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OsteosarcomaPhase 1NCT03628209

Nivolumab or Nivolumab and Azacitidine in Patients With Recurrent, Resectable Osteosarcoma

A Phase 1 study testing Nivolumab, Azacitidine and Post Treatment Surgery in people with Osteosarcoma and Osteosarcoma in Children, run by H. Lee Moffitt Cancer Center and Research Institute at 22 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of this study is to evaluate the safety and efficacy of nivolumab, or nivolumab in combination with azacitidine in participants with recurrent, resectable osteosarcoma Read more about this study

Who is it for?
Ages Up to 39 years
What is being tested?
Nivolumab, Azacitidine and Post Treatment Surgery
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
22 sitesUnited States
Study dates
3 Oct 2019to Apr 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

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 Up to 39 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Participants must have had a histologic diagnosis of osteosarcoma at original diagnosis
  • Disease Status: Patients with an isolated pulmonary recurrence of osteosarcoma can be enrolled on this study.
  • Any history of metastatic disease at a site other than lung would make the patient ineligible for this study.
  • The patient's treating team must consider the patient's disease to be resectable and the patient must be willing to undergo resection of all disease, including any lung lesion meeting criteria for likely metastatic disease, defined as: 3 or more lesions ≥ 3 mm in diameter OR a single lesion ≥ 5 mm.
  • Patients with bilateral disease are eligible provided their disease is considered resectable. Resectable pulmonary nodules are defined as nodules that can be removed without performing a pneumonectomy (e.g., nodules immediately adjacent to the main stem bronchus or main pulmonary vessels).
  • Must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2, using the Karnofsky scale for patients > 16 years of age and the Lansky scale for patients ≤ 16 years of age
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  • Prior Therapy: Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to the start of protocol therapy.
  • Participants must have normal organ and marrow function within 7 days of starting protocol therapy
  • All participants and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent/assent document
  • Additional criteria may apply

Who cannot join

  • Pregnancy or Breast Feeding
  • Males and females of reproductive potential may not participate unless they have agreed to the use of, at minimum, two methods of contraception, with one method being highly effective and the other method being either highly effective or less effective as outlined in study protocol documentation
  • Concomitant Medications: Patients receiving the following are not eligible:
  • Corticosteroids or other immunosuppressive medications
  • Patients who are currently receiving other investigational agents or other anti-cancer therapy
  • Intercurrent Illnesses: Patients with uncontrolled intercurrent illness including, but not limited to:
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  • Ongoing or active infection
  • Symptomatic congestive heart failure
  • Unstable angina pectoris
  • Cardiac arrhythmia
  • Psychiatric illness/social situations that would limit compliance with study requirements
  • Autoimmune disorders: Patients with a history of any Grade autoimmune disorder are not eligible.
  • Asymptomatic laboratory abnormalities (e.g., ANA, rheumatoid factor, altered thyroid function studies) will not render a patient ineligible in the absence of a diagnosis of an autoimmune disorder.
  • Patients with ≥ Grade 2 hypothyroidism due to history of autoimmunity are not eligible. Note: Hypothyroidism due to previous irradiation or thyroidectomy will not impact eligibility
  • Allergies: Patients with a history of allergic reactions attributed to compounds of similar chemical or biologic composition to Nivolumab (e.g., another humanized antibody) or Azacitidine are not eligible
  • Safety and Monitoring: Patients who are considered unable to comply with the safety monitoring requirements of the study are not eligible
  • Patients with known HIV or hepatitis B or C are excluded
  • Patients who have received prior solid organ transplantation are not eligible
  • Patients who have received prior anti-PD-1 directed therapy (mAb or small molecule) are not eligible

Trial locations and contacts

22 sites in 1 country.

22 sites in 1 country

United States22 sites

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045

  • Johns Hopkins University, Sidney Kimmel Cancer Center

    Baltimore, Maryland, 21287

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14203

  • University of North Carolina at Chapel Hill, UNC Lineberger Comprehensive Cancer Center

    Chapel Hill, North Carolina, 27599

  • Carolina Medical Center, Levine Cancer Institute

    Charlotte, North Carolina, 28203

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Duke Health

    Durham, North Carolina, 27708

  • Shand's Hospital for Children at the University of Florida

    Gainesville, Florida, 32610

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106

  • University of Texas M.D. Anderson Cancer Center

    Houston, Texas, 77030

  • Nemours Children's Hospital

    Jacksonville, Florida, 32207

  • University of Kentucky, Markey Cancer Center

    Lexington, Kentucky, 40536

  • Children's Hospital of Los Angeles, USC Norris Comprehensive Cancer Center

    Los Angeles, California, 90027

  • University of Miami - Sylvester Comprehensive Cancer Center

    Miami, Florida, 33136

  • Vanderbilt-Ingram Cancer Center

    Nashville, Tennessee, 37232

  • Nemours Children's Clinic

    Orlando, Florida, 32806

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701

  • H. Lee Moffitt Cancer Center and Research Institute, Coordinating Center

    Tampa, Florida, 33612

  • Alfred I DuPont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

About this study

Official title: A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination With Azacitidine in Patients With Recurrent, Resectable Osteosarcoma

The purpose of this study is to evaluate the safety and efficacy of nivolumab, or nivolumab in combination with azacitidine in participants with recurrent, resectable osteosarcoma

Read the detailed description

Treatment will be administered in 28 day cycles with the first cycle in the neoadjuvant setting. This will be followed by surgery to render the participant in surgical remission. Subsequently the participant will continue to receive treatment for up to 12 additional cycles or until recurrence, whichever occurs first. For participants with known bilateral lung recurrence, the nodule[s] in one lung should be resected, prior to the first cycle of chemotherapy. This trial was initially designed as a Phase 1/2 study; However, phase 2 will not proceed.

Being tested
Nivolumab, Azacitidine, Post Treatment Surgery
Keywords
resectable osteosarcoma
Medical subject headings
Osteosarcoma, Sarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NivolumabDrug

    Also called Opdivo®

    Participants will be treated with Nivolumab intravenously (IV), 3 mg/kg on days 1 and 15 of each cycle.

  • AzacitidineDrug

    Also called Vidaza®

    Phase I Dose Escalation - Dose level 1: NA. Dose level 2: 60 mg/m^2. Dose level 3: 75 mg/m^2. Phase II Expansion - Treated at recommended Phase II dose (RP2D).

  • Post Treatment SurgeryProcedure or surgery

    Also called Standard of Care, Resection of disease

    Resection surgery at end of Cycle 1 treatment, day 28-35.

Study groups in NCT03628209
Study groupRoleWhat they receive
Dose Escalation, Resection, Dose ExpansionExperimentalParticipants will receive 1 cycle of neoadjuvant Nivolumab or Nivolumab + Azacitidine, followed by surgery to render them in surgical remission. Subsequently they will continue to receive Nivolumab or Nivolumab + Azacitidine for 12 additional cycles or until recurrence, whichever occurs first. Once the recommended Phase II dose (RP2D) is identified during Phase I, the Dose Expansion Phase II will be opened at this dose level. The Phase II portion of the study will consist of a maximum 33 evaluable patients (27-30 in addition to the 3-6 enrolled at RP2D on the Phase I portion).
Receives: Drug: Nivolumab; Drug: Azacitidine; Procedure: Post Treatment Surgery

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase I: Recommended Phase II Dose (RP2D)

    If one or fewer dose limiting toxicities (DLT's) occur in 6 participants the study will advance to the next dose level. If 2 or more DLT's occur at a dose level, the prior dose level will be identified as the RP2D.

    Measured: 60 days

  2. Phase II: Rate of Continued Complete Remission (CR)

    Continued complete remission by computed tomography (CT) scan 1 year after surgery.

    Measured: 1 year post surgery

Secondary outcomes (2)

  1. Percentage of Participants with Event Free Survival (EFS)

    EFS: The time from diagnostic biopsy until the earliest of: death, local recurrence, new metastatic disease, progression of metastatic disease or secondary malignancy, or date of last contact.

    Measured: 1 year post surgery

  2. Overall Survival (OS) Rate

    The percentage of participants alive at 1 year post surgery.

    Measured: 1 year post surgery

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Aug 2018
  2. Study start3 Oct 2019
  3. Primary completion29 Dec 2024
  4. Study completionApr 2027 (estimated)

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Phase Ib lead-in with extension to Phase II
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03628209
Sponsor's protocol ID
MCC-19487
Other IDs
CA209-9WW
Status last verified
Jul 2026
First submitted
8 Aug 2018
Last updated
30 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Nivolumab, Azacitidine and Post Treatment Surgery in people with Osteosarcoma and Osteosarcoma in Children, run by H. Lee Moffitt Cancer Center and Research Institute at 22 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 10 inclusion and 19 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: under way, not looking for participants, as of the sponsor's last update on 30 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 22 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

Phase I: Recommended Phase II Dose (RP2D) (time frame: 60 days). 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 NCT03628209.

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 30 Jul 2026.

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Clinical Trial NCT03628209 | AzTrials