Trial Outcomes & Findings for Multicenter Study Of CPX-1 (Irinotecan HCl: Floxuridine) Liposome Injection In Patients With Advanced Colorectal Cancer (NCT NCT00361842)

NCT ID: NCT00361842

Last Updated: 2021-07-02

Results Overview

Disease response was assessed using RECIST 1.0. Measurable disease and target lesions were determined prior to study entry. Changes in the largest diameter (unidimensional measurement) of the sum of the target tumor lesions were used in the RECIST criteria. Best response on study was classified as follows. Complete Response (CR), disappearance of all clinical and radiological evidence of tumor. Partial Response (PR) at least a 30% decrease in the sum of the longest diameter of target lesions taking as reference the baseline sum of the longest diameters. Stable Disease (SD), steady state of disease. Neither sufficient shrinkage to qualify for PR or sufficient increase to qualify for PD. Progressive Disease (PD) at least a 20% increase in the sum of the longest diameters of measured lesions taking as references the smallest sum of longest diameters recorded since the treatment started. Appearance of new lesions also constituted progressive disease.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

65 participants

Primary outcome timeframe

From date of first dose until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months

Results posted on

2021-07-02

Participant Flow

Participant milestones

Participant milestones
Measure
Irinotecan - Naïve
Irinotecan - Exposed
Overall Study
STARTED
28
35
Overall Study
COMPLETED
1
1
Overall Study
NOT COMPLETED
27
34

Reasons for withdrawal

Reasons for withdrawal
Measure
Irinotecan - Naïve
Irinotecan - Exposed
Overall Study
Withdrawal by Subject
2
3
Overall Study
Death
2
1
Overall Study
Disease progression
19
20
Overall Study
Adverse Event
2
3
Overall Study
Occurrence of unacceptable toxicity
1
5
Overall Study
Other
0
2

Baseline Characteristics

Multicenter Study Of CPX-1 (Irinotecan HCl: Floxuridine) Liposome Injection In Patients With Advanced Colorectal Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Irinotecan - Naïve
n=26 Participants
Irinotecan - Exposed
n=33 Participants
Total
n=59 Participants
Total of all reporting groups
Age, Continuous
59.9 years
STANDARD_DEVIATION 9.62 • n=5 Participants
60.2 years
STANDARD_DEVIATION 10.92 • n=7 Participants
60.1 years
STANDARD_DEVIATION 10.28 • n=5 Participants
Sex: Female, Male
Female
13 Participants
n=5 Participants
15 Participants
n=7 Participants
28 Participants
n=5 Participants
Sex: Female, Male
Male
13 Participants
n=5 Participants
18 Participants
n=7 Participants
31 Participants
n=5 Participants
Region of Enrollment
Canada
8 participants
n=5 Participants
17 participants
n=7 Participants
25 participants
n=5 Participants
Region of Enrollment
United States
18 participants
n=5 Participants
16 participants
n=7 Participants
34 participants
n=5 Participants

PRIMARY outcome

Timeframe: From date of first dose until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months

Population: The response rate efficacy evaluable (RECIST) analysis set (n=21 in group 1 and n= 24 in group 2) included all enrolled participants who received at least 1 cycle of CPX-1 therapy (2 doses, Days 1 and 15) and had at least 1 measurable lesion assessed for response (either after every second cycle of treatment or earlier to document disease progression) after beginning treatment. Tumor response, based on the RECIST criteria, was assessed at baseline and every 8 weeks.

Disease response was assessed using RECIST 1.0. Measurable disease and target lesions were determined prior to study entry. Changes in the largest diameter (unidimensional measurement) of the sum of the target tumor lesions were used in the RECIST criteria. Best response on study was classified as follows. Complete Response (CR), disappearance of all clinical and radiological evidence of tumor. Partial Response (PR) at least a 30% decrease in the sum of the longest diameter of target lesions taking as reference the baseline sum of the longest diameters. Stable Disease (SD), steady state of disease. Neither sufficient shrinkage to qualify for PR or sufficient increase to qualify for PD. Progressive Disease (PD) at least a 20% increase in the sum of the longest diameters of measured lesions taking as references the smallest sum of longest diameters recorded since the treatment started. Appearance of new lesions also constituted progressive disease.

