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A Phase 2 Study of Amrubicin in Relapsed or Refractory Thymic Malignancies

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ClinicalTrials.gov Identifier: NCT01364727
Recruitment Status : Completed
First Posted : June 2, 2011
Results First Posted : March 14, 2017
Last Update Posted : April 16, 2019
Sponsor:
Collaborator:
Celgene
Information provided by (Responsible Party):
Heather Wakelee, Stanford University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Conditions Thymoma
Thymus Cancer
Thymic Carcinoma
Intervention Drug: Amrubicin
Enrollment 33
Recruitment Details  
Pre-assignment Details

35 participants were fully screened for this study,and all 35 are included in Baseline Characteristics.

However, 2 were ineligible and did not receive treatment, and so are not included in Outcomes nor Adverse Events.

Arm/Group Title Amrubicin
Hide Arm/Group Description Amrubicin 35mg/m2 IV days 1-3 every 3 weeks until progression or toxicity
Period Title: Overall Study
Started 35
Completed 33
Not Completed 2
Reason Not Completed
Not Eligible             2
Arm/Group Title Amrubicin
Hide Arm/Group Description Amrubicin 35mg/m2 IV days 1-3 every 3 weeks until progression or toxicity
Overall Number of Baseline Participants 35
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
<=18 years
0
   0.0%
Between 18 and 65 years
24
  68.6%
>=65 years
11
  31.4%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
Female
14
  40.0%
Male
21
  60.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
Hispanic or Latino
1
   2.9%
Not Hispanic or Latino
34
  97.1%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
American Indian or Alaska Native
0
   0.0%
Asian
10
  28.6%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
   5.7%
White
23
  65.7%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Histology  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
Thymic Carcinoma
20
  57.1%
Thymoma
14
  40.0%
Unknown
1
   2.9%
1.Primary Outcome
Title Overall Response Rate (ORR)
Hide Description

Participants received amrubicin 35 mg/m2 IV days 1 to 3, every 3 weeks, until progression or toxicity.

Tumor response rate was assessed radiographically by the Response Evaluation Criteria In Solid Tumors (RECIST), and the overall response rate (ORR) was expressed as the sum of the Complete Response (CR) rate and the Partial Response (PR) rate.

RECIST criteria define when cancer patients improve ("respond"); stay the same ("stable"); or worsen ("progression") during treatments. The criteria presume that linear measures are an adequate substitute for 2-dimensional (2D) methods and includes 4 response categories:

  • CR = Disappearance of all target lesions
  • PR = 30% decrease in the sum of the longest diameter of target lesions
  • Progressive disease (PD) = 20% increase in the sum of the longest diameter of target lesions
  • Stable disease (SD) = Small changes that do not meet above criteria
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Includes those participants with Complete Response (CR) plus those with Partial Response (PR).
Arm/Group Title Amrubicin
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 33
Measure Type: Count of Participants
Unit of Measure: Participants
Complete Response (CR)
0
   0.0%
Partial Response (PR)
6
  18.2%
Overall response rate (ORR)
6
  18.2%
2.Secondary Outcome
Title Median Progression-free Survival (PFS)
Hide Description Median Progression-free survival in patients with thymic malignancies treated with amrubicin
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Some participants (2) continue to survive without progression, although a median and the 95% confidence interval (95% CI) for the 33 participants have been defined.
Arm/Group Title Amrubicin
Hide Arm/Group Description:
Amrubicin 35mg/m2 IV days 1 to 3 every 3 weeks until progression or toxicity
Overall Number of Participants Analyzed 33
Median (95% Confidence Interval)
Unit of Measure: Months
8.5
(6.7 to 38.0)
3.Secondary Outcome
Title Disease Control Rate (DCR)
Hide Description

Disease control rate (DCR) is the sum of Complete Response (CR) rate + Partial Response (PR) rate + Stable Disease (SD) rate , and is expressed here as the sum of the Overall Response Rate (ORR = CR + PR) plus the Stable Disease (SD) rate, ORR + SD.

Response was assessed by the RECIST criteria, elaborated above.

Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Includes those participants with Complete Response (CR) plus those with Partial Response (PR), plus those with Stable Disease).
Arm/Group Title Amrubicin
Hide Arm/Group Description:
Amrubicin 35mg/m2 IV days 1-3 every 3 weeks until progression or toxicity
Overall Number of Participants Analyzed 33
Measure Type: Count of Participants
Unit of Measure: Participants
Overall Response Rate (ORR)
6
  18.2%
Stable Disease (SD)
23
  69.7%
Disease control rate (DCR)
29
  87.9%
Time Frame 2 Years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Amrubicin
Hide Arm/Group Description Amrubicin 35mg/m2 IV days 1-3 every 3 weeks until progression or toxicity
All-Cause Mortality
Amrubicin
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Amrubicin
Affected / at Risk (%) # Events
Total   16/33 (48.48%)    
Blood and lymphatic system disorders   
Anemia * 1  1/33 (3.03%)  1
Febrile neutropenia * 1  1/33 (3.03%)  5
Thromboembolic event * 1  1/33 (3.03%)  1
Cardiac disorders   
Atrial fibrillation * 1  1/33 (3.03%)  1
Infections and infestations   
Infections and infestations * 1  1/33 (3.03%)  1
Sepsis * 1  2/33 (6.06%)  2
Investigations   
Neutrophil count decreased * 1  3/33 (9.09%)  3
White blood cell decreased * 1  1/33 (3.03%)  1
Nervous system disorders   
Lethargy * 1  1/33 (3.03%)  1
Psychiatric disorders   
Confusion * 1  1/33 (3.03%)  1
Renal and urinary disorders   
Urinary tract infection * 1  1/33 (3.03%)  1
Respiratory, thoracic and mediastinal disorders   
Hypoxia * 1  1/33 (3.03%)  1
Lung Infection * 1  2/33 (6.06%)  2
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Amrubicin
Affected / at Risk (%) # Events
Total   33/33 (100.00%)    
Blood and lymphatic system disorders   
Anemia * 1  32/33 (96.97%)  32
lymph nodes palpable * 1  1/33 (3.03%)  1
Blood bilirubin increased * 1  1/33 (3.03%)  2
Edema * 1 [1]  9/33 (27.27%)  9
Platelet count decreased * 1  12/33 (36.36%)  12
Thromboembolic event * 1  2/33 (6.06%)  2
White blood cell decreased * 1  4/33 (12.12%)  4
Cardiac disorders   
Cardiac disorders * 1 [2]  7/33 (21.21%)  7
Palpitations * 1  3/33 (9.09%)  3
Pericardial effusion * 1  1/33 (3.03%)  1
Sinus tachycardia * 1  4/33 (12.12%)  4
Vascular disorders * 1 [3]  2/33 (6.06%)  2
Ear and labyrinth disorders   
External ear pain * 1  1/33 (3.03%)  1
Endocrine disorders   
Hypocalcemia * 1  1/33 (3.03%)  1
Eye disorders   
Blurred vision * 1  2/33 (6.06%)  2
Dry eye * 1  1/33 (3.03%)  1
eye lacrimation * 1  1/33 (3.03%)  1
Gastrointestinal disorders   
Anorexia * 1  16/33 (48.48%)  16
Conjunctivitis * 1  3/33 (9.09%)  3
Constipation * 1  18/33 (54.55%)  18
Diarrhea * 1  13/33 (39.39%)  13
Dysgeusia * 1  4/33 (12.12%)  4
Gastroesophageal reflux disease * 1  6/33 (18.18%)  6
Gastrointestinal disorder and pain * 1 [4]  10/33 (30.30%)  10
Mucositis oral * 1  29/33 (87.88%)  29
Nausea * 1  20/33 (60.61%)  20
Rectal hemorrhage * 1  2/33 (6.06%)  2
Tooth infection * 1  3/33 (9.09%)  3
Toothache * 1  1/33 (3.03%)  1
Vomiting * 1  7/33 (21.21%)  7
General disorders   
Abdominal pain * 1  5/33 (15.15%)  6
Breast pain * 1  1/33 (3.03%)  1
Buttock pain * 1  2/33 (6.06%)  2
Dizziness * 1  1/33 (3.03%)  1
Fatigue * 1  33/33 (100.00%)  33
Fever * 1  9/33 (27.27%)  9
Night sweats * 1  2/33 (6.06%)  2
Lethargy * 1  1/33 (3.03%)  1
Malaise * 1  2/33 (6.06%)  2
Pain * 1 [5]  25/33 (75.76%)  25
Vertigo * 1  2/33 (6.06%)  2
Weight loss * 1  1/33 (3.03%)  1
Voice alteration * 1  1/33 (3.03%)  1
Immune system disorders   
Allergic reaction * 1  4/33 (12.12%)  4
Infections and infestations   
Anorectal infection * 1  3/33 (9.09%)  3
Bladder infection * 1  1/33 (3.03%)  1
Infections and infestations * 1 [6]  14/33 (42.42%)  14
Mucosal infection * 1  1/33 (3.03%)  1
Investigations   
Alanine aminotransferase increased * 1  5/33 (15.15%)  5
Alkaline phosphatase increased * 1  5/33 (15.15%)  5
Aspartate aminotransferase increased * 1  2/33 (6.06%)  2
Chills * 1  1/33 (3.03%)  1
Ejection fraction decreased * 1  4/33 (12.12%)  4
Investigations * 1 [7]  5/33 (15.15%)  5
Neutrophil count decreased * 1  8/33 (24.24%)  8
