Trial Outcomes & Findings for Efficacy and Safety Study of GPX-150 to Treat Soft Tissue Sarcoma (NCT NCT02267083)

Last Updated: 2018-01-10

Results Overview

The primary efficacy endpoint is the number of patients who were progression-free at 12 months, which is obtained by inversion of the Kaplan-Meier curve for progression-free survival (PFS) at 12 months. Of note, the statistical comparison to historical sarcoma data described in the protocol was not performed due to an enrollment of less than the planned sample size of 30 subjects. Progression is defined using RECIST 1.1, as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

12 months from the beginning of study treatment

Results posted on

2018-01-10

Participant Flow

Participant milestones

Participant milestones
Measure	GPX-150 GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Overall Study STARTED	22
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	19

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety Study of GPX-150 to Treat Soft Tissue Sarcoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GPX-150 n=22 Participants GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Age, Continuous	59.4 years STANDARD_DEVIATION 13.6 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Region of Enrollment United States	22 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months from the beginning of study treatment

Population: Consists of all subjects in the safety population (22 subjects) and who had at least one on-study tumor assessment using RECIST 1.1 (19 subjects). One subject withdrew consist before first on-study tumor assessment and two subjects died before first on-study tumor assessment.

Outcome measures

Outcome measures
Measure	GPX-150 n=19 Participants GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Number of Subjects Progression-free at 12 Months Per RECIST 1.1	3 Participants

SECONDARY outcome

Timeframe: 6 months from the beginning of the study treatment

Population: Consists of all subjects in the safety population who had at least one on-study tumor assessment using RECIST 1.1 (19 subjects). One subject withdrew consent before first on-study tumor assessment and two subjects died before first on-study tumor assessment.

This secondary efficacy endpoint is the progression-free rate (PFR) at 6 months, obtained from the Kaplan-Meier curve for progression-free survival (PFS).

Outcome measures

Outcome measures
Measure	GPX-150 n=19 Participants GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Number of Subjects Progression-free at Six Months Per RECIST 1.1	8 Participants

SECONDARY outcome

Timeframe: From the beginning of study treatment and up to 12 months

Population: Subjects who received at least one dose of GPX-150 were included in the safety analyses.

Subjects who received at least one dose were included in safety analyses. Adverse events were tabulated by System Organ Class (SOC) and Preferred Term (PT) and coded using MedDRA Version 19.1. Safety and tolerability was determined by frequency, nature, and severity of adverse events and the profile of toxicities.

Outcome measures

Outcome measures
Measure	GPX-150 n=22 Participants GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Number of Subjects Experiencing Adverse Events	22 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Assessed during screening, then every 6 weeks for the first 24 weeks on study, and then every 9 weeks for the next 24 weeks for up to 1 year. Subjects will be in the study for up to 1 year, or until disease progression or unacceptable toxicity.

Population: An on-study tumor assessment was done on 19 subjects within the ITT population; 2 subjects died before having a RECIST 1.1 measurement.

Tumor assessments using contrast enhanced computerized tomography (CT) scan of the Chest/Abdomen/Pelvis were performed to assess overall tumor burden. Response rate (RR), where response is defined as complete response (CR), partial response (PR), or stable disease (SD). Determination of CR or PR requires confirmation at the time of the next tumor assessment. An outcome of SD requires at least one assessment 6 weeks after the initiation of dosing. Progression is defined using RECIST 1.1, as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study).

Outcome measures

Outcome measures
Measure	GPX-150 n=19 Participants GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Number of Subjects With Tumor Response Per RECIST 1.1 PD at final turmor assessment	14 Participants
Number of Subjects With Tumor Response Per RECIST 1.1 SD at final tumor assessment	5 Participants
Number of Subjects With Tumor Response Per RECIST 1.1 CR at final tumor assessment	0 Participants
Number of Subjects With Tumor Response Per RECIST 1.1 PR at final tumor assessment	0 Participants