Outcome measures

Outcome measures
Measure
Irinotecan - Naïve
n=21 Participants
* No prior exposure to irinotecan * No more than 1 regimen for metastatic disease * No more than 2 regimens overall; * 1 for neoadjuvant/adjuvant and 1 for metastatic/advanced disease
Irinotecan - Exposed
n=24 Participants
* Prior exposure to irinotecan was required * Disease progression on or within 6 months after completion of prior irinotecan-containing regimen * CPX-1 treatment must start within 12 months after documentation of disease progression on irinotecan (other therapies are permitted after irinotecan and before study entry)
Objective Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.0
Complete Response (CR)
0 Participants
0 Participants
Objective Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.0
Partial Response (PR)
2 Participants
0 Participants
Objective Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.0
Stable Disease (SD)
11 Participants
12 Participants
Objective Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.0
Progressive Disease (PD)
8 Participants
11 Participants

PRIMARY outcome

Timeframe: Every 8 weeks

Population: The efficacy evaluable analysis set or "Full Analysis Set" (25 patients in Group 1 and 32 participants in Group 2) included all enrolled participants who received at least 1 dose of CPX-1 therapy, and had at least one post-baseline PFS assessment.

Progression-free survival (PFS) was defined as the time from the first dose to the documentation of progressive disease (PD), death, or lost to follow-up at last assessment visit.

Outcome measures

Outcome measures
Measure
Irinotecan - Naïve
n=25 Participants
* No prior exposure to irinotecan * No more than 1 regimen for metastatic disease * No more than 2 regimens overall; * 1 for neoadjuvant/adjuvant and 1 for metastatic/advanced disease
Irinotecan - Exposed
n=32 Participants
* Prior exposure to irinotecan was required * Disease progression on or within 6 months after completion of prior irinotecan-containing regimen * CPX-1 treatment must start within 12 months after documentation of disease progression on irinotecan (other therapies are permitted after irinotecan and before study entry)
Progression-free Survival (PFS) Per RECIST Version 1.0
4.69 months
Standard Error 0.84
3.48 months
Standard Error 0.44

SECONDARY outcome

Timeframe: Every 8 weeks

Population: No participants had CR. Two participants in the naïve group had objective responses. One participant showed a PR on study day 57 that lasted for 5.8 months. One participant had a PR starting on study day 60 and persisting for 9.1 months at which time the data was censored. Due to the censored observation, the median DoR was not estimable (NE).

The duration of overall response was measured from the time that measurement criteria were met for CR or PR (whichever status was recorded first) until the first date that recurrent or progressive disease was objectively documented (taking as reference for progressive disease the smallest measurements recorded since the treatment started). The duration of overall complete response was measured from the time measurement criteria were first met for complete response until the first date that recurrent disease was objectively documented.

Outcome measures

Outcome measures
Measure
Irinotecan - Naïve
n=2 Participants
* No prior exposure to irinotecan * No more than 1 regimen for metastatic disease * No more than 2 regimens overall; * 1 for neoadjuvant/adjuvant and 1 for metastatic/advanced disease
Irinotecan - Exposed
* Prior exposure to irinotecan was required * Disease progression on or within 6 months after completion of prior irinotecan-containing regimen * CPX-1 treatment must start within 12 months after documentation of disease progression on irinotecan (other therapies are permitted after irinotecan and before study entry)
Duration of Response (DoR) Per RECIST Version 1.0
NA months
Interval 5.8 to 9.1
One participant had a PR starting on study day 60 and persisting for 9.1 months at which time the data was censored. Due to the censored observation, the median DoR was not estimable (NE).

