Osteosarcoma Metastatic
Conditions
Keywords
Osteosarcoma, relapsed, progressive, metastatic, lung
Brief summary
Phase Ib/IIa open label safety and efficacy study designed to determine the maximum tolerated dose of inhaled cisplatin liposomal (SLIT cisplatin) administered every 14 days to patients with relapsed/progressive osteosarcoma metastatic to the lung.
Interventions
Sponsors
Insmed Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
13 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Histologically proven, Progressive or recurrent high grade osteosarcoma metastatic to the lung * Patients with histologically proven, fully malignant high-grade osteosarcoma of bone * Measureable pulmonary metastases * Less than grade 3 neuropathies, insignificant decreases in cardiac or auditory function * ECOG performance status of 0, 1 or 2 * FEV1 of 50% or greater of predicted value * FEV1/FVC ratio of 65% or greater * Serum creatinine of ≤ 1.5 mg/dl * Total bilirubin ≤ 1.5mg/dl and SGOT or SGPT \< 2.5 times upper normal limit * ANC of ≥ 1,000/mm3 and platelet count of ≥ 100,000/mm3
Exclusion criteria
* Grade 3 or higher painful neuropathy persisting after a prior platinum containing regimen * Patients who are pregnant or are of childbearing potential and not using methods to avoid pregnancy * Concurrent systemic chemotherapy * Greater than Grade 2 pulmonary toxicity * Pulmonary atelectasis * Reactive airway disease which has resulted in hospitalization within the last year or which requires daily treatment with bronchodilator therapy * Concurrent serious infections * Unstable or serious concurrent medical condition * Recent major surgery or thoracic radiation therapy or chemotherapy * Significant pulmonary fibrosis secondary to prior radiation * Major ventilatory distribution abnormalities * Osteosarcoma secondary to radiation or premalignant conditions * History of prior malignancy * Low grade osteosarcoma, parosteal or periosteal sarcoma
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The Study Medication Was to be Considered Effective if the Population Response Rate Was Found to be Greater Than 20% and Individuals Who Demonstrated a CR or PR or Whose Tumours Demonstrated a Grade 3 or 4 Histologic Response at the Time of Surgery. | 4 to 48 weeks |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Cisplatin Liposomal 24 mg/m2 Inhaled liposomal cisplatin was administered over 1 day in a 14-day treatment cycle by inhalation for a maximum of 6 cycles.
Cisplatin liposomal | 7 |
| Cisplatin Liposomal 36 mg/m2 The study allowed for a dose escalation of liposomal cisplatin to 36 mg/m2 if no adverse events of Grade 3 or higher occurred after at least 3 cycles of drug administration at 24 mg/m2
Cisplatin liposomal | 12 |
| Total | 19 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 |
| Overall Study | Disease Progression | 5 | 9 |
| Overall Study | Physician Decision | 0 | 1 |
Baseline characteristics
| Characteristic | Cisplatin Liposomal 24 mg/m2 | Total | Cisplatin Liposomal 36 mg/m2 |
|---|---|---|---|
| Age, Continuous | 19 years STANDARD_DEVIATION 4.8 | 18.2 years STANDARD_DEVIATION 3.4 | 17.8 years STANDARD_DEVIATION 2.3 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 2 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 3 Participants | 2 Participants |
| Race (NIH/OMB) White | 4 Participants | 13 Participants | 9 Participants |
| Region of Enrollment United States | 7 participants | 19 participants | 12 participants |
| Sex: Female, Male Female | 5 Participants | 7 Participants | 2 Participants |
| Sex: Female, Male Male | 2 Participants | 12 Participants | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 12 |
| other Total, other adverse events | 4 / 7 | 8 / 12 |
| serious Total, serious adverse events | 1 / 7 | 2 / 12 |
Outcome results
Primary
The Study Medication Was to be Considered Effective if the Population Response Rate Was Found to be Greater Than 20% and Individuals Who Demonstrated a CR or PR or Whose Tumours Demonstrated a Grade 3 or 4 Histologic Response at the Time of Surgery.
Time frame: 4 to 48 weeks
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cisplatin Liposomal 24 mg/m2 | The Study Medication Was to be Considered Effective if the Population Response Rate Was Found to be Greater Than 20% and Individuals Who Demonstrated a CR or PR or Whose Tumours Demonstrated a Grade 3 or 4 Histologic Response at the Time of Surgery. | CR/PR | 2 participants |
| Cisplatin Liposomal 24 mg/m2 | The Study Medication Was to be Considered Effective if the Population Response Rate Was Found to be Greater Than 20% and Individuals Who Demonstrated a CR or PR or Whose Tumours Demonstrated a Grade 3 or 4 Histologic Response at the Time of Surgery. | Histological response | 1 participants |
| Cisplatin Liposomal 36 mg/m2 | The Study Medication Was to be Considered Effective if the Population Response Rate Was Found to be Greater Than 20% and Individuals Who Demonstrated a CR or PR or Whose Tumours Demonstrated a Grade 3 or 4 Histologic Response at the Time of Surgery. | CR/PR | 2 participants |
| Cisplatin Liposomal 36 mg/m2 | The Study Medication Was to be Considered Effective if the Population Response Rate Was Found to be Greater Than 20% and Individuals Who Demonstrated a CR or PR or Whose Tumours Demonstrated a Grade 3 or 4 Histologic Response at the Time of Surgery. | Histological response | 1 participants |