Trial Outcomes & Findings for Inhalation SLIT Cisplatin (Liposomal) for the Treatment of Osteosarcoma Metastatic to the Lung (NCT NCT00102531)

NCT ID: NCT00102531

Last Updated: 2017-08-01

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 19 participants
• Primary outcome timeframe: 4 to 48 weeks
• Results posted on: 2017-08-01

Participant Flow

Participant milestones

Measure	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	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
Overall Study STARTED	7	12
Overall Study COMPLETED	2	1
Overall Study NOT COMPLETED	5	11

Reasons for withdrawal

Measure	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	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
Overall Study Adverse Event	0	1
Overall Study Disease Progression	5	9
Overall Study Physician Decision	0	1

Baseline Characteristics

Inhalation SLIT Cisplatin (Liposomal) for the Treatment of Osteosarcoma Metastatic to the Lung

Baseline characteristics by cohort

Measure	Cisplatin Liposomal 24 mg/m2 n=7 Participants Inhaled liposomal cisplatin was administered over 1 day in a 14-day treatment cycle by inhalation for a maximum of 6 cycles. Cisplatin liposomal	Cisplatin Liposomal 36 mg/m2 n=12 Participants 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	Total n=19 Participants Total of all reporting groups
Age, Continuous	19 years STANDARD_DEVIATION 4.8 • n=5 Participants	17.8 years STANDARD_DEVIATION 2.3 • n=7 Participants	18.2 years STANDARD_DEVIATION 3.4 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	10 Participants n=7 Participants	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	9 Participants n=7 Participants	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants	12 participants n=7 Participants	19 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 to 48 weeks

Outcome measures

Measure	Cisplatin Liposomal 24 mg/m2 n=7 Participants Inhaled liposomal cisplatin was administered over 1 day in a 14-day treatment cycle by inhalation	Cisplatin Liposomal 36 mg/m2 n=12 Participants 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
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	2 participants
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	1 participants

Adverse Events

Cisplatin Liposomal 24 mg/m2

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Cisplatin Liposomal 36 mg/m2

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	Cisplatin Liposomal 24 mg/m2 n=7 participants at risk Inhaled liposomal cisplatin was administered over 1 day in a 14-day treatment cycle by inhalation for a maximum of 6 cycles. Cisplatin liposomal	Cisplatin Liposomal 36 mg/m2 n=12 participants at risk 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
Gastrointestinal disorders Vomiting	14.3% 1/7 • AEs were followed until resolution until 30 days post last dose.	0.00% 0/12 • AEs were followed until resolution until 30 days post last dose.
General disorders pyrexia	14.3% 1/7 • AEs were followed until resolution until 30 days post last dose.	0.00% 0/12 • AEs were followed until resolution until 30 days post last dose.
Metabolism and nutrition disorders dehydration	14.3% 1/7 • AEs were followed until resolution until 30 days post last dose.	0.00% 0/12 • AEs were followed until resolution until 30 days post last dose.
Musculoskeletal and connective tissue disorders musculoskeletal chest pain	0.00% 0/7 • AEs were followed until resolution until 30 days post last dose.	16.7% 2/12 • AEs were followed until resolution until 30 days post last dose.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/7 • AEs were followed until resolution until 30 days post last dose.	8.3% 1/12 • AEs were followed until resolution until 30 days post last dose.
Respiratory, thoracic and mediastinal disorders Respiratory distress	0.00% 0/7 • AEs were followed until resolution until 30 days post last dose.	8.3% 1/12 • AEs were followed until resolution until 30 days post last dose.

Other adverse events

Measure	Cisplatin Liposomal 24 mg/m2 n=7 participants at risk Inhaled liposomal cisplatin was administered over 1 day in a 14-day treatment cycle by inhalation for a maximum of 6 cycles. Cisplatin liposomal	Cisplatin Liposomal 36 mg/m2 n=12 participants at risk 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
Gastrointestinal disorders Gastrointestinal Disorders	28.6% 2/7 • AEs were followed until resolution until 30 days post last dose.	33.3% 4/12 • AEs were followed until resolution until 30 days post last dose.
General disorders General Disorders and Admin Site Conditions	14.3% 1/7 • AEs were followed until resolution until 30 days post last dose.	16.7% 2/12 • AEs were followed until resolution until 30 days post last dose.
Reproductive system and breast disorders Respiratory, Thoracic and Mediastinal Disorders	42.9% 3/7 • AEs were followed until resolution until 30 days post last dose.	58.3% 7/12 • AEs were followed until resolution until 30 days post last dose.
Nervous system disorders Nervous System Disorders	14.3% 1/7 • AEs were followed until resolution until 30 days post last dose.	8.3% 1/12 • AEs were followed until resolution until 30 days post last dose.

Additional Information

Margaret Galluzzi

Insmed

Phone: (908) 977-9900

Email: Margaret.Galluzzi@Insmed.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place