OsteosarcomaPhase 2NCT03006848
A Phase II Trial of Avelumab in Patients With Recurrent or Progressive Osteosarcoma
A Phase 2 study testing Avelumab and Questionnaires in people with Osteosarcoma, run by St. Jude Children's Research Hospital at 4 sites in United States.
Trial summary
This clinical trial seeks to determine if avelumab will be effective in facilitating removal of all gross tumor in the event of a relapse of osteosarcoma in pediatric patients. Avelumab will be evaluated using dosing that has previously been determined in adult studies. Read more about this study
- Who is it for?
- Ages 12 to 49 years
- Conditions the study is for
- Osteosarcoma
- What is being tested?
- Avelumab and Questionnaires
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 4 sitesUnited States
- Study dates
- 16 Feb 2017to 18 Mar 2020
- 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 12 to 49 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be > 12 years of age but < 50 years of age at the time of enrollment.
- Patients must have histologic verification of osteosarcoma at initial diagnosis or relapse.
- Patients must have had evidence of having relapsed, progressed or become refractory to conventional therapy.
- Patients must have measurable disease, documented by clinical, radiographic or histologic criteria. Disease must be bi-dimensionally measurable by computed tomography (CT) or magnetic resonance imaging (MRI).
- Patients must have a performance status of ≥ 50 using the Karnofsky scale for patients > 16 years of age and the Lansky scale for patients ≤ 16 years of age.
- Patients must have a life expectancy of ≥ 6 weeks.
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- Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
- Myelosuppressive chemotherapy: must not have received within 3 weeks of entry onto this study.
- Biologic (anti-neoplastic agent): at least 7 days since the completion of therapy with a biologic agent.
- Immunotherapies: at least 42 days must have elapsed since a prior therapy that included a monoclonal antibody or any other type of immunotherapy (e.g. chimeric antigen receptor (CAR) T cell therapy).
- Radiation therapy (RT): ≥ 2 weeks for local palliative RT (small port); ≥ 6 months must have elapsed if prior craniospinal RT or if ≥ 50% radiation of the pelvis; ≥ 6 weeks must have elapsed if other substantial bone marrow (BM) radiation.
- Organ Function Requirements:
- Adequate bone marrow function defined as:
- Peripheral absolute neutrophil count (ANC) ≥ 1500/mm3
- Platelet count ≥ 100,000/mm3 (transfusion independent)
- Hemoglobin ≥ 9.0 g/dL (may receive RBC transfusions)
- Adequate renal function defined as:
- Creatinine clearance or radioisotope GFR ≥70 mL/min/1.73m2 OR
- Serum creatinine based on age/gender as follows: (threshold creatinine values were derived from the Schwartz formula for estimating GFR).
- Age is: 12 to <13 years, then maximum creatinine is 1.2 mg/DL for male and female.
- Age is: 13 to <16 years, then maximum creatinine is 1.5 mg/DL for male and 1.4 mg/DL for female.
- Age is: ≥16 years, then maximum creatinine is 1.7 mg/DL for male and 1.4 mg/DL for female.
- Adequate liver function defined as:
- Total Bilirubin ≤ 1.5x the institutional upper limit of normal (IULN) for age
- ALT (SGPT) and AST (SGOT) < 2.5 x IULN for age (or < 5 x IULN for patients with documented metastatic disease to the liver)
- Serum albumin > 2 g/dL
- Serum lipase ≤ upper limit of normal (IULN).
- Patients must have documented pulse oximetry ≥ 92% on room air.
- Female patients of childbearing potential must have a negative serum or urine pregnancy test within 7 days of enrollment.
- Male or female patients who are sexually active and of reproductive potential must agree to use an effective contraceptive method throughout the study and for at least 60 days after last avelumab treatment administration. Abstinence is an acceptable form of contraception.
- Patients must not currently be using other investigational agents.
- Patients must not currently be using other anti-cancer agent.
- Patients must be able to comply with the safety monitoring of the study in the opinion of the investigator.
- Written, informed consent and assent following Institutional Review Board, NCI, FDA and OHRP guidelines.
Who cannot join
- Central nervous system (CNS) metastases.
- Current use of immunosuppressive medication, EXCEPT for the following: a. intranasal, inhaled, topical steroids, or local steroid injection (e.g., intra-articular injection); b. Systemic corticosteroids at physiologic doses ≤ 10 mg/day of prednisone or equivalent; c. Steroids as premedication for hypersensitivity reactions (e.g., CT scan premedication).
- Active autoimmune disease that might deteriorate when receiving an immuno-stimulatory agent. Patients with diabetes type I, vitiligo, psoriasis, or hypo- or hyperthyroid diseases not requiring immunosuppressive treatment are eligible.
- Active infection requiring systemic therapy.
- Known history of testing positive for HIV or known acquired immunodeficiency syndrome.
- Hepatitis B virus (HBV) or hepatitis C virus (HCV) infection at screening (positive HBV surface antigen or HCV RNA if anti-HCV antibody screening test positive).
