5/31 • 6 months
Gastrointestinal disorders
Abdominal pain
25.8%
8/31 • 6 months
Gastrointestinal disorders
Abdominal pain upper
6.5%
2/31 • 6 months
Gastrointestinal disorders
Ascites
9.7%
3/31 • 6 months
Gastrointestinal disorders
Constipation
19.4%
6/31 • 6 months
Gastrointestinal disorders
Nausea
19.4%
6/31 • 6 months
Gastrointestinal disorders
Vomiting
16.1%
5/31 • 6 months
General disorders
Asthenia
6.5%
2/31 • 6 months
General disorders
Fatigue
48.4%
15/31 • 6 months
General disorders
Inflammation
12.9%
4/31 • 6 months
General disorders
Pain
16.1%
5/31 • 6 months
General disorders
Pyrexia
35.5%
11/31 • 6 months
Injury, poisoning and procedural complications
Incision site pain
9.7%
3/31 • 6 months
Investigations
Hemoglobin decreased
6.5%
2/31 • 6 months
Metabolism and nutrition disorders
Decreased appetite
9.7%
3/31 • 6 months
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
6.5%
2/31 • 6 months
Musculoskeletal and connective tissue disorders
Myalgia
6.5%
2/31 • 6 months
Nervous system disorders
Dizziness
6.5%
2/31 • 6 months
Respiratory, thoracic and mediastinal disorders
Hemoptysis
6.5%
2/31 • 6 months
Skin and subcutaneous tissue disorders
Hyperhidrosis
12.9%
4/31 • 6 months
Skin and subcutaneous tissue disorders
Night sweats
19.4%
6/31 • 6 months
Skin and subcutaneous tissue disorders
Rash
12.9%
4/31 • 6 months
Skin and subcutaneous tissue disorders
Umbilical erythema
9.7%
3/31 • 6 months

Additional Information

Dr. Barry H. Smith, CEO

The Rogosin Institute

Phone: 212-746-1551

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place