Metabolism and nutrition disorders   
Creatinine increased * 1  2/33 (6.06%)  2
Hypoalbuminemia * 1  2/33 (6.06%)  2
Hypokalemia * 1  3/33 (9.09%)  3
Hypomagnesemia * 1  2/33 (6.06%)  2
Hyponatremia * 1  2/33 (6.06%)  2
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  1/33 (3.03%)  1
Arthritis * 1  1/33 (3.03%)  1
Back pain * 1  7/33 (21.21%)  7
Bone pain * 1  3/33 (9.09%)  3
Chest wall pain * 1  1/33 (3.03%)  1
Generalized muscle weakness * 1  1/33 (3.03%)  1
Muscle weakness lower limb * 1  1/33 (3.03%)  1
Musculoskeletal and connective tissue disorder - Other, left chest pain * 1 [8]  5/33 (15.15%)  5
Nervous system disorders   
Headache * 1  6/33 (18.18%)  6
Peripheral motor neuropathy * 1  3/33 (9.09%)  3
Peripheral sensory neuropathy * 1  1/33 (3.03%)  1
Syncope * 1  3/33 (9.09%)  3
Psychiatric disorders   
Anxiety * 1  3/33 (9.09%)  3
Depression * 1  1/33 (3.03%)  1
Insomnia * 1  5/33 (15.15%)  5
Restlessness * 1  1/33 (3.03%)  1
Urinary tract infection and pain * 1  5/33 (15.15%)  5
Renal and urinary disorders   
bladder calcifications * 1  1/33 (3.03%)  1
Urinary frequency * 1  2/33 (6.06%)  2
Reproductive system and breast disorders   
Erectile dysfunction * 1  1/33 (3.03%)  1
right breast gynocomastia * 1  1/33 (3.03%)  1
Respiratory, thoracic and mediastinal disorders   
Cough * 1 [9]  27/33 (81.82%)  27
Dyspnea * 1  19/33 (57.58%)  19
Epistaxis * 1  1/33 (3.03%)  1
Hiccups * 1  2/33 (6.06%)  2
Respiratory, thoracic and mediastinal disorders * 1 [10]  9/33 (27.27%)  9
Sinusitis * 1  3/33 (9.09%)  3
Sleep apnea * 1  1/33 (3.03%)  1
Sore throat * 1  4/33 (12.12%)  4
Upper respiratory infection * 1  12/33 (36.36%)  12
Wheezing * 1  3/33 (9.09%)  3
Skin and subcutaneous tissue disorders   
Alopecia * 1  15/33 (45.45%)  15
Bruising * 1  1/33 (3.03%)  1
Dry mouth * 1  1/33 (3.03%)  1
Dry skin * 1  1/33 (3.03%)  1
Hyperhidrosis * 1  2/33 (6.06%)  2
Nail discoloration * 1  1/33 (3.03%)  1
Palmar-plantar erythrodysesthesia syndrome * 1  5/33 (15.15%)  5
Pruritus * 1  1/33 (3.03%)  1
Rash acneiform * 1  6/33 (18.18%)  6
Rash maculo-papular * 1  4/33 (12.12%)  4
Rash pustular * 1  1/33 (3.03%)  1
Skin and subcutaneous tissue disorders * 1 [11]  6/33 (18.18%)  6
Skin infection * 1  2/33 (6.06%)  2
Skin ulceration * 1  1/33 (3.03%)  1
Surgical and medical procedures   
anal exam * 1  1/33 (3.03%)  1
colonoscopy * 1  1/33 (3.03%)  1
Vascular disorders   
Portal vein thrombosis * 1  1/33 (3.03%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[1]
Including: Face, trunk, neck and limbs
[2]
Including: Bigeminal Rhythm, chest tightness, collateral veins, mitral regurgitation, persistently split S2 and systolic murmur
[3]
Including: Prominent chest veins and Port thrombus
[4]
Including: bloody stool, Epigastric pain, esophageal spasms, perirectal abscess, rectal tenesmus and Thrush
[5]
Including: Neck pain, pain in extremetynon-cardia chest pain, oral pain and pain.
[6]
Including: C.diff infection, diverticulitis, gastroenteritis, Herpes Zoster, sinus infection, Thrush and other.
[7]
Including: Blood CO2 elevated, blue finger, pancytopenia, white blood count increased and other
[8]
including: left chest pain and right rib pain
[9]
Including: productive
[10]
including: chest pruritis, course breath sounds, decreased breath sounds, decreased breath sounds, nasal inflammation, post nasal drip, other.
[11]
Including: blood blister, folliculitis, mall skin lesion or bite and Tenia Versicolor.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Heather A Wakelee, MD
Organization: Stanford University Medical Center
Phone: 650-736-7221
EMail: hwakelee@stanford.edu
Layout table for additonal information
Responsible Party: Heather Wakelee, Stanford University
ClinicalTrials.gov Identifier: NCT01364727     History of Changes
Other Study ID Numbers: IRB-20444
SU-01142011-7369 ( Other Identifier: Stanford University )
THOR0003 ( Other Identifier: OnCore )
First Submitted: May 31, 2011
First Posted: June 2, 2011
Results First Submitted: January 23, 2017
Results First Posted: March 14, 2017
Last Update Posted: April 16, 2019