Adverse Events

GPX-150

Serious events: 22 serious events

Other events: 22 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	GPX-150 n=22 participants at risk GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Blood and lymphatic system disorders Febrile neutropenia	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Blood and lymphatic system disorders Anaemia	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Cardiac disorders Myocardial infarction	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Gastrointestinal disorders Abdominal pain	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Gastrointestinal disorders Diarrhoea	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Gastrointestinal disorders Ileus	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Gastrointestinal disorders Nausea	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Gastrointestinal disorders Small intestine obstruction	4.5% 1/22 • Number of events 2 • 1 year, 7 months
Gastrointestinal disorders Vomiting	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Hepatobiliary disorders Cholecystitis chronic	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Infections and infestations Influenza	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Infections and infestations Soft tissue infection	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Injury, poisoning and procedural complications Fracture	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Investigations Lymphocyte count decreased	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Investigations White blood cell count decreased	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Metabolism and nutrition disorders Dehydration	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Liposarcoma metastatic	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Sarcoma metastatic	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Nervous system disorders Central nervous system lesion	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Nervous system disorders Dizziness	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Nervous system disorders Spinal cord compression	4.5% 1/22 • Number of events 1 • 1 year, 7 months
Respiratory, thoracic and mediastinal disorders Respiratory failure	4.5% 1/22 • Number of events 1 • 1 year, 7 months

Other adverse events

Other adverse events
Measure	GPX-150 n=22 participants at risk GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. GPX-150 for Injection: GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity. The dose of GPX-150 may be reduced by 25% if any dose reduction criteria are met. Two reductions are allowed per subject during the course of the study.
Blood and lymphatic system disorders Anemia	22.7% 5/22 • Number of events 12 • 1 year, 7 months
Gastrointestinal disorders Abdominal pain	31.8% 7/22 • Number of events 10 • 1 year, 7 months
Gastrointestinal disorders Constipation	45.5% 10/22 • Number of events 14 • 1 year, 7 months
Gastrointestinal disorders Diarrhea	18.2% 4/22 • Number of events 4 • 1 year, 7 months
Gastrointestinal disorders Dry mouth	13.6% 3/22 • Number of events 3 • 1 year, 7 months
Gastrointestinal disorders Nausea	59.1% 13/22 • Number of events 26 • 1 year, 7 months
Gastrointestinal disorders Rectal haemorrhage	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Gastrointestinal disorders Stomatitis	9.1% 2/22 • Number of events 5 • 1 year, 7 months
Gastrointestinal disorders Vomiting	45.5% 10/22 • Number of events 20 • 1 year, 7 months
General disorders Chest pain	9.1% 2/22 • Number of events 3 • 1 year, 7 months
General disorders Chills	13.6% 3/22 • Number of events 3 • 1 year, 7 months
General disorders Fatigue	50.0% 11/22 • Number of events 17 • 1 year, 7 months
General disorders Oedema peripheral	13.6% 3/22 • Number of events 3 • 1 year, 7 months
General disorders Pyrexia	9.1% 2/22 • Number of events 4 • 1 year, 7 months
Immune system disorders Hypersensitivity	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Investigations Alanine aminotransferase increased	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Investigations Aspartate aminotransferase increased	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Investigations Blood alkaline phosphatase increased	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Investigations Injection fraction decreased	22.7% 5/22 • Number of events 7 • 1 year, 7 months
Metabolism and nutrition disorders Anorexia nervosa	22.7% 5/22 • Number of events 5 • 1 year, 7 months
Metabolism and nutrition disorders Dehydration	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Metabolism and nutrition disorders Hypokalaemia	9.1% 2/22 • Number of events 8 • 1 year, 7 months
Musculoskeletal and connective tissue disorders Arthralgia	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Musculoskeletal and connective tissue disorders Back pain	27.3% 6/22 • Number of events 8 • 1 year, 7 months
Musculoskeletal and connective tissue disorders Pain in extremity	27.3% 6/22 • Number of events 6 • 1 year, 7 months
Nervous system disorders Dizziness	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Nervous system disorders Headache	13.6% 3/22 • Number of events 5 • 1 year, 7 months
Psychiatric disorders Insomnia	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Congenital, familial and genetic disorders Cough	22.7% 5/22 • Number of events 6 • 1 year, 7 months
Respiratory, thoracic and mediastinal disorders Dyspnoea	13.6% 3/22 • Number of events 3 • 1 year, 7 months
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Skin and subcutaneous tissue disorders Alopecia	9.1% 2/22 • Number of events 2 • 1 year, 7 months
Skin and subcutaneous tissue disorders Pruritis	9.1% 2/22 • Number of events 2 • 1 year, 7 months

Additional Information

Elizabeth Moore, Regulatory Affairs Agent

Gem Pharmaceuticals, LLC

Phone: 8583532019

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place