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- Patient who has received vaccination within 4 weeks of the first dose of avelumab and while on trials is prohibited except for administration of inactivated vaccines.
- Known prior severe hypersensitivity to investigational product or any component in its formulations, including known severe hypersensitivity reactions to monoclonal antibodies (NCI CTCAE v4.03 Grade ≥ 3).
- Clinically significant (i.e., active) cardiovascular disease: cerebral vascular accident/stroke (< 6 months prior to enrollment), myocardial infarction (< 6 months prior to enrollment), unstable angina, congestive heart failure (≥ New York Heart Association Classification Class II, see Appendix II), or serious cardiac arrhythmia requiring medication.
- Persisting toxicity related to prior therapy (NCI CTCAE v. 4.03 Grade > 1); however, alopecia, sensory neuropathy Grade ≤ 2, or other Grade ≤ 2 not constituting a safety risk based on investigator's judgment are acceptable
- Other severe acute or chronic medical conditions including colitis, inflammatory bowel disease, pneumonitis, pulmonary fibrosis or psychiatric conditions including recent (within the past year) or active suicidal ideation or behavior; or laboratory abnormalities that may increase the risk associated with study participation or study treatment administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
- Patients with active diarrhea > CTCAE v4.03 Grade 2.
- Patients who have previously received a prior organ transplantation, including allogeneic stem cell transplantation.
- Female patients who are pregnant or actively breastfeeding.
- Patients who have previously received anti-PD1 or anti-PD-L1 therapy. Patients who have previously received anti-CTLA-4 therapy (e.g. ipilimumab) are eligible for study.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Texas Children's Cancer Center
Houston, Texas, 77030
Children's Hospital Los Angeles
Los Angeles, California, 90027
St Jude Children's Research Hospital
Memphis, Tennessee, 38105
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Phase II Trial of Avelumab, A Fully Human Antibody That Targets Cells Expressing PD-L1, in Patients With Recurrent or Progressive Osteosarcoma
This clinical trial seeks to determine if avelumab will be effective in facilitating removal of all gross tumor in the event of a relapse of osteosarcoma in pediatric patients. Avelumab will be evaluated using dosing that has previously been determined in adult studies.
Primary Objectives:
Read more
- To estimate the response rate to 4 cycles of avelumab in patients with recurrent or progressive osteosarcoma.
- To estimate the 16-week progression free survival of patients with recurrent or progressive osteosarcoma after treatment with avelumab.
Secondary Objective:
- To describe the toxicities associated with the administration of avelumab in patients with recurrent or progressive osteosarcoma.
- To assess the quality of life of patients with recurrent or progressive osteosarcoma undergoing treatment with avelumab, and to explore relationships between clinical factors and patient-reported health-related quality of life (HRQOL) outcomes.
Exploratory Objectives:
- To explore factors associated with response in patients treated with avelumab after recurrent or progressive osteosarcoma (e.g. tumor PD-L1 expression).
- To measure parameters of immune activation including subsets of peripheral blood mononuclear cells (PBMCs) and serum markers of immune activation.
- To evaluate the role of T-cells in immune checkpoint blockade via measures of cell proliferation, co-inhibitory receptor expression on CD8 T cells, T cell repertoire, and epigenetic programming.
Read the detailed description
This is a Phase 2 study using a traditional Simon two-stage design. Patients 12 years or greater with recurrent/refractory osteosarcoma 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. Progression free survival and response to therapy after 4 cycles of treatment will be assessed. In addition, the toxicity profile of avelumab in this population will be closely monitored.
- Conditions
- Osteosarcoma
- Being tested
- Avelumab, Questionnaires
- Keywords
- Osteosarcoma, PD-L1, Immunotherapy
- Medical subject headings
- Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvelumabDrug
Also called MSB0010718C, anti-PD-L1
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.
QuestionnairesOther
Also called PROMIS Pediatric Profile, PROMIS Adult Profile
To assess quality of life, patients will complete questionnaires at four time points.
| Study group | Role | What they receive |
|---|---|---|
| Avelumab | Experimental | All participants with recurrent/refractory osteosarcoma who consent to the study.
Interventions: Avelumab and quality of life questionnaires. Receives: Drug: Avelumab; Other: Questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Rate
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.
Measured: At the end of 4 cycles of avelumab (approximately 4 months)
Progression-free Survival
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.
Measured: At the end of 4 cycles of avelumab (approximately 4 months)
Secondary outcomes (1)
Target Toxicities
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).
Measured: At the end of treatment (up to 2 years after enrollment of last participant)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2016 (estimated)
- Study start16 Feb 2017
- Primary completion18 Mar 2020
- Study completion18 Mar 2020
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for OsteosarcomaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- 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
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer, Gateway for Cancer Research
- Responsible party
- Sponsor
- Investigators
- Michael W. Bishop, MD, MS, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03006848
- Sponsor's protocol ID
- OSTPDL1
- Other IDs
- Pfizer W1211733 (OSTPDL1), Gateway RESAG
- Status last verified
- Jul 2026
- First submitted
- 22 Dec 2016
- Last updated
- 21 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.
