Study of RNA-lipid Particle (RNA-LP) Vaccines for Recurrent Pulmonary Osteosarcoma (OSA)


Study First Submitted Date 2022-12-13
Study First Posted Date 2022-12-21
Last Update Posted Date 2023-01-27
Start Month Year October 2023
Primary Completion Month Year October 2026
Verification Month Year January 2023
Verification Date 2023-01-31
Last Update Posted Date 2023-01-27


Sequence: 52502201
Name Pulmonary Osteosarcoma
Downcase Name pulmonary osteosarcoma

Id Information

Sequence: 40395391
Id Source secondary_id
Id Value OCR43060
Id Type Other Identifier
Id Type Description University of Florida

Design Groups

Sequence: 55958431
Group Type Experimental
Title RNA-LP vaccine


Sequence: 52810313
Intervention Type Biological
Name RNA-LP vaccine
Description All subjects will receive two doses of RNA-LP vaccine every two weeks and then once monthly for 12 months afterward. Subjects on the phase 1 portion of the study will be assigned to receive either 0.01 mg/kg mRNA encapsulated in 0.15 mg/kg LPs (dose level -1), 0.02 mg/kg mRNA encapsulated in 0.3 mg/kg LPs (dose level 0), 0.04 mg mRNA encapsulated in 0.6 mg/kg LPs (dose level +1), 0.08 mg mRNA encapsulated in 1.2 mg/kg LPs (dose level +2) following a 3 + 3 design. All subjects on the Phase II portion will receive the maximum tolerated dose determined in the phase I portion of the study.


Sequence: 80317831 Sequence: 80317832 Sequence: 80317833 Sequence: 80317834
Name pulmonary osteosarcoma Name RNA-LP Name vaccines Name immunotherapy
Downcase Name pulmonary osteosarcoma Downcase Name rna-lp Downcase Name vaccines Downcase Name immunotherapy

Design Outcomes

Sequence: 178614959 Sequence: 178614960 Sequence: 178614961 Sequence: 178614962
Outcome Type primary Outcome Type primary Outcome Type secondary Outcome Type secondary
Measure Maximum tolerated dose Measure 12 month event-free survival Measure Event-free survival Measure Overall survival
Time Frame 14 months Time Frame 12 months Time Frame 3 years Time Frame 3 years
Description Determine the maximum tolerated dose of RNA-LP vaccine. Description Determine if RN-LP vaccine therapy extends 12 month event-free survival. Event-free survival is defined as the time from start of treatment to either disease progression or death, whichever occurs first. Description Determine the event-free survival in patients with recurrent pulmonary oseteosarcoma receiving RNA-LP vaccine. Event-free survival is defined as the time from start of treatment to either disease progression or death, whichever occurs first. Description Determine the overall survival in patients with recurrent pulmonary oseteosarcoma receiving RNA-LP vaccine.

Browse Conditions

Sequence: 194749681 Sequence: 194749682 Sequence: 194749683 Sequence: 194749684 Sequence: 194749685 Sequence: 194749686 Sequence: 194749687
Mesh Term Osteosarcoma Mesh Term Neoplasms, Bone Tissue Mesh Term Neoplasms, Connective Tissue Mesh Term Neoplasms, Connective and Soft Tissue Mesh Term Neoplasms by Histologic Type Mesh Term Neoplasms Mesh Term Sarcoma
Downcase Mesh Term osteosarcoma Downcase Mesh Term neoplasms, bone tissue Downcase Mesh Term neoplasms, connective tissue Downcase Mesh Term neoplasms, connective and soft tissue Downcase Mesh Term neoplasms by histologic type Downcase Mesh Term neoplasms Downcase Mesh Term sarcoma
Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48625948 Sequence: 48625949
Agency Class OTHER Agency Class OTHER
Lead Or Collaborator lead Lead Or Collaborator collaborator
Name University of Florida Name The V Foundation for Cancer Research

Overall Officials

Sequence: 29458088
Role Principal Investigator
Name John Ligon, MD
Affiliation University of Florida

