Soft Tissue SarcomaPhase 2NCT02267083
Efficacy and Safety Study of GPX-150 to Treat Soft Tissue Sarcoma
A Phase 2 study testing GPX-150 for Injection in people with Soft Tissue Sarcoma, run by Gem Pharmaceuticals at 4 sites in United States.
Trial summary
This study will assess the safety and efficacy of GPX-150 administered intravenously every 3 weeks in the treatment of patients with soft tissue sarcoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Soft Tissue Sarcoma
- What is being tested?
- GPX-150 for Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 4 sitesUnited States
- Study dates
- 7 Jan 2015to 18 Aug 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years.
- Histological documentation of soft tissue sarcoma (biopsy may be historical and may have been obtained from primary tumor or a metastatic site).
- Advanced and/or metastatic malignant soft tissue sarcoma of intermediate or high histologic grade. Excluded are the following sarcoma subtypes:
- Well-differentiated liposarcoma or atypical lipomatous tumor
- Embryonal or alveolar rhabdomyosarcoma
- Ewing sarcoma of soft tissue or bone
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- Gastrointestinal stromal tumor (GIST)
- Dermatofibrosarcoma protuberans
- Alveolar soft part sarcoma
- Solitary fibrous tumor
- Clear cell sarcoma
- Kaposi sarcoma
- Extraskeletal myxoid chondrosarcoma
- PEComa (perivascular epithelial cell tumor)
- Myoepithelioma / mixed tumor
- Measurable disease as per RECIST 1.1.
- Subject has received either:
- No prior chemotherapy for current sarcoma, or
- A single course of gemcitabine and/or docetaxel as adjuvant therapy that was completed at least 6 months prior to planned first dose
- ECOG Performance Status of 0 - 2.
- Adequate cardiac function:
- LVEF above the institution's lower limit of normal
- QTcF ≤ 450 msec for males or 470 msec for females.
- Willing and able to provide written informed consent.
- Male and female subjects must agree to use a highly reliable method of birth control for the duration of the study.
- Women of childbearing potential must have a serum pregnancy test performed within 28 days prior to the first day of study drug dosing.
Who cannot join
- Sarcomas arising from bone or cartilage, e.g. chondrosarcoma, osteosarcoma, chordoma.
- Subject is eligible for a potentially curative therapy.
- Prior primary chemotherapy.
- Prior radiotherapy to > 25% of bone marrow volume.
- Treatment within 28 days prior to Dose 1 with:
- Palliative surgery or radiotherapy.
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- Approved anticancer therapy including chemotherapy or immunotherapy.
- Contraindicated treatments noted in the product labelling for doxorubicin, including trastuzumab and inhibitors and inducers of CYP3A4, CYP2D6, or P-gp.
- An investigational therapy.
- Any major surgery (e.g. requiring general anesthesia).
- Inadequate bone marrow, liver, and renal function, as assessed by the following laboratory parameters:
- Absolute neutrophil count (ANC) < 1,500/mm3.
- Platelet count < 100,000/mm3.
- Total bilirubin > 1.5×ULN (upper limit of normal).
- ALT and AST > 2.5×ULN. For patients with documented liver metastases, ALT and AST > 5×ULN.
- Serum creatinine > 1.5 x ULN.
- International Normalized Ratio (INR) and activated partial thromboplastin time [PTT] ≥ 1.5×ULN, if not therapeutically anticoagulated.
- Serum albumin < 3.0 gm/dL.
- Congestive heart failure > Class II New York Heart Association Functional Classification, current pericarditis, myocardial infarction within 6 months, or symptomatic coronary artery disease.
- Unstable or clinically significant concurrent medical condition that would, in the opinion of the investigator, jeopardize the safety of a patient and/or their compliance with the protocol.
- Active infection requiring systemic antibacterial/antibiotic, antifungal, or antiviral therapy.
- Documented metastases to brain or meninges.
- Any malignancy other than soft tissue sarcoma within the last 5 years prior to screening, with the exception of cervical carcinoma in situ, basal cell carcinoma, or superficial bladder tumors that have been successfully and curatively treated with no evidence of recurrent or residual disease.
- Body surface area (BSA) ≥ 2.4 m2.
- Currently pregnant or nursing.
- Known allergy to any of the study drugs or their excipients.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Northwestern University
Chicago, Illinois, 60611
Penn State Milton S Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Iowa Holden Comprehensive Cancer Center
Iowa City, Iowa, 52242
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: Phase 2 Efficacy and Safety Study of Intravenous GPX-150, an Anthracycline Analog, in Patients With Soft Tissue Sarcoma
This study will assess the safety and efficacy of GPX-150 administered intravenously every 3 weeks in the treatment of patients with soft tissue sarcoma.
Read the detailed description
This is an open-label, single arm study of GPX-150 in patients with soft tissue sarcoma. Approximately 22 patients will be treated in this study. The population for this study is adult patients with histologically proven advanced and/or metastatic malignant soft tissue sarcoma of intermediate or high histologic grade.
All patients who meet all entry criteria will receive GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity or subject withdrawal.
