Cabozantinib for Refractory Osteosarcoma

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
M D Anderson Cancer Center, Houston, TX
Refractory Osteosarcoma+6 More
Cabozantinib - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether atezolizumab and cabozantinib are safe and effective in treating adolescents and young adults with osteosarcoma that has come back (recurrent) or has spread to other places in the body

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Eligible Conditions

  • Refractory Osteosarcoma
  • Metastatic Bone Sarcomas
  • Locally Advanced Osteosarcoma
  • Unresectable Osteosarcoma
  • Recurrent Osteosarcoma

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Cabozantinib will improve 1 primary outcome and 4 secondary outcomes in patients with Refractory Osteosarcoma. Measurement will happen over the course of At 4 and 6 months.

Month 6
PFS
Year 2
Overall survival (OS)
Year 2
Progression-free survival (PFS)
Up to 2 years
Incidence of adverse events
Objective response rate

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

1 Treatment Group

Treatment (atezolizumab, cabozantinib)
1 of 1
Experimental Treatment

This trial requires 40 total participants across 1 different treatment group

This trial involves a single treatment. Cabozantinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Treatment (atezolizumab, cabozantinib)Patients receive atezolizumab IV over 60 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Atezolizumab
FDA approved
Cabozantinib
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years for reporting.

Closest Location

M D Anderson Cancer Center - Houston, TX

Eligibility Criteria

This trial is for patients born any sex of any age. You must have received 1 prior treatment for Refractory Osteosarcoma or one of the other 6 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Histologically confirmed diagnosis of osteosarcoma
Metastatic or unresectable locally advanced disease
Signed informed consent form
Age >= 12 years at time of signing informed consent form
Ability to comply with the study protocol, in the investigator's judgment
Patients must have relapsed or become refractory to conventional therapy including some combination of cisplatin, doxorubicin, methotrexate, and/or ifosfamide
Measurable disease per RECIST version (v)1.1 (Note: Previously irradiated lesions can be considered as measurable disease only if progressive disease has been unequivocally documented at that site since radiation)
Availability of a representative tumor specimen for exploratory biomarker research. Archival samples are permitted if the tumor samples been obtained within 6 months prior to enrollment and the patient has not received intervening therapy
A formalin-fixed paraffin-embedded (FFPE) tumor specimen in a paraffin block (preferred) or at least 15 slides containing unstained, freshly cut, serial sections should be submitted along with an associated pathology report prior to study enrollment. If only 10-14 slides are available, the patient may still be eligible for the study, after principal investigator confirmation has been obtained
If archival tumor tissue is unavailable or is determined to be unsuitable for required testing, tumor tissue must be obtained from a biopsy performed at screening

Patient Q&A Section

What is the average age someone gets osteosarcoma?

"The average age of diagnosis for osteosarcoma varies across different regions and countries. Further studies are needed to determine the most efficient way to diagnose osteosarcoma earlier." - Anonymous Online Contributor

Unverified Answer

How many people get osteosarcoma a year in the United States?

"The incidence rate of osteosarcoma is relatively high despite the rarity of the disease. Most cases occur in children between 6 months and 7 years old. Because osteosarcoma continues to be a very rare disease, most studies concerning the epidemiology of osteosarcoma have been limited. Results from a recent clinical trial highlights the need for further research regarding the incidence of osteosarcoma and its risk factors." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of osteosarcoma?

"In our study we found a higher frequency of patients with Ewing sarcoma and ALL among the non-O+ group compared to the O+ group. This could indicate that as compared to the O+ group the non-O+ group show a better prognosis. The difference between the two groups may be due to the patient's age at diagnosis being different between the O+ and non-O+ groups.\n" - Anonymous Online Contributor

Unverified Answer

How quickly does osteosarcoma spread?

"The majority of the patients had the disease localized at diagnosis. However, locally advanced disease was found in 19% of patients. Most patients presented with localized disease. The poor survival indicates that local control is important. Although the prognosis was dismal, patients with localized disease survived longer than patients with distant metastasis. Local control is as important as adjuvant therapy to achieve long-term survival." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving cabozantinib?

"This is the first study investigating the use of cabozantinib as a second-line treatment in patients with solid tumours. Cabozantinib showed a promising activity profile, but further data from a larger cohort of patients are required to confirm these results." - Anonymous Online Contributor

Unverified Answer

What is the latest research for osteosarcoma?

"As most recent studies tend to be small, they are difficult to compare. Some exhibit a surprising lack of consistency for the treatment of osteosarcoma." - Anonymous Online Contributor

Unverified Answer

Does osteosarcoma run in families?

"Results from a recent paper suggest that genetic factors may have a role in the etiology of osteosarcoma. We hypothesize that genes involved in bone formation and bone remodeling, as well as in cell proliferation and differentiation, may contribute to the pathogenesis of osteosarcoma. Results from a recent paper provides additional evidence for consideration of endocrine mutant gene syndromes and familial cancers in the context of unexplained osteosarcoma." - Anonymous Online Contributor

Unverified Answer

What is the survival rate for osteosarcoma?

"The OSR for adult patients with localized disease is 90%, with a 5-year survival of 93%. Survival is lower in adults than in children. Given the rarity of osteosarcoma, and the fact that there is no standardized treatment protocol for this type of [bone cancer](https://www.withpower.com/clinical-trials/bone-cancer), the OSR depends upon many factors including the specific patient's age, the stage of the disease at the time of diagnosis, and the type of therapy used.\n\nOsteosarcoma was the second most common cancer in boys younger than 15 years old in 2012. Boys begin developing the cancer earlier than girls because they have more active growth hormones that cause their bones to grow faster." - Anonymous Online Contributor

Unverified Answer

Has cabozantinib proven to be more effective than a placebo?

"Cabozantinib monotherapy results in significant improvements in PFS and OS compared with placebo in children and young adults with osteosarcoma. The use of cabozantinib as single agent therapy appears safe in patients with low-grade histology tumors >2 cm and may provide further improvement in outcomes over existing therapies." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating osteosarcoma?

"The current therapeutic advances have not been as rewarding as hoped but do guarantee a better prognosis for patients with osteosarcoma. The cure rate for cartilage tumors remains low. There is no convincing evidence that adjuvant therapies improve survival rates in osteosarcoma. However, additional studies on the use of radiotherapy for osteosarcoma need to be conducted." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of cabozantinib?

"Cabozantinib treatment was well tolerated with most common adverse events related to PDGFR inhibition. The most common grade ≥3 adverse event was fatigue. Further investigation is warranted to determine its safety profile in pediatric patients, particularly those <18 years old." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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