Trial Outcomes & Findings for Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) (NCT NCT01270724)

NCT ID: NCT01270724

Last Updated: 2025-10-27

Results Overview

To estimate response rate after at least two and up to four courses of induction chemotherapy with GemPOx regimen in patients with recurrent intracranial MMGCT

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: 16-32 weeks depending on individual patient response (remaining disease burden) to chemotherapy
• Results posted on: 2025-10-27

Participant Flow

Nine patients with confirmed relapsed or refractory intracranial GCT were enrolled after signing informed consent, and received at least two cycles of GemPOx, of which all but one had relapsed or refractory NGGCTs. One patient with progressive disease was found to have pathologically confirmed malignant transformation to pure embryonal rhabdomyosarcoma (without GCT elements), hence was ineligible and not included in the analysis.

Participant milestones

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) Two to four cycles of induction therapy with open label GemPOx followed by consolidation and autologous stem cell transplant (ASCT). Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx).: Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
Overall Study STARTED	10
Overall Study 2012	6
Overall Study 2013	1
Overall Study 2014	1
Overall Study 2016	1
Overall Study 2017	1
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

10 patients were consented and enrolled on therapy; one patient was declared ineligible on path review therefore not included in the final analysis or manuscript. 9 patients total will be included in final analyses.

Baseline characteristics by cohort

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=10 Participants Two to four cycles of induction therapy with open label GemPOx followed by consolidation and autologous stem cell transplant (ASCT). Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx).: Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT. Nine patients with confirmed relapsed or refractory intracranial GCT were enrolled after signing informed consent, and received at least two cycles of GemPOx, of which all but one had relapsed or refractory NGGCTs. One patient with progressive disease was found to have pathologically confirmed malignant transformation to pure embryonal rhabdomyosarcoma (without GCT elements), hence was ineligible and not included in the analysis. Patients who experienced sufficient responses proceeded to receive HDCx with AuHPCR. Treatment response was determined based on radiographic tumor assessments and tumor markers.
Age, Categorical <=18 years	5 Participants n=10 Participants • 10 patients were consented and enrolled on therapy; one patient was declared ineligible on path review therefore not included in the final analysis or manuscript. 9 patients total will be included in final analyses.
Age, Categorical Between 18 and 65 years	5 Participants n=10 Participants • 10 patients were consented and enrolled on therapy; one patient was declared ineligible on path review therefore not included in the final analysis or manuscript. 9 patients total will be included in final analyses.
Age, Categorical >=65 years	0 Participants n=10 Participants • 10 patients were consented and enrolled on therapy; one patient was declared ineligible on path review therefore not included in the final analysis or manuscript. 9 patients total will be included in final analyses.
Sex: Female, Male Female	1 Participants n=10 Participants
Sex: Female, Male Male	9 Participants n=10 Participants
Region of Enrollment United States	10 participants n=10 Participants
Patients enrolled on study with eligible diagnosis of CNS GCT including pure germinoma and MMGCT	9 Participants n=10 Participants

PRIMARY outcome

Timeframe: 16-32 weeks depending on individual patient response (remaining disease burden) to chemotherapy

To estimate response rate after at least two and up to four courses of induction chemotherapy with GemPOx regimen in patients with recurrent intracranial MMGCT

Outcome measures

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=9 Participants Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx): Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
Response Rate CR, Complete Response	0 Participants
Response Rate PR, Partial Response	0 Participants
Response Rate SD, Stable Disease	4 Participants
Response Rate PD, Progressive Disease	2 Participants
Response Rate PR1, Partial Response with Normal Markers	3 Participants
Response Rate PR2, Partial Response with Abnormal (but not rising) Markers	0 Participants

PRIMARY outcome

Timeframe: Mean follow-up of 44 months

Outcome measures

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=9 Participants Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx): Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
The Rate of Completion of Induction Chemotherapy and Progression to High-dose Chemotherapy (HDC) With Autologous Hematopoietic Progenitor Cell Rescue (AuHPCR)	7 Participants

SECONDARY outcome

Timeframe: 2 years, 3 years and 5 years

To assess the overall survival (OS) and event-free survival (EFS) of patients treated on the GemPOx induction regimen followed by the HDC and AuHPCR in patients with progressive or recurrent CNS GCT.

