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

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ClinicalTrials.gov Identifier: NCT02267083
Recruitment Status : Completed
First Posted : October 17, 2014
Results First Posted : January 10, 2018
Last Update Posted : January 10, 2018
Sponsor:
Information provided by (Responsible Party):
Gem Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Soft Tissue Sarcoma
Intervention Drug: GPX-150 for Injection
Enrollment 22
Recruitment Details  
Pre-assignment Details  
Arm/Group Title GPX-150
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 22
Completed 3
Not Completed 19
Arm/Group Title GPX-150
Hide Arm/Group Description

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 Number of Baseline Participants 22
Hide Baseline Analysis Population Description
The Safety Population consists of all subjects who received at least 1 dose of GPX-150 and the Intent-to-Treat (ITT) Population consists of all subjects in the Safety Population who had at least one tumor assessment using RECIST 1.1 or who died prior to any tumor assessment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 22 participants
59.4  (13.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Female
7
  31.8%
Male
15
  68.2%
Region of Enrollment   [1] 
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 22 participants
22
[1]
Measure Description: Subjects diagnosed with advanced and/or metastatic soft tissue sarcoma of intermediate or high histologic grade.
1.Primary Outcome
Title Number of Subjects Progression-free at 12 Months Per RECIST 1.1
Hide Description 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).
Time Frame 12 months from the beginning of study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title GPX-150
Hide Arm/Group Description:

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 Number of Participants Analyzed 19
Measure Type: Count of Participants
Unit of Measure: Participants
3
  15.8%
2.Secondary Outcome
Title Number of Subjects Progression-free at Six Months Per RECIST 1.1
Hide Description This secondary efficacy endpoint is the progression-free rate (PFR) at 6 months, obtained from the Kaplan-Meier curve for progression-free survival (PFS).
Time Frame 6 months from the beginning of the study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title GPX-150
Hide Arm/Group Description:

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 Number of Participants Analyzed 19
Measure Type: Count of Participants
Unit of Measure: Participants
8
  42.1%
3.Secondary Outcome
Title Number of Subjects Experiencing Adverse Events
Hide Description 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.
Time Frame From the beginning of study treatment and up to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who received at least one dose of GPX-150 were included in the safety analyses.
Arm/Group Title GPX-150
Hide Arm/Group Description:

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 Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
22
 100.0%
4.Other Pre-specified Outcome
Title Number of Subjects With Tumor Response Per RECIST 1.1
Hide Description 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).
Time Frame 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.
Hide Outcome Measure Data
Hide Analysis Population Description
An on-study tumor assessment was done on 19 subjects within the ITT population; 2 subjects died before having a RECIST 1.1 measurement.
Arm/Group Title GPX-150
Hide Arm/Group Description:

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 Number of Participants Analyzed 19
Measure Type: Count of Participants
Unit of Measure: Participants
PD at final turmor assessment
14
  73.7%
SD at final tumor assessment
5
  26.3%
CR at final tumor assessment
0
   0.0%
PR at final tumor assessment
0
   0.0%
Time Frame 1 year, 7 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title GPX-150
Hide Arm/Group Description

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.

All-Cause Mortality
GPX-150
Affected / at Risk (%)
Total   2/22 (9.09%)    
Show Serious Adverse Events Hide Serious Adverse Events
GPX-150
Affected / at Risk (%) # Events
Total   22/22 (100.00%)    
Blood and lymphatic system disorders   
Febrile neutropenia   1/22 (4.55%)  1
Anaemia   1/22 (4.55%)  1
Cardiac disorders   
Myocardial infarction   1/22 (4.55%)  1
Gastrointestinal disorders   
Abdominal pain   1/22 (4.55%)  1
Diarrhoea   1/22 (4.55%)  1
Ileus   1/22 (4.55%)  1
Nausea   1/22 (4.55%)  1
Small intestine obstruction   1/22 (4.55%)  2
Vomiting   2/22 (9.09%)  2
Hepatobiliary disorders   
Cholecystitis chronic   1/22 (4.55%)  1
Infections and infestations   
Influenza   1/22 (4.55%)  1
Soft tissue infection   1/22 (4.55%)  1
Injury, poisoning and procedural complications   
Fracture   1/22 (4.55%)  1
Investigations   
Lymphocyte count decreased   1/22 (4.55%)  1
White blood cell count decreased   1/22 (4.55%)  1
Metabolism and nutrition disorders   
Dehydration   1/22 (4.55%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Liposarcoma metastatic   1/22 (4.55%)  1
Sarcoma metastatic   1/22 (4.55%)  1
Nervous system disorders   
Central nervous system lesion   1/22 (4.55%)  1
Dizziness   1/22 (4.55%)  1
Spinal cord compression   1/22 (4.55%)  1
Respiratory, thoracic and mediastinal disorders   
Respiratory failure   1/22 (4.55%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GPX-150
Affected / at Risk (%) # Events
Total   22/22 (100.00%)    
Blood and lymphatic system disorders   
Anemia   5/22 (22.73%)  12
Congenital, familial and genetic disorders   
Cough   5/22 (22.73%)  6
Gastrointestinal disorders   
Abdominal pain   7/22 (31.82%)  10
Constipation   10/22 (45.45%)  14
Diarrhea   4/22 (18.18%)  4
Dry mouth   3/22 (13.64%)  3
Nausea   13/22 (59.09%)  26
Rectal haemorrhage   2/22 (9.09%)  2
Stomatitis   2/22 (9.09%)  5
Vomiting   10/22 (45.45%)  20
General disorders   
Chest pain   2/22 (9.09%)  3
Chills   3/22 (13.64%)  3
Fatigue   11/22 (50.00%)  17
Oedema peripheral   3/22 (13.64%)  3
Pyrexia   2/22 (9.09%)  4
Immune system disorders   
Hypersensitivity   2/22 (9.09%)  2
Investigations   
Alanine aminotransferase increased   2/22 (9.09%)  2
Aspartate aminotransferase increased   2/22 (9.09%)  2
Blood alkaline phosphatase increased   2/22 (9.09%)  2
Injection fraction decreased   5/22 (22.73%)  7
Metabolism and nutrition disorders   
Anorexia nervosa   5/22 (22.73%)  5
Dehydration   2/22 (9.09%)  2
Hypokalaemia   2/22 (9.09%)  8
Musculoskeletal and connective tissue disorders   
Arthralgia   2/22 (9.09%)  2
Back pain   6/22 (27.27%)  8
Pain in extremity   6/22 (27.27%)  6
Nervous system disorders   
Dizziness   2/22 (9.09%)  2
Headache   3/22 (13.64%)  5
Psychiatric disorders   
Insomnia   2/22 (9.09%)  2
Respiratory, thoracic and mediastinal disorders   
Dyspnoea   3/22 (13.64%)  3
Pulmonary embolism   2/22 (9.09%)  2
Skin and subcutaneous tissue disorders   
Alopecia   2/22 (9.09%)  2
Pruritis   2/22 (9.09%)  2
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Elizabeth Moore, Regulatory Affairs Agent
Organization: Gem Pharmaceuticals, LLC
Phone: 8583532019
Responsible Party: Gem Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02267083     History of Changes
Other Study ID Numbers: GPX-150-002
First Submitted: September 23, 2014
First Posted: October 17, 2014
Results First Submitted: August 10, 2017
Results First Posted: January 10, 2018
Last Update Posted: January 10, 2018