Clinical trial NCT01763970: Stereotactic Radiation Therapy for Pediatric Sarcomas
Official title: A Phase II Study of Hyperfractionated Stereotactic Radiotherapy in the Treatment of Metastatic Pediatric Sarcomas of Bony Sites
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sarcoma
Metastatic Disease
Bony Sites
Keywords: Stereotactic
Radiotherapy
Hypofractionated
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sponsor class: OTHER
First posted: 2013-01-09
Enrollment: 14
Sex: ALL
Minimum age (years): 4
Maximum age (years): 40
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The stereotactic body radiation therapy (SBRT) literature focuses on clinical outcomes in the adult population. However, SBRT has a particularly strong rationale for application in pediatrics given that high biologically effective doses have been shown to increase control in histologies, such as sarcoma, which are common in the pediatrics population. With stereotactic radiation therapy techniques, a reduction in normal tissue dose surrounding the target lesion of interest may also be accomplished resulting in lower toxicity. Given that pediatric patients with sarcomas, presenting with limited metastases in lung and bone, are still considered to be a curable population with aggressive local therapy, SBRT could have a significant impact on outcomes in oligometastatic patients who may be otherwise unresectable.

Detailed description: Pediatric patients with sarcoma who have limited metastases are still potentially curable with aggressive local therapy. However, conventional moderate dose radiation is unlikely to provide durable local control. Given the recent technologic advances in radiation delivery, it is now possible to deliver tumoricidal doses, using stereotactic radiation over a short time course with highly focal techniques. Stereotactic radiation has proven efficacious in the intracranial setting and in multiple extracranial sites in adults. It has not yet been well studied in the pediatrics population where there is a particularly strong rationale due to the ablative doses that can be delivered to tumor while simultaneously reducing high dose to normal tissues. The proposed trial is a single arm phase II study to determine the efficacy of SBRT in pediatric sarcomas with surgically unresectable metastatic disease. Oligometastatic sites eligible for treatment in this study include bony sites of disease. SBRT will be delivered to each eligible site to a total dose of 4000 delivered in 5 fractions of 800 per fractions each day. Following completion of SBRT, patients will undergo treatment response assessment with the use of diagnostic imaging, clinical examination, and completion of the Brief Pain Inventory to assess quality of life. The primary objective of this study is to determine the efficacy of SBRT delivered to a dose of 4000 centigray (cGy) in 5 fractions of 800 cGy each for patients greater than 3 years of age and \< 40 years of age with metastatic disease of bone secondary to pediatric sarcoma. The secondary objectives of this study include describing the toxicity of SBRT with this regimen; assessing clinical response rate of each target lesion; assessing long-term clinical outcomes; and assessing quality of life following completion of treatment. For patients with potentially curable oligometastatic disease, surgical resection in conjunction with systemic therapy remains the standard of care. Patients on this study will continue to receive chemotherapy outside of the 2 week window for SBRT. Issues that may limit participation include our inability to assess late effects that may not develop till at least 10 years after therapy. For this reason, we will limit the population in this study to patients who are surgically unresectable and would be otherwise incurable with current standard systemic therapies.

Eligibility criteria: Inclusion Criteria:

* histologically or cytologically confirmed metastatic sarcoma of the soft tissue or bone
* must have measurable disease
* disease must be surgically unresectable as determined by a tumor board or surgeon
* greater than 3 years of age
* less than or equal to 40 years of age
* life expectancy of at least 9 months
* adequate performance status (Lansky Performance Status greater than or equal to 50).
* ability to understand and willingness to sign informed consent document

Exclusion Criteria:

* patients who have had chemotherapy or radiotherapy within 2 weeks prior to entering the study
* patients who have had any prior radiotherapy to the treatment site(s)
* patients may not participate on any other treatment protocol while they are receiving treatment on this protocol and for up to 3 months after these protocol treatments have ended
* pregnant women
* refusal of women of child bearing potential to take a pregnancy test prior to treatment

Primary outcomes:
measure: Lesion-specific Local Control at 6 Months Post-SBRT as Assessed by Percentage of Lesions Locally Controlled
timeFrame: 6 months post-SBRT
Secondary outcomes:
measure: Patient-specific Local Control at 6 Months Post-SBRT as Assessed by the Percentage of Patients Locally Controlled
timeFrame: 6 months post-SBRT
measure: Percentage of Patients With Progression-free Survival at 6 Months Post-SBRT
timeFrame: 6 months post-SBRT
measure: Percentage of Patients With Overall Survival at 6 Months Post-SBRT
timeFrame: 6 months post-SBRT
measure: Change in Quality of Life (QoL) as Assessed by the Brief Pain Inventory
timeFrame: Baseline and one-month post-SBRT
measure: Number of Participants Experiencing Toxicity of SBRT
timeFrame: 12 months after treatment starts
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford Medical Center
state: California
city: Washington
country: United States
facility: Sibley Memorial Hospital
state: District of Columbia
city: Baltimore
country: United States
facility: The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
state: Maryland
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
name: Matthew Ladra, M.D.
role: PRINCIPAL_INVESTIGATOR
References: