Trial Outcomes & Findings for Amrubicin Chemotherapy as First Line in Metastatic or Unresectable Soft Tissue Sarcoma (NCT NCT01259375)

Last Updated: 2017-04-17

Results Overview

Per response evaluation criteria in solid tumors criteria (RECIST 1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR) \>= 30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

48 months

Results posted on

2017-04-17

Participant Flow

Participant milestones

Participant milestones
Measure	All Patients All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Overall Study STARTED	24
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Overall Study Adverse Event	1

Baseline Characteristics

Amrubicin Chemotherapy as First Line in Metastatic or Unresectable Soft Tissue Sarcoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Groups n=24 Participants All patients that received treatment.
Age, Continuous	52 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Region of Enrollment United States	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 48 months

Population: One patient discontinued study treatment after the first cycle due to side effects and was not evaluable for response.

Outcome measures

Outcome measures
Measure	All Patients n=23 Participants All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Response Rate for Amrubicin in Patients With Metastatic or Advanced Sarcoma as First Line Therapy.	13 percentage of pt with a partial response Interval 2.8 to 33.6

PRIMARY outcome

Timeframe: six months

Per Response Evaluation Criteria in Solid Tumors Criteria (RECIST v 1.1) for target lesions and assessed by CT; time elapsed between treatment initiation and tumor progression or death. In target lesions, progression is defined per Response Evaluation Criteria in Solid Tumors Criteria (RECIST 1.1) as at least a 20% increase in the sum of the LD of target lesions, taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. In non-target lesions, progression is defined via RECIST v 1.1 as the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.

Outcome measures

Outcome measures
Measure	All Patients n=23 Participants All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Progression Free Survival	42.7 percentage of pt with 6 mnth PFS Interval 24.0 to 76.3

SECONDARY outcome

Timeframe: 4 months

Population: One patient discontinued study treatment after the first cycle due to side effects and was not evaluable for response, but was evaluable for safety and toxicity.

Outcome measures

Outcome measures
Measure	All Patients n=24 Participants All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Number of Participants With Serious Adverse Events	7 participants who experienced an SAE

SECONDARY outcome

Timeframe: two years

This variable reviews the percentage of Overall Survival at 24 months and presents the percentage of participants who survived at 2 years.

Outcome measures

Outcome measures
Measure	All Patients n=23 Participants All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Overall Survival	52.8 percentage of participants Interval 35.9 to 77.6

SECONDARY outcome

Timeframe: 48 months

Population: This exploratory objective was not assessed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 48 months

Outcome measures

Outcome measures
Measure	All Patients n=3 Participants All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Differential Response to Amurbicin Among Certain Histologic Subtypes of Sarcoma. liposarcoma	1 participants
Differential Response to Amurbicin Among Certain Histologic Subtypes of Sarcoma. myxoid liposarcoma with TLS-CHOP translocation	1 participants
Differential Response to Amurbicin Among Certain Histologic Subtypes of Sarcoma. leiomyoscarcoma	1 participants

Adverse Events

All Patients

Serious events: 7 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=24 participants at risk All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Myelodysplastic syndrome	4.2% 1/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders Pneumothorax	4.2% 1/24 • Through study treatment, an average of 4 months.
Blood and lymphatic system disorders febrile neutropenia	16.7% 4/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders back pain	4.2% 1/24 • Through study treatment, an average of 4 months.
Infections and infestations e. coli positive	4.2% 1/24 • Through study treatment, an average of 4 months.

Other adverse events

Other adverse events
Measure	All Patients n=24 participants at risk All participants enrolled. Amrubicin: Synthetic 9-aminoanthracycline Patients will receive 40mg/m2/day intravenously
Gastrointestinal disorders abdominal discomfort	8.3% 2/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders abdominal pain	8.3% 2/24 • Through study treatment, an average of 4 months.
Psychiatric disorders Agitation	8.3% 2/24 • Through study treatment, an average of 4 months.
Skin and subcutaneous tissue disorders alopecia	87.5% 21/24 • Through study treatment, an average of 4 months.
Blood and lymphatic system disorders anemia	25.0% 6/24 • Through study treatment, an average of 4 months.
Metabolism and nutrition disorders anorexia	33.3% 8/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders back pain	12.5% 3/24 • Through study treatment, an average of 4 months.
Eye disorders blurred vision	8.3% 2/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders body aches	8.3% 2/24 • Through study treatment, an average of 4 months.
General disorders chills	8.3% 2/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders congestion	12.5% 3/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders constipation	16.7% 4/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders cough	45.8% 11/24 • Through study treatment, an average of 4 months.
Psychiatric disorders despression	8.3% 2/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders diarrhea	16.7% 4/24 • Through study treatment, an average of 4 months.
Eye disorders dry eyes	8.3% 2/24 • Through study treatment, an average of 4 months.
Skin and subcutaneous tissue disorders dry skin	8.3% 2/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders dysgeusia	20.8% 5/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders dyspenia	20.8% 5/24 • Through study treatment, an average of 4 months.
General disorders edema	16.7% 4/24 • Through study treatment, an average of 4 months.
General disorders fatigue	75.0% 18/24 • Through study treatment, an average of 4 months.
General disorders fever	20.8% 5/24 • Through study treatment, an average of 4 months.
Nervous system disorders headaches	25.0% 6/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders hiccups	8.3% 2/24 • Through study treatment, an average of 4 months.
Metabolism and nutrition disorders hyperglycemia	8.3% 2/24 • Through study treatment, an average of 4 months.
Cardiac disorders hypertension	8.3% 2/24 • Through study treatment, an average of 4 months.
Metabolism and nutrition disorders hypoalbuminemia	8.3% 2/24 • Through study treatment, an average of 4 months.
Psychiatric disorders insomnia	20.8% 5/24 • Through study treatment, an average of 4 months.
Blood and lymphatic system disorders neutropenia	20.8% 5/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders oral thrush	8.3% 2/24 • Through study treatment, an average of 4 months.
Blood and lymphatic system disorders thrombocytopenia	12.5% 3/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders pleural effusion	8.3% 2/24 • Through study treatment, an average of 4 months.
Skin and subcutaneous tissue disorders rash	12.5% 3/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders reflux	29.2% 7/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders Rhinorrhea	16.7% 4/24 • Through study treatment, an average of 4 months.
Cardiac disorders Sinus bradycardia	16.7% 4/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders Sinusitis	8.3% 2/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders Sore Throat	16.7% 4/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders vomiting	20.8% 5/24 • Through study treatment, an average of 4 months.
Investigations weight loss	8.3% 2/24 • Through study treatment, an average of 4 months.
Investigations white blood cell decreased	20.8% 5/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders decreased joint function	8.3% 2/24 • Through study treatment, an average of 4 months.
General disorders malaise	8.3% 2/24 • Through study treatment, an average of 4 months.
Respiratory, thoracic and mediastinal disorders mucositis	20.8% 5/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders myalgia	8.3% 2/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders nausea	70.8% 17/24 • Through study treatment, an average of 4 months.
Gastrointestinal disorders mouth sores	8.3% 2/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders hip pain	8.3% 2/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders lower extremity pain	12.5% 3/24 • Through study treatment, an average of 4 months.
Cardiac disorders tachycardia	12.5% 3/24 • Through study treatment, an average of 4 months.
General disorders gait disturbance	8.3% 2/24 • Through study treatment, an average of 4 months.
Musculoskeletal and connective tissue disorders generalized muscle weakness	8.3% 2/24 • Through study treatment, an average of 4 months.

Additional Information

Dr. Sunil Sharma

Huntsman Cancer Institute

Phone: 801-587-5559

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place