Central Contacts

Sequence: 12094266
Contact Type primary
Name Shannon Alford, MPH
Phone (352) 273-8146
Role Contact

Design Group Interventions

Sequence: 68600064
Design Group Id 55958431
Intervention Id 52810313


Sequence: 30954203
Gender All
Minimum Age 0 Years
Maximum Age 39 Years
Healthy Volunteers No
Criteria Inclusion Criteria: Age < 39 years. Histopathologically proven recurrent pulmonary osteosarcoma. Must be eligible for thoracotomy (Arms 1 and 2 only). Patient must have been enrolled on a screening consent and have had sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs). Patients must have recovered from the effects of surgery, biopsy, postoperative infection, and other complications. Karnofsky Performance Status (KPS) or Lansky Performance Status (LPS) ≥ 60. Bone Marrow: ANC (Absolute neutrophil count) ≥ 1,000µl (absent treatment) Platelets ≥ 100,000/µl (absent treatment for at least 3 days) Hemoglobin > 8 g/dL Renal: BUN ≤ 25 mg/dl Creatinine ≤ 1.7 mg/dl Hepatic Bilirubin ≤ 2.0 mg/dl ALT ≤ 5 times institutional upper limits of normal for age AST ≤ 5 times institutional upper limits of normal for age Signed informed consent. If the patient's mental status precludes his/her giving informed consent, written informed consent may be given by the legally authorized representative per institutional policy. For subjects of childbearing potential (SOCBP), negative urine or serum pregnancy test at enrollment. SOCBP must be willing to use acceptable contraceptive methods to avoid pregnancy throughout the study and for at least 24 weeks after the last dose of study drug. Subjects with partners of childbearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 24 weeks following the last dose of study drug. Exclusion Criteria: HIV, Hepatitis B, CMV, or Hepatitis C seropositive. Known active infection or immunosuppressive disease. Known severe, active co-morbidity, defined as follows: Unstable angina and/or congestive heart failure requiring hospitalization. Unstable cardiac arrhythmias, abnormalities, or transmural myocardial infarction within the last 6 months. Acute bacterial or fungal infection requiring intravenous antibiotics Chronic Obstructive Pulmonary Disease exacerbation or other known respiratory illness requiring hospitalization or precluding study therapy Hepatic insufficiency resulting in clinical jaundice and/or coagulation defects. Acquired Immune Deficiency Syndrome (AIDS) based upon current CDC definition. The need to exclude patients with AIDS from this protocol is necessary because the treatments involved in this protocol may be significantly immunosuppressive. Patients with autoimmune disease requiring medical management with immunosuppressants. Major medical illnesses or psychiatric impairments that, in the investigator's opinion, will prevent administration or completion of protocol therapy. Known active connective tissue disorders such as lupus or scleroderma that, in the investigator's opinion, place the patient at high risk for radiation toxicity. Pregnancy or subjects of childbearing potential and their partners who are sexually active and who are unwilling or unable to use an acceptable method of contraception for the entire study period; this exclusion is necessary because the treatment involved in this study may be significantly teratogenic. Subjects must not be pregnant or breast-feeding. Subjects who require systemic corticosteroids above physiologic doses or not weaned to physiologic dosing within 1 week of scheduled vaccination. Subjects who are receiving any other investigational agents or who have been treated on any other therapeutic clinical protocols within 30 days prior to study entry. Subjects who are unwilling or unable to receive treatment and undergo follow-up evaluations at the enrolled Sunshine Consortium treatment site.
Adult True
Child True
Older Adult False

Calculated Values

Sequence: 253883387
Registered In Calendar Year 2022
Were Results Reported False
Has Single Facility False
Minimum Age Num 0
Maximum Age Num 39
Minimum Age Unit Years
Maximum Age Unit Years
Number Of Primary Outcomes To Measure 2
Number Of Secondary Outcomes To Measure 2


Sequence: 30699783
Allocation N/A
Intervention Model Sequential Assignment
Observational Model
Primary Purpose Treatment
Time Perspective
Masking None (Open Label)

Responsible Parties

Sequence: 29066550
Responsible Party Type Sponsor