Trial Outcomes & Findings for Pegylated Arginine Deiminase in Treating Patients With Metastatic Melanoma That Cannot Be Removed by Surgery (NCT NCT00450372)

Results Overview

Response rate is defined as a partial response, PR, and complete response, CR, lasting for at least 30 days per RECIST criteria, v. 1.0. Complete response will be defined as disappearance of all target lesions. Partial response will be defined as at least a 30% decrease in the sum of longest diameter (LD) of target lesions, taking as reference the baseline sum of LD

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

38 participants

Primary outcome timeframe

Up to 16 months

Results posted on

2017-02-08

Participant Flow

Participant milestones

Participant milestones
Measure	Single Arm
Overall Study STARTED	38
Overall Study COMPLETED	37
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Single Arm
Overall Study Physician Decision	1

Baseline Characteristics

Pegylated Arginine Deiminase in Treating Patients With Metastatic Melanoma That Cannot Be Removed by Surgery

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm n=38 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	25 Participants n=5 Participants
Age, Continuous	69 years n=5 Participants
Gender Female	13 Participants n=5 Participants
Gender Male	25 Participants n=5 Participants
Region of Enrollment United States	38 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 16 months

Population: Of the 38 subjects enrolled, only 10 were ASS-positive and 17 were ASS-negative. The remaining 11 subjects, who declined pre-treatment biopsies, were not assessed.

Response rate is defined as a partial response, PR, and complete response, CR, lasting for at least 30 days per RECIST criteria, v. 1.0. Complete response will be defined as disappearance of all target lesions. Partial response will be defined as at least a 30% decrease in the sum of longest diameter (LD) of target lesions, taking as reference the baseline sum of LD

Outcome measures

Outcome measures
Measure	ADI-PED 20: Argininosuccinate Synthetase Positive (ASS+) n=10 Participants Patients with Argininosuccinate Synthetase (ASS) expression present in tumor	ADI-PEG 20: Argininosuccinate Synthetase Negative (ASS-) n=17 Participants Patients without Argininosuccinate Synthetase (ASS) expression present in tumor
Response Rate (Partial and Complete Response) in Patients With or Without ASS Expression Present in Tumor.	0 participants	4 participants

SECONDARY outcome

Timeframe: Up to 16 months

Population: Of the 38 subjects enrolled, only 10 were ASS-positive and 17 were ASS-negative. The remaining 11 subjects, who declined pre-treatment biopsies, were not assessed.

Overall survival will be estimated using the product-limit method of Kaplan \& Meier.

Outcome measures

Outcome measures
Measure	ADI-PED 20: Argininosuccinate Synthetase Positive (ASS+) n=10 Participants Patients with Argininosuccinate Synthetase (ASS) expression present in tumor	ADI-PEG 20: Argininosuccinate Synthetase Negative (ASS-) n=17 Participants Patients without Argininosuccinate Synthetase (ASS) expression present in tumor
Median Overall Survival	9.3 months Interval 2.5 to 9.3	14.6 months Interval 4.4 to 14.6

SECONDARY outcome

Timeframe: Up to 16 months

Population: Of the 38 subjects enrolled, only 10 were ASS-positive and 17 were ASS-negative. The remaining 11 subjects, who declined pre-treatment biopsies, were not assessed.

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Outcome measures

Outcome measures
Measure	ADI-PED 20: Argininosuccinate Synthetase Positive (ASS+) n=10 Participants Patients with Argininosuccinate Synthetase (ASS) expression present in tumor	ADI-PEG 20: Argininosuccinate Synthetase Negative (ASS-) n=17 Participants Patients without Argininosuccinate Synthetase (ASS) expression present in tumor
Median Time to Progression	1.8 months Interval 0.7 to 3.4	3.6 months Interval 1.6 to 12.7

Adverse Events

Single Arm

Serious events: 9 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm n=37 participants at risk
Injury, poisoning and procedural complications Allergic Reaction	2.7% 1/37
Blood and lymphatic system disorders Neutropenia - Grade 3	8.1% 3/37
Blood and lymphatic system disorders Neutropenia - Grade 4	2.7% 1/37
Blood and lymphatic system disorders Thrombocytopenia	2.7% 1/37
Blood and lymphatic system disorders Anaemia	2.7% 1/37
General disorders Fatigue	5.4% 2/37

Other adverse events

Other adverse events
Measure	Single Arm n=37 participants at risk
Blood and lymphatic system disorders Myelosuppression	13.5% 5/37

Additional Information

Lynn Feun MD

UM/Sylvester Comprehensive Cancer Center

Phone: 305-243-4909

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place