We hypothesize that specific CSF miRNA will prove to be accurate markers for tumor presence and predictors of response to therapy, with normalization being associated with improved progression-free survival (PFS) in patients under treatment for recurrent central nervous system (CNS) germ cell tumors (GCT).

Outcome measures

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=9 Participants Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx): Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
OS and PFS 2-year PFS	66.7 Percentage of patients Interval 28.2 to 87.9
OS and PFS 3-year PFS	55.6 Percentage of patients Interval 20.4 to 80.5
OS and PFS 5-year PFS	44.4 Percentage of patients Interval 13.6 to 71.9
OS and PFS 2-year OS	66.7 Percentage of patients Interval 28.2 to 87.9
OS and PFS 3-year OS	66.7 Percentage of patients Interval 28.2 to 87.9
OS and PFS 5-year OS	55.6 Percentage of patients Interval 20.4 to 80.5

POST_HOC outcome

Timeframe: Months in follow-up (up to 86 months follow-up)

The outcome of each patient at the last point of follow-up

Outcome measures

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=9 Participants Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx): Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
Last Patient Follow-up Outcome PR, Partial Response	0 Participants
Last Patient Follow-up Outcome SD, Stable Disease	0 Participants
Last Patient Follow-up Outcome PD, Progressive Disease	0 Participants
Last Patient Follow-up Outcome NED, No Evidence of Disease	4 Participants
Last Patient Follow-up Outcome DOD, Dead of Disease	4 Participants
Last Patient Follow-up Outcome Unknown, Lost to Follow-up	1 Participants

Adverse Events

Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx)

Serious events: 2 serious events

Other events: 10 other events

Deaths: 4 deaths

Serious adverse events

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=10 participants at risk Two to four cycles of induction therapy with open label GemPOx followed by consolidation and autologous stem cell transplant (ASCT). Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx): Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
Respiratory, thoracic and mediastinal disorders Death	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
General disorders Death	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months

Other adverse events

Measure	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) n=10 participants at risk Two to four cycles of induction therapy with open label GemPOx followed by consolidation and autologous stem cell transplant (ASCT). Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx): Two to four cycles of induction therapy with GemPOx followed by consolidation and ASCT.
Immune system disorders Allergic reaction	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Renal and urinary disorders Acute kidney injury	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations ALT	10.0% 1/10 • Number of events 13 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Anorexia	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations AST	10.0% 1/10 • Number of events 7 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Psychiatric disorders Confusion	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Nervous system disorders Cerebrospinal fluid leakage	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Nervous system disorders Encephalopathy	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
General disorders Fatigue	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations GGT	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations Hemoglobin	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hyperglycemia	10.0% 1/10 • Number of events 2 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hypernatremia	10.0% 1/10 • Number of events 4 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hypocalcemia	10.0% 1/10 • Number of events 2 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hypoglycemia	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hypokalemia	10.0% 1/10 • Number of events 3 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hyponatremia	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Metabolism and nutrition disorders Hypophosphatemia	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Infections and infestations Infections and infestations, other	10.0% 1/10 • Number of events 2 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations Low WBC	10.0% 1/10 • Number of events 11 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations Lymphocyte count decreased	10.0% 1/10 • Number of events 3 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Gastrointestinal disorders Mucositis	10.0% 1/10 • Number of events 4 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Blood and lymphatic system disorders Neutropenia	10.0% 1/10 • Number of events 16 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations Neutrophil count decreased	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Musculoskeletal and connective tissue disorders Pain	10.0% 1/10 • Number of events 3 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Gastrointestinal disorders Pain, oral	10.0% 1/10 • Number of events 2 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Investigations Platelet count decrease	10.0% 1/10 • Number of events 8 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months
Eye disorders Ptosis	10.0% 1/10 • Number of events 1 • Each participant was followed for up to 5 months for adverse events (up to 4 cycles of Induction chemotherapy followed by one cycle of Consolidation therapy), or 30 days after the last dose of study drug was received. All-Cause Mortality was assessed for an average of 44 months

Additional Information

Megan Blue

Nationwide Children's Hospital

Phone: 614-722-3686

Email: megan.blue@nationwidechildrens.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place