Prior to initiation of treatment, subjects will undergo screening and baseline evaluations. During all study visits, subjects will be evaluated for safety. The dose of GPX-150 may be reduced when subjects meet specified dose reduction safety criteria. Subjects will be evaluated regularly for safety and tolerability. Tumor measurements will be calculated at baseline (within 28 days prior to treatment initiation), then at regular intervals while receiving treatment for up to 1 year. After discontinuing the treatment phase of the study, safety assessments and tumor measurements will be performed 3 weeks after the last dose of study drug.
- Conditions
- Soft Tissue Sarcoma
- Being tested
- GPX-150 for Injection
- Keywords
- soft tissue sarcoma
- Medical subject headings
- Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GPX-150 for InjectionDrug
Also called GPX-150, 5-imino-13-deoxy-doxorubicin HCl
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.
| Study group | Role | What they receive |
|---|---|---|
| GPX-150 | Experimental | GPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. Receives: Drug: GPX-150 for Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects Progression-free at 12 Months Per RECIST 1.1
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).
Measured: 12 months from the beginning of study treatment
Secondary outcomes (2)
Number of Subjects Progression-free at Six Months Per RECIST 1.1
This secondary efficacy endpoint is the progression-free rate (PFR) at 6 months, obtained from the Kaplan-Meier curve for progression-free survival (PFS).
Measured: 6 months from the beginning of the study treatment
Number of Subjects Experiencing Adverse Events
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.
Measured: From the beginning of study treatment and up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2014 (estimated)
- Study start7 Jan 2015
- Primary completion11 Nov 2015
- Study completion18 Aug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Soft Tissue SarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gem Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mohammed Milhem, MD, Principal investigator, University of Iowa Holden Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02267083
- Sponsor's protocol ID
- GPX-150-002
- Status last verified
- Dec 2017
- First submitted
- 23 Sep 2014
- Last updated
- 10 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Efficacy and Safety Study of Intravenous GPX-150, an Anthracycline Analog, in Patients With Soft Tissue Sarcoma
- ClinicalTrials.gov ID
- NCT02267083
- Acronym / sponsor ID
- GPX-150-002
- Conditions
- Soft Tissue Sarcoma
- Keywords
- soft tissue sarcoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Gem PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 7 Jan 2015
- Primary completion
- 11 Nov 2015 (actual)
- Study completion
- 18 Aug 2016 (actual)
- First posted
- 17 Oct 2014 (estimated)
- Results
- Posted 10 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will assess the safety and efficacy of GPX-150 administered intravenously every 3 weeks in the treatment of patients with soft tissue sarcoma.
Detailed description
This is an open-label, single arm study of GPX-150 in patients with soft tissue sarcoma. Approximately 22 patients will be treated in this study. The population for this study is adult patients with histologically proven advanced and/or metastatic malignant soft tissue sarcoma of intermediate or high histologic grade.
All patients who meet all entry criteria will receive GPX-150 at a starting dose of 265 mg/m2 every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity or subject withdrawal.
Prior to initiation of treatment, subjects will undergo screening and baseline evaluations. During all study visits, subjects will be evaluated for safety. The dose of GPX-150 may be reduced when subjects meet specified dose reduction safety criteria. Subjects will be evaluated regularly for safety and tolerability. Tumor measurements will be calculated at baseline (within 28 days prior to treatment initiation), then at regular intervals while receiving treatment for up to 1 year. After discontinuing the treatment phase of the study, safety assessments and tumor measurements will be performed 3 weeks after the last dose of study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GPX-150 | Experimental | Drug: GPX-150 for InjectionGPX-150 for Injection, 265 mg/m2, every 21 days for 16 cycles or until death, disease progression, or unacceptable toxicity, or subject withdrawal. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (26 listed)
- Age ≥18 years.
- Histological documentation of soft tissue sarcoma (biopsy may be historical and may have been obtained from primary tumor or a metastatic site).
- Advanced and/or metastatic malignant soft tissue sarcoma of intermediate or high histologic grade. Excluded are the following sarcoma subtypes:
- Measurable disease as per RECIST 1.1.
- Subject has received either:
Key exclusion criteria (26 listed)
- Sarcomas arising from bone or cartilage, e.g. chondrosarcoma, osteosarcoma, chordoma.
- Subject is eligible for a potentially curative therapy.
- Prior primary chemotherapy.
- Prior radiotherapy to > 25% of bone marrow volume.
- Treatment within 28 days prior to Dose 1 with:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GPX-150 for InjectionAlso: GPX-150, 5-imino-13-deoxy-doxorubicin HClGPX-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. | Drug | GPX-150 |
Primary outcome
Number of Subjects Progression-free at 12 Months Per RECIST 1.1
Time frame: 12 months from the beginning of study treatment
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).
2 secondary outcomes
Number of Subjects Progression-free at Six Months Per RECIST 1.1
Time frame: 6 months from the beginning of the study treatment
Number of Subjects Experiencing Adverse Events
Time frame: From the beginning of study treatment and up to 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mohammed Milhem, MD principal investigator
University of Iowa Holden Comprehensive Cancer Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GPX-150 for Injection in people with Soft Tissue Sarcoma, run by Gem Pharmaceuticals at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 26 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jan 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects Progression-free at 12 Months Per RECIST 1.1 (time frame: 12 months from the beginning of study treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2018.