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Checkpoint Inhibitor

Regorafenib + Nivolumab for Osteosarcoma

Phase 2
Waitlist Available
Research Sponsored by Sarcoma Alliance for Research through Collaboration
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Myelosuppressive chemotherapy: Patients must not have received myelosuppressive chemotherapy within 3 weeks of enrollment
Performance Status: Lansky (≤ 16 years of age) or Karnofsky (>16 years of age) performance score of ≥ 70, or Eastern Cooperative Oncology Group (ECOG) performance score 0 or 1. See Appendix A. Patients who are unable to walk because of paralysis, but who are up and about in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
Must not have
Major surgery (thoracotomy or laparotomy, etc.), laparoscopic biopsy, or significant traumatic injury within 28 days prior to Day 1 of study or who have not recovered adequately from prior surgery
Requirement of oral anticoagulant therapy with oral vitamin K antagonists (warfarin)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up approximately 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new combination therapy for people with a certain type of bone cancer that has returned or does not respond to treatment.

Who is the study for?
This trial is for patients aged 5 and older with high-grade osteosarcoma that's come back or hasn't responded to treatment. They need measurable disease on scans, good heart and lung function, no recent major surgeries or uncontrolled diseases, and must not be pregnant. Participants should have recovered from prior treatments and agree to use birth control.
What is being tested?
The study tests the combination of Regorafenib (a cancer growth blocker) at different doses with Nivolumab (an immune system booster) in those whose osteosarcoma has resisted initial therapy. It aims to see if this combo can shrink tumors better than previous treatments.
What are the potential side effects?
Regorafenib may cause liver issues, fatigue, hand-foot skin reactions, diarrhea, mouth sores; while Nivolumab might lead to immune-related side effects like inflammation in organs such as lungs or intestines, skin rash or hormone gland problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I haven't had chemotherapy that lowers blood cell counts in the last 3 weeks.
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I can take care of myself and am up and about, even if I use a wheelchair.
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My cancer is a high-grade osteosarcoma not linked to Paget's disease or outside the bones.
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My cancer is a high-grade osteosarcoma, not linked to Paget's disease or outside the bones.
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I can swallow pills whole.
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My heart failure is not severe.
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I can take care of myself and do most of my daily activities.
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My osteosarcoma has returned or didn't respond after initial treatment.
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My osteosarcoma has returned or didn't respond after initial treatment.
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My liver function tests are within the required range.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I haven't had major surgery or serious injuries in the last 28 days.
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I am taking warfarin for blood thinning.
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I have had a serious blood clot that needed blood thinners in the last 6 months.
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I have not received a live vaccine in the last 30 days.
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I am taking or need strong medication that affects liver enzymes.
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I have had a bowel perforation or developed a fistula in the past.
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I have been treated with drugs targeting the immune system or blood vessel growth.
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I am not pregnant or nursing.
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I have a severe gut condition causing major diarrhea.
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I have not had severe bleeding in the last 28 days.
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My body surface area is less than 0.4 square meters.
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I have fluid in my chest or abdomen causing significant breathing problems.
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I have had a stem cell or organ transplant in the past.
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I am allergic to ingredients in regorafenib, nivolumab, or similar drugs.
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I regularly take medication that weakens my immune system.
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I have been diagnosed with active HIV.
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My blood pressure is not controlled by medication.
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I have a wound, ulcer, or fracture that is not healing.
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I do not have any infections that are not responding to treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~approximately 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Compare the 4-month progression-free survival rate to historical controls
Secondary study objectives
Describe adverse events
Estimate 1-year and 2-year Overall Survival
Objective Response Rate
+2 more

Side effects data

From 2024 Phase 3 trial • 529 Patients • NCT02017717
80%
Fatigue
70%
Diarrhoea
70%
Headache
40%
Vomiting
40%
Aspartate aminotransferase increased
40%
Rash maculo-papular
40%
Alanine aminotransferase increased
40%
Lipase increased
30%
Partial seizures
30%
Hemiparesis
30%
Gait disturbance
30%
Fall
30%
Cough
30%
Dry skin
30%
Amylase increased
30%
Nausea
30%
Confusional state
20%
Malignant neoplasm progression
20%
Pyrexia
20%
Candida infection
20%
Mucosal infection
20%
Decreased appetite
20%
Back pain
20%
Dysphonia
20%
Hypotension
20%
Colitis
20%
Hyperthyroidism
20%
Oedema peripheral
20%
Muscular weakness
20%
Hypothyroidism
10%
Tinnitus
10%
Cushingoid
10%
Diabetic ketoacidosis
10%
Procedural haemorrhage
10%
Blood bilirubin increased
10%
Bradycardia
10%
Sinus tachycardia
10%
Hyperglycaemia
10%
Hypocalcaemia
10%
Neck pain
10%
Brain oedema
10%
Hydrocephalus
10%
Lethargy
10%
Seizure
10%
Hypertension
10%
Palpitations
10%
Cheilitis
10%
Presyncope
10%
Face oedema
10%
Oedema
10%
Conjunctivitis
10%
Enterocolitis infectious
10%
Oral candidiasis
10%
Pneumonia
10%
Sinusitis
10%
Staphylococcal infection
10%
Blood alkaline phosphatase increased
10%
Spinal pain
10%
Tremor
10%
Dizziness
10%
Dysarthria
10%
Urinary retention
10%
Dyspnoea exertional
10%
Nasal congestion
10%
Pneumonitis
10%
Dermatitis
10%
Erythema
10%
Rash
10%
Klebsiella infection
10%
Hypomagnesaemia
10%
Syncope
10%
Haemorrhage intracranial
10%
Pancreatitis
10%
Cholecystitis
10%
Upper respiratory tract infection
10%
Acute kidney injury
10%
Dermatitis bullous
10%
Lymphopenia
10%
Optic nerve disorder
10%
Visual impairment
10%
Dehydration
10%
Hypokalaemia
10%
Scoliosis
10%
Cognitive disorder
10%
Memory impairment
10%
Hallucination
10%
Insomnia
10%
Irritability
10%
Urinary incontinence
10%
Dyspnoea
10%
Dermatitis acneiform
10%
Pelvic venous thrombosis
10%
Sepsis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 1: Arm N1+I3
Cohort 2: Arm B
Part A Cohort 1c: Arm N3+RT+TMZ
Part A Cohort 1d: Arm N3+RT
Part B Cohort 1c: Arm N3+RT+TMZ
Part B Cohort 1d: Arm N3+RT
Cohort 1: Arm N3
Cohort 1b: Arm N3+I1
Cohort 2: Arm N3

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Regorafenib and NivolumabExperimental Treatment3 Interventions
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Nivolumab
2015
Completed Phase 3
~4010

Find a Location

Who is running the clinical trial?

Sarcoma Alliance for Research through CollaborationLead Sponsor
25 Previous Clinical Trials
1,906 Total Patients Enrolled
3 Trials studying Osteosarcoma
223 Patients Enrolled for Osteosarcoma

Media Library

Nivolumab (Checkpoint Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04803877 — Phase 2
Osteosarcoma Research Study Groups: Regorafenib and Nivolumab
Osteosarcoma Clinical Trial 2023: Nivolumab Highlights & Side Effects. Trial Name: NCT04803877 — Phase 2
Nivolumab (Checkpoint Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04803877 — Phase 2
~11 spots leftby Dec 2025