Adverse Events

Irinotecan - Naïve

Serious events: 13 serious events
Other events: 26 other events
Deaths: 2 deaths

Irinotecan - Exposed

Serious events: 11 serious events
Other events: 33 other events
Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure
Irinotecan - Naïve
n=26 participants at risk
Irinotecan - Exposed
n=33 participants at risk
Gastrointestinal disorders
Diarrhea
15.4%
4/26
3.0%
1/33
Blood and lymphatic system disorders
Neutropenia
11.5%
3/26
3.0%
1/33
Gastrointestinal disorders
Pyrexia
7.7%
2/26
3.0%
1/33
Infections and infestations
Pneumonia
7.7%
2/26
3.0%
1/33
Blood and lymphatic system disorders
Febrile Neutropenia
0.00%
0/26
6.1%
2/33
Gastrointestinal disorders
Vomiting
7.7%
2/26
0.00%
0/33
Metabolism and nutrition disorders
Dehydration
7.7%
2/26
0.00%
0/33
Cardiac disorders
Cardio-respiratory Arrest
3.8%
1/26
0.00%
0/33
Gastrointestinal disorders
Colitis
0.00%
0/26
3.0%
1/33
Gastrointestinal disorders
Enteritis
3.8%
1/26
0.00%
0/33
Gastrointestinal disorders
GI Hemorrhage
3.8%
1/26
0.00%
0/33
Gastrointestinal disorders
Nausea
3.8%
1/26
0.00%
0/33
Gastrointestinal disorders
Small Intestine Obstruction
0.00%
0/26
3.0%
1/33
General disorders
General Physical Deterioration
3.8%
1/26
0.00%
0/33
Gastrointestinal disorders
Infusion Related Reaction
0.00%
0/26
3.0%
1/33
Infections and infestations
Gastroenteritis
0.00%
0/26
3.0%
1/33
Infections and infestations
Sepsis
0.00%
0/26
3.0%
1/33
Infections and infestations
Staphylococcal Sepsis
3.8%
1/26
0.00%
0/33
Injury, poisoning and procedural complications
Accidental Overdose
0.00%
0/26
3.0%
1/33
Injury, poisoning and procedural complications
GI Stoma Complication
3.8%
1/26
0.00%
0/33
Investigations
Neutrophil Count Decreased
0.00%
0/26
3.0%
1/33
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastasis to Lung
0.00%
0/26
3.0%
1/33
Psychiatric disorders
Anxiety
3.8%
1/26
0.00%
0/33
Respiratory, thoracic and mediastinal disorders
Orthopnea
0.00%
0/26
3.0%
1/33
Respiratory, thoracic and mediastinal disorders
Pneumonia Aspiration
0.00%
0/26
3.0%
1/33
Vascular disorders
Peripheral Arterial Occlusive Disease
3.8%
1/26
0.00%
0/33
General disorders
Disease Progression
0.00%
0/26
6.1%
2/33

