Trial Outcomes & Findings for A Phase II Trial of Avelumab in Patients With Recurrent or Progressive Osteosarcoma (NCT NCT03006848)

Last Updated: 2025-09-04

Results Overview

The study is designed by treating RECIST response \[complete response + partial response (CR+PR)\] after 4-cycle treatment of avelumab and the 16-week progression-free survival (PFS) as dual binary endpoints. Patients who fail to be evaluated at the end of the 4-cycle will be counted as failure.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

At the end of 4 cycles of avelumab (approximately 4 months)

Results posted on

2025-09-04

Participant Flow

A total of 19 patients were enrolled on the study from February 2017 to April 2021

18 out of 19 enrolled patients received treatment. The patient who did not receive treatment was found to be ineligible and thus was a screen failure.

Participant milestones

Participant milestones
Measure	Avelumab All participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. Avelumab: Patients will be administered avelumab at a dose of 10 mg/kg intravenously (IV) over 60 minutes on days 1 and 15 of each cycle, with a cycle lasting 28 days. Patients will receive avelumab every 2 weeks in cycles of 28 days for up to 24 months, or 26 cycles. Questionnaires: To assess quality of life, patients will complete questionnaires at four time points.
Overall Study STARTED	18
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	18

Reasons for withdrawal

Reasons for withdrawal
Measure	Avelumab All participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. Avelumab: Patients will be administered avelumab at a dose of 10 mg/kg intravenously (IV) over 60 minutes on days 1 and 15 of each cycle, with a cycle lasting 28 days. Patients will receive avelumab every 2 weeks in cycles of 28 days for up to 24 months, or 26 cycles. Questionnaires: To assess quality of life, patients will complete questionnaires at four time points.
Overall Study Adverse Event	1
Overall Study Physician Decision	1
Overall Study Lack of Efficacy	16

Baseline Characteristics

A Phase II Trial of Avelumab in Patients With Recurrent or Progressive Osteosarcoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Avelumab n=18 years All participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. Avelumab: Patients will be administered avelumab at a dose of 10 mg/kg intravenously (IV) over 60 minutes on days 1 and 15 of each cycle, with a cycle lasting 28 days. Patients will receive avelumab every 2 weeks in cycles of 28 days for up to 24 months, or 26 cycles. Questionnaires: To assess quality of life, patients will complete questionnaires at four time points.
Age, Continuous	17.0 years STANDARD_DEVIATION 3.3 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	14 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Declined Respond	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Unknown	1 Participants n=5 Participants
Region of Enrollment United States · St. Jude Children's Research Hospital	14 Participants n=5 Participants
Region of Enrollment United States · Memorial Sloan-Kettering Cancer Center	2 Participants n=5 Participants
Region of Enrollment United States · Children's Hospital of Los Angeles	1 Participants n=5 Participants
Region of Enrollment United States · Texas Children's Research Hospital	1 Participants n=5 Participants
Disease Status at time of enrollment Progressive Disease	13 Participants n=5 Participants
Disease Status at time of enrollment Recurrent Disease	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: At the end of 4 cycles of avelumab (approximately 4 months)

Population: RECIST response \[complete response + partial response (CR+PR)\]

Outcome measures

Outcome measures
Measure	Avelumab n=18 Participants All participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. Avelumab: Patients will be administered avelumab at a dose of 10 mg/kg intravenously (IV) over 60 minutes on days 1 and 15 of each cycle, with a cycle lasting 28 days. Patients will receive avelumab every 2 weeks in cycles of 28 days for up to 24 months, or 26 cycles. Questionnaires: To assess quality of life, patients will complete questionnaires at four time points.
Response Rate	0 percentage of participants Interval 0.0 to 0.0

PRIMARY outcome

Timeframe: At the end of 4 cycles of avelumab (approximately 4 months)

Population: Of the 18 eligible patients who received treatment, 17 patients had disease progression while on study and one patient died off study.

Outcome measures

Outcome measures
Measure	Avelumab n=18 Participants All participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. Avelumab: Patients will be administered avelumab at a dose of 10 mg/kg intravenously (IV) over 60 minutes on days 1 and 15 of each cycle, with a cycle lasting 28 days. Patients will receive avelumab every 2 weeks in cycles of 28 days for up to 24 months, or 26 cycles. Questionnaires: To assess quality of life, patients will complete questionnaires at four time points.
Progression-free Survival	8.0 percentage of weeks Interval 6.7 to 9.1

SECONDARY outcome

Timeframe: At the end of treatment (up to 2 years after enrollment of last participant)

Population: \[Not Specified\]

Target toxicities for avelumab treatment are defined as any grade 3-5 dyspnea, infusion-related reactions, or immune related adverse events at least possibly attributable to the agent observed anytime during the 26-cycle treatment period that a patient is on study (including the period between off treatment and off study).

Outcome measures

Outcome measures
Measure	Avelumab n=18 Participants All participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. Avelumab: Patients will be administered avelumab at a dose of 10 mg/kg intravenously (IV) over 60 minutes on days 1 and 15 of each cycle, with a cycle lasting 28 days. Patients will receive avelumab every 2 weeks in cycles of 28 days for up to 24 months, or 26 cycles. Questionnaires: To assess quality of life, patients will complete questionnaires at four time points.
Target Toxicities Dyspnea	2 participants
Target Toxicities Infection and infestations	1 participants
Target Toxicities Metabolism and nutrition disorders	2 participants
Target Toxicities Blood and lymphatic system disorders	1 participants
Target Toxicities Cardiac disorders	1 participants
Target Toxicities Investigations	2 participants

Adverse Events

Avelumab

Serious events: 7 serious events

Other events: 15 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	Avelumab n=18 participants at risk \[Not specified\]
Vascular disorders Blood and lymphatic system disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Respiratory, thoracic and mediastinal disorders Infections and infestations	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Nervous system disorders Nervous system disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Gastrointestinal disorders Gastrointestinal disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Metabolism and nutrition disorders Metabolism and nutrition disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Investigations Investigations	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Cardiac disorders Cardiac disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]

Other adverse events

Other adverse events
Measure	Avelumab n=18 participants at risk \[Not specified\]
Blood and lymphatic system disorders Blood and lymphatic system disorders	5.6% 1/18 • Number of events 2 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Cardiac disorders Cardiac disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Endocrine disorders Endocrine disorders	5.6% 1/18 • Number of events 2 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Gastrointestinal disorders Gastrointestinal disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
General disorders General disorders and administration site conditions	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Immune system disorders Immune system disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Infections and infestations Infections and infestations	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Injury, poisoning and procedural complications Injury, poisoning and procedural complications	11.1% 2/18 • Number of events 2 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Investigations Investigations	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Metabolism and nutrition disorders Metabolism and nutrition disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorders	16.7% 3/18 • Number of events 4 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Nervous system disorders Nervous system disorders	5.6% 1/18 • Number of events 2 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Renal and urinary disorders Renal and urinary disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders	5.6% 1/18 • Number of events 1 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	5.6% 1/18 • Number of events 2 • Acute adverse events were collected from study enrollment up to 4 weeks after completion of chemotherapy/radiation therapy, whichever came last. Long term toxicity was captured at every visit up to 3 years from study enrollment. \[Not specified\]

Additional Information

Dr. Michael Bishop

St Jude Children's Research Hospital

Phone: 866-278-5833

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place