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

NCT02267083 | Completed Phase 2 Results

• 1 other identifier: GPX-150-002
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Soft Tissue Sarcoma

Keywords

soft tissue sarcoma

Outcome Measures

Primary Outcomes (1)

• 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).

  12 months from the beginning of study treatment

Secondary Outcomes (2)

• Number of Subjects Progression-free at Six Months Per RECIST 1.1

  6 months from the beginning of the study treatment

• Number of Subjects Experiencing Adverse Events

  From the beginning of study treatment and up to 12 months

Other Outcomes (1)

• Number of Subjects With Tumor Response Per RECIST 1.1

  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.

Study Arms (1)

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.

Drug: GPX-150 for Injection

Interventions

GPX-150 for Injection DRUG

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.

Also known as: GPX-150, 5-imino-13-deoxy-doxorubicin HCl

GPX-150

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Gem Pharmaceuticals: lead

Study Sites (4)

Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Iowa Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Penn State Milton S Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

MeSH Terms

Conditions

Sarcoma

Interventions

5-imino-13-deoxydoxorubicin; Injections

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Elizabeth Moore, Regulatory Affairs Agent
• Organization: Gem Pharmaceuticals, LLC

elizabethmoore109@comcast.net

8583532019

Study Officials

• Mohammed Milhem, MD

  University of Iowa Holden Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2014
• First Posted: October 17, 2014
• Study Start: January 7, 2015
• Primary Completion: November 11, 2015
• Study Completion: August 18, 2016
• Last Updated: January 10, 2018
• Results First Posted: January 10, 2018

Record last verified: 2017-12

Locations

US (4)