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Safety and Efficacy of Fidaxomicin Versus Placebo for Prophylaxis Against Clostridium Difficile-Associated Diarrhea in Adults Undergoing Hematopoietic Stem Cell Transplantation (MK-5119-001) (DEFLECT-1)

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ClinicalTrials.gov Identifier: NCT01691248
Recruitment Status : Completed
First Posted : September 24, 2012
Results First Posted : April 12, 2016
Last Update Posted : September 18, 2018
Sponsor:
Information provided by (Responsible Party):
Optimer Pharmaceuticals LLC

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Prevention
Condition Clostridium Difficile-Associated Diarrhea (CDAD)
Interventions Drug: fidaxomicin
Drug: Placebo
Enrollment 611
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Fidaxomicin Placebo
Hide Arm/Group Description 200 mg Fidaxomicin tablet once daily for no longer than 40 days Placebo tablet once daily for no longer than 40 days
Period Title: Overall Study
Started 305 [1] 306 [1]
Treated 301 [2] 299 [2]
Safety Analysis Set 300 [3] 300 [3]
Completed 194 192
Not Completed 111 114
Reason Not Completed
Protocol Violation             43             27
Adverse Event             24             22
Withdrawal by Subject             20             18
Confirmed CDAD             14             31
Lost to Follow-up             2             5
Reason not provided             4             4
Not Treated             4             7
[1]
Randomized
[2]
Received at least 1 dose of study drug
[3]
A participant randomized to Fidaxomicin, received placebo instead.
Arm/Group Title Fidaxomicin Placebo Total
Hide Arm/Group Description 200 mg Fidaxomicin tablet once daily for no longer than 40 days Placebo tablet once daily for no longer than 40 days Total of all reporting groups
Overall Number of Baseline Participants 301 299 600
Hide Baseline Analysis Population Description
Modified Intent-to-Treat (mITT) consisting of all randomized participants undergoing hematopoietic stem cell transplantation (HSCT) who received at least 1 dose of study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 301 participants 299 participants 600 participants
55.1  (12.00) 55.1  (13.23) 55.1  (12.62)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 301 participants 299 participants 600 participants
Female
125
  41.5%
103
  34.4%
228
  38.0%
Male
176
  58.5%
196
  65.6%
372
  62.0%
1.Primary Outcome
Title Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to 30 Days Post-treatment Follow-up.
Hide Description CDAD is defined as follows: Diarrhea: (change in bowel habits with >3 unformed bowel movements in a 24 hour period) and the presence of either toxin A and/or B (or their respective genes, tcdA and/or tcdB) of C. difficile in the stool determined by C. difficile toxin assay. Wald 95% Confidence Intervals (CI) are presented.
Time Frame Up to 30 days post-treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
mITT consisting of all randomized participants undergoing HSCT who received at least 1 dose of study drug.
Arm/Group Title Fidaxomicin Placebo
Hide Arm/Group Description:
200 mg Fidaxomicin tablet once daily for no longer than 40 days
Placebo tablet once daily for no longer than 40 days
Overall Number of Participants Analyzed 301 299
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
28.6
(23.5 to 33.7)
30.8
(25.5 to 36.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2778
Comments 1-sided Wald test for a difference in proportions using an unpooled estimate of variance.
Method One-sided Wald p-value
Comments [Not Specified]
Method of Estimation Estimation Parameter Percentage Difference
Estimated Value 2.2
Confidence Interval (2-Sided) 95%
-5.1 to 9.5
Estimation Comments Placebo minus Fidaxomicin
2.Secondary Outcome
Title Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to 60 Days Post-treatment.
Hide Description CDAD is defined as follows: Diarrhea: (change in bowel habits with >3 unformed bowel movements in a 24 hour period) and the presence of either toxin A and/or B (or their respective genes, tcdA and/or tcdB) of C. difficile in the stool determined by C. difficile toxin assay. Wald 95% Confidence Intervals (CI) are presented.
Time Frame Up to 60 days post-treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
mITT consisting of all randomized participants undergoing HSCT who received at least 1 dose of study drug.
Arm/Group Title Fidaxomicin Placebo
Hide Arm/Group Description:
200 mg Fidaxomicin tablet once daily for no longer than 40 days
Placebo tablet once daily for no longer than 40 days
Overall Number of Participants Analyzed 301 299
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
35.2
(29.8 to 40.6)
35.8
(30.4 to 41.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4420
Comments 1-sided Wald test for a difference in proportions using an unpooled estimate of variance.
Method One-sided Wald p-value
Comments [Not Specified]
Method of Estimation Estimation Parameter Percentage difference
Estimated Value 0.6
Confidence Interval (2-Sided) 95%
-7.1 to 8.2
Estimation Comments Placebo minus Fidaxomicin
3.Secondary Outcome
Title Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to Day 70 of Study.
Hide Description CDAD is defined as follows: Diarrhea: (change in bowel habits with >3 unformed bowel movements in a 24 hour period) and the presence of either toxin A and/or B (or their respective genes, tcdA and/or tcdB) of C. difficile in the stool determined by C. difficile toxin assay. Wald 95% Confidence Intervals (CI) are presented.
Time Frame Up to Day 70 of study
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
mITT consisting of all randomized participants undergoing HSCT who received at least 1 dose of study drug.
Arm/Group Title Fidaxomicin Placebo
Hide Arm/Group Description:
200 mg Fidaxomicin tