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

Phase 1
Active, not recruiting
Conditions
Osteosarcoma Recurrent
Sarcoma
Osteosarcoma in Children
Osteosarcoma
Interventions
Procedure: Post Treatment Surgery
Registration Number
NCT03628209
Lead Sponsor
H. Lee Moffitt Cancer Center and Research Institute
Brief 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

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.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
21
Inclusion Criteria
  • 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

  • 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

Exclusion Criteria
  • 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:

    • 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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Dose Escalation, Resection, Dose ExpansionNivolumabParticipants 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).
Dose Escalation, Resection, Dose ExpansionAzacitidineParticipants 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).
Dose Escalation, Resection, Dose ExpansionPost Treatment SurgeryParticipants 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).
Primary Outcome Measures
NameTimeMethod
Phase I: Recommended Phase II Dose (RP2D)60 days

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.

Phase II: Rate of Continued Complete Remission (CR)1 year post surgery

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

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants with Event Free Survival (EFS)1 year post surgery

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.

Overall Survival (OS) Rate1 year post surgery

The percentage of participants alive at 1 year post surgery.

Trial Locations

Locations (22)

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

🇺🇸

Los Angeles, California, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Nationwide Children's Hospital

🇺🇸

Columbus, Ohio, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Duke Health

🇺🇸

Durham, North Carolina, United States

Vanderbilt-Ingram Cancer Center

🇺🇸

Nashville, Tennessee, United States

University of Texas M.D. Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Children's Hospital of Colorado

🇺🇸

Aurora, Colorado, United States

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

🇺🇸

Tampa, Florida, United States

University of Miami - Sylvester Comprehensive Cancer Center

🇺🇸

Miami, Florida, United States

Johns Hopkins University, Sidney Kimmel Cancer Center

🇺🇸

Baltimore, Maryland, United States

Roswell Park Comprehensive Cancer Center

🇺🇸

Buffalo, New York, United States

Carolina Medical Center, Levine Cancer Institute

🇺🇸

Charlotte, North Carolina, United States

University of Texas Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Nemours Children's Hospital

🇺🇸

Jacksonville, Florida, United States

Johns Hopkins All Children's Hospital

🇺🇸

Saint Petersburg, Florida, United States

Connecticut Children's Medical Center

🇺🇸

Hartford, Connecticut, United States

Alfred I DuPont Hospital for Children

🇺🇸

Wilmington, Delaware, United States

Nemours Children's Clinic

🇺🇸

Orlando, Florida, United States

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

🇺🇸

Chapel Hill, North Carolina, United States

Shand's Hospital for Children at the University of Florida

🇺🇸

Gainesville, Florida, United States

University of Kentucky, Markey Cancer Center

🇺🇸

Lexington, Kentucky, United States

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