Other adverse events

Other adverse events
Measure
Irinotecan - Naïve
n=26 participants at risk
Irinotecan - Exposed
n=33 participants at risk
Blood and lymphatic system disorders
Anemia
19.2%
5/26
15.2%
5/33
Blood and lymphatic system disorders
Neutropenia
19.2%
5/26
42.4%
14/33
Gastrointestinal disorders
Abdominal Pain
38.5%
10/26
18.2%
6/33
Gastrointestinal disorders
Abdominal Pain Upper
7.7%
2/26
9.1%
3/33
Gastrointestinal disorders
Constipation
26.9%
7/26
24.2%
8/33
Gastrointestinal disorders
Diarrhea
84.6%
22/26
78.8%
26/33
Gastrointestinal disorders
Dry Mouth
3.8%
1/26
6.1%
2/33
Gastrointestinal disorders
Dyspepsia
15.4%
4/26
15.2%
5/33
Gastrointestinal disorders
Flatulence
23.1%
6/26
12.1%
4/33
Gastrointestinal disorders
Nausea
80.8%
21/26
72.7%
24/33
Gastrointestinal disorders
Vomiting
69.2%
18/26
51.5%
17/33
General disorders
Asthenia
23.1%
6/26
9.1%
3/33
General disorders
Chills
15.4%
4/26
9.1%
3/33
General disorders
Fatigue
57.7%
15/26
66.7%
22/33
General disorders
Mucosal Inflammation
7.7%
2/26
9.1%
3/33
General disorders
Edema, Peripheral
3.8%
1/26
6.1%
2/33
General disorders
Pyrexia
15.4%
4/26
24.2%
8/33
Infections and infestations
Nasopharyngitis
7.7%
2/26
3.0%
1/33
Infections and infestations
Urinary Tract Infection
11.5%
3/26
9.1%
3/33
Investigations
Neutrophil Count Decreased
0.00%
0/26
9.1%
3/33
Investigations
Weight Decreased
23.1%
6/26
33.3%
11/33
Metabolism and nutrition disorders
Anorexia
38.5%
10/26
39.4%
13/33
Metabolism and nutrition disorders
Decreased Appetite
11.5%
3/26
12.1%
4/33
Metabolism and nutrition disorders
Dehydration
30.8%
8/26
18.2%
6/33
Metabolism and nutrition disorders
Hypokalemia
19.2%
5/26
12.1%
4/33
Musculoskeletal and connective tissue disorders
Arthralgia
15.4%
4/26
0.00%
0/33
Musculoskeletal and connective tissue disorders
Back Pain
15.4%
4/26
9.1%
3/33
Musculoskeletal and connective tissue disorders
Muscular Weakness
3.8%
1/26
6.1%
2/33
Musculoskeletal and connective tissue disorders
Musculoskeletal Pain
11.5%
3/26
6.1%
2/33
Musculoskeletal and connective tissue disorders
Myalgia
3.8%
1/26
9.1%
3/33
Nervous system disorders
Dizziness
3.8%
1/26
6.1%
2/33
Nervous system disorders
Headache
19.2%
5/26
18.2%
6/33
Psychiatric disorders
Anxiety
3.8%
1/26
6.1%
2/33
Psychiatric disorders
Depression
11.5%
3/26
15.2%
5/33
Psychiatric disorders
Insomnia
7.7%
2/26
3.0%
1/33
Respiratory, thoracic and mediastinal disorders
Cough
19.2%
5/26
3.0%
1/33
Respiratory, thoracic and mediastinal disorders
Dyspnea, Exertional
3.8%
1/26
6.1%
2/33
Skin and subcutaneous tissue disorders
Alopecia
30.8%
8/26
27.3%
9/33
Skin and subcutaneous tissue disorders
Hyperhidrosis
0.00%
0/26
9.1%
3/33
Skin and subcutaneous tissue disorders
Night Sweats
7.7%
2/26
3.0%
1/33
Skin and subcutaneous tissue disorders
Rash
3.8%
1/26
15.2%
5/33
Vascular disorders
Hot Flush
7.7%
2/26
3.0%
1/33
Vascular disorders
Hypotension
0.00%
0/26
12.1%
4/33

Additional Information

Director, Clinical Trial Disclosure & Transparency

Jazz Pharmaceuticals

Phone: 2158709177

Results disclosure agreements

  • Principal investigator is a sponsor employee The sponsor can review trial results communications prior to public release and can embargo such communications for a period of at least 60 days from the time submitted to sponsor for review. If requested by sponsor, the PI will withhold publication for up to an additional 30 days. Furthermore, the first publication of study results must be a joint publication of all study sites unless a joint manuscript has not been submitted for publication within 12 months of completion of the study.
  • Publication restrictions are in place

Restriction type: OTHER