PubMed 31401903
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Avelumab, A Fully Human Antibody That Targets Cells Expressing PD-L1, in Patients With Recurrent or Progressive Osteosarcoma
- ClinicalTrials.gov ID
- NCT03006848
- Acronym / sponsor ID
- OSTPDL1
- Conditions
- Osteosarcoma
- Keywords
- Osteosarcoma, PD-L1, Immunotherapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Pfizer, Gateway for Cancer Research
- Study start
- 16 Feb 2017 (actual)
- Primary completion
- 18 Mar 2020 (actual)
- Study completion
- 18 Mar 2020 (actual)
- First posted
- 30 Dec 2016 (estimated)
- Results
- Posted 16 Sep 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This clinical trial seeks to determine if avelumab will be effective in facilitating removal of all gross tumor in the event of a relapse of osteosarcoma in pediatric patients. Avelumab will be evaluated using dosing that has previously been determined in adult studies.
Primary Objectives:
- To estimate the response rate to 4 cycles of avelumab in patients with recurrent or progressive osteosarcoma.
- To estimate the 16-week progression free survival of patients with recurrent or progressive osteosarcoma after treatment with avelumab.
Secondary Objective:
- To describe the toxicities associated with the administration of avelumab in patients with recurrent or progressive osteosarcoma.
- To assess the quality of life of patients with recurrent or progressive osteosarcoma undergoing treatment with avelumab, and to explore relationships between clinical factors and patient-reported health-related quality of life (HRQOL) outcomes.
Exploratory Objectives:
- To explore factors associated with response in patients treated with avelumab after recurrent or progressive osteosarcoma (e.g. tumor PD-L1 expression).
- To measure parameters of immune activation including subsets of peripheral blood mononuclear cells (PBMCs) and serum markers of immune activation.
- To evaluate the role of T-cells in immune checkpoint blockade via measures of cell proliferation, co-inhibitory receptor expression on CD8 T cells, T cell repertoire, and epigenetic programming.
Detailed description
This is a Phase 2 study using a traditional Simon two-stage design. Patients 12 years or greater with recurrent/refractory osteosarcoma 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. Progression free survival and response to therapy after 4 cycles of treatment will be assessed. In addition, the toxicity profile of avelumab in this population will be closely monitored.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avelumab | Experimental | Drug: Avelumab, Other: QuestionnairesAll participants with recurrent/refractory osteosarcoma who consent to the study. Interventions: Avelumab and quality of life questionnaires. |
Who may qualify
- Ages
- 12 to 49 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (34 listed)
- Patients must be > 12 years of age but < 50 years of age at the time of enrollment.
- Patients must have histologic verification of osteosarcoma at initial diagnosis or relapse.
- Patients must have had evidence of having relapsed, progressed or become refractory to conventional therapy.
- Patients must have measurable disease, documented by clinical, radiographic or histologic criteria. Disease must be bi-dimensionally measurable by computed tomography (CT) or magnetic resonance imaging (MRI).
- Patients must have a performance status of ≥ 50 using the Karnofsky scale for patients > 16 years of age and the Lansky scale for patients ≤ 16 years of age.
Key exclusion criteria (15 listed)
- Central nervous system (CNS) metastases.
- Current use of immunosuppressive medication, EXCEPT for the following: a. intranasal, inhaled, topical steroids, or local steroid injection (e.g., intra-articular injection); b. Systemic corticosteroids at physiologic doses ≤ 10 mg/day of prednisone or equivalent; c. Steroids as premedication for hypersensitivity reactions (e.g., CT scan premedication).
- Active autoimmune disease that might deteriorate when receiving an immuno-stimulatory agent. Patients with diabetes type I, vitiligo, psoriasis, or hypo- or hyperthyroid diseases not requiring immunosuppressive treatment are eligible.
- Active infection requiring systemic therapy.
- Known history of testing positive for HIV or known acquired immunodeficiency syndrome.
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 |
|---|---|---|
| AvelumabAlso: MSB0010718C, anti-PD-L1Patients 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. | Drug | Avelumab |
| QuestionnairesAlso: PROMIS Pediatric Profile, PROMIS Adult ProfileTo assess quality of life, patients will complete questionnaires at four time points. | Other | Avelumab |
Primary outcome
Response Rate
Time frame: At the end of 4 cycles of avelumab (approximately 4 months)
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.
Progression-free Survival
Time frame: At the end of 4 cycles of avelumab (approximately 4 months)
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.
1 secondary outcome
Target Toxicities
Time frame: At the end of treatment (up to 2 years after enrollment of last participant)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31401903Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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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.
Michael W. Bishop, MD, MS principal investigator
St. Jude Children's Research Hospital
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 Avelumab and Questionnaires in people with Osteosarcoma, run by St. Jude Children's Research Hospital 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 34 inclusion and 15 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 21 Jul 2026. 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 16 Sep 2021). 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?
Response Rate (time frame: At the end of 4 cycles of avelumab (approximately 4 months)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03006848, which also lists 1 linked publication.
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 21 Jul 2026.