Trial Outcomes & Findings for Palifosfamide in Treating Patients With Recurrent Germ Cell Tumors (NCT NCT01808534)

NCT ID: NCT01808534

Last Updated: 2015-07-02

Results Overview

The percent of patients who were shown as having a partial remission or better based on definitions of response in RECIST 1.1. At least a 30% decrease in the sum of the diameters of target lesions, in reference to baseline sum diameters, needs to be confirmed to be considered as partial response or better. Note: There were no patients with a partial or complete response.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

Up to 2 years

Results posted on

2015-07-02

Participant Flow

This protocol was based on a two stage design, getting 12 patients at the first stage and 8 more if at least one CR or PR was observed in the first 12. Due to low accrual, the study was stopped at 5 patients for this study.

Participant milestones

Participant milestones
Measure
Treatment (Palifosfamide)
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Study
STARTED
5
Overall Study
COMPLETED
1
Overall Study
NOT COMPLETED
4

Reasons for withdrawal

Reasons for withdrawal
Measure
Treatment (Palifosfamide)
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Study
Disease progression
3
Overall Study
Physician Decision
1

Baseline Characteristics

Palifosfamide in Treating Patients With Recurrent Germ Cell Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment (Palifosfamide)
n=5 Participants
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
5 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
36.6 years
STANDARD_DEVIATION 13.83 • n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
4 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
1 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
4 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Up to 2 years

Population: All patients who enrolled and received treatment with at least one post baseline assessment.

The percent of patients who were shown as having a partial remission or better based on definitions of response in RECIST 1.1. At least a 30% decrease in the sum of the diameters of target lesions, in reference to baseline sum diameters, needs to be confirmed to be considered as partial response or better. Note: There were no patients with a partial or complete response.

Outcome measures

Outcome measures
Measure
Treatment (Palifosfamide)
n=5 Participants
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Response Rate (Defined as Partial Response or Complete Response)
0 percentage of participants
Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Up to 2 years

The duration of remission is from the time of confirmed partial or complete response until progression or death. Patients continuing in remission at the end of the study will be treated as censored. Summarized by Kaplan-Meier methods including 90% confidence intervals for the median using method of Brookmeyer and Crowley. Note: There were no patients who achieved partial or complete response.

Outcome measures

Outcome measures
Measure
Treatment (Palifosfamide)
n=5 Participants
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Duration of Remission in Patients Who Achieve a Partial or Complete Response
0 months
Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Up to 2 years

Population: All patients who enrolled and received treatment.

Summarized by Kaplan-Meier methods including 90% confidence intervals for the median using method of Brookmeyer and Crowley. Time until progression, death or last evaluation will be calculated. If a patient did not progress or die, they will be censored at their last evaluation in the analysis.

Outcome measures

Outcome measures
Measure
Treatment (Palifosfamide)
n=5 Participants
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Progression Free Survival (PFS)
0.69 months
Interval 0.07 to 6.18

SECONDARY outcome

Timeframe: Up to 2 years

Population: All patients who enrolled and received treatment.

Summarized by Kaplan-Meier methods including 90% confidence intervals for the median using method of Brookmeyer and Crowley. Time until death or last evaluation will be calculated. If a patient did not die, they will be censored in the analysis at their last known alive date.

Outcome measures

Outcome measures
Measure
Treatment (Palifosfamide)
n=5 Participants
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Survival
0.95 months
Interval 0.56 to 15.63

SECONDARY outcome

Timeframe: Up to 2 years

Population: All patients who enrolled and received treatment.

Number of unique patients who had a treatment related (possible, probable or definite) adverse event that was graded 3 or greater according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.03.

Outcome measures

Outcome measures
Measure
Treatment (Palifosfamide)
n=5 Participants
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Treatment Related Adverse Events Grade 3 or Higher
1 participants

Adverse Events

Treatment (Palifosfamide)

Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Treatment (Palifosfamide)
n=5 participants at risk
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders
Small intestinal obstruction
20.0%
1/5 • Up to 2 years
Metabolism and nutrition disorders
Hypoglycemia
20.0%
1/5 • Up to 2 years

Other adverse events

Other adverse events
Measure
Treatment (Palifosfamide)
n=5 participants at risk
Patients receive palifosfamide IV over 30 minutes on days 1-3. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders
Abdominal pain
20.0%
1/5 • Up to 2 years
Gastrointestinal disorders
Constipation
60.0%
3/5 • Up to 2 years
Gastrointestinal disorders
Diarrhea
20.0%
1/5 • Up to 2 years
Gastrointestinal disorders
Nausea
40.0%
2/5 • Up to 2 years
General disorders
Edema limbs
20.0%
1/5 • Up to 2 years
General disorders
Fatigue
80.0%
4/5 • Up to 2 years
General disorders
Non-cardiac chest pain
20.0%
1/5 • Up to 2 years
General disorders
Pain
20.0%
1/5 • Up to 2 years
Metabolism and nutrition disorders
Anorexia
40.0%
2/5 • Up to 2 years
Musculoskeletal and connective tissue disorders
Back pain
40.0%
2/5 • Up to 2 years
Nervous system disorders
Peripheral sensory neuropathy
20.0%
1/5 • Up to 2 years
Psychiatric disorders
Insomnia
20.0%
1/5 • Up to 2 years
Renal and urinary disorders
Acute kidney injury
20.0%
1/5 • Up to 2 years
Respiratory, thoracic and mediastinal disorders
Dyspnea
60.0%
3/5 • Up to 2 years
Respiratory, thoracic and mediastinal disorders
Hypoxia
20.0%
1/5 • Up to 2 years
Respiratory, thoracic and mediastinal disorders
Pleural effusion
20.0%
1/5 • Up to 2 years
Skin and subcutaneous tissue disorders
Pruritus
20.0%
1/5 • Up to 2 years
Vascular disorders
Thromboembolic event
20.0%
1/5 • Up to 2 years

Additional Information

Dr. Lawrence H. Einhorn

IndianaU

Phone: (317)274-3515

Results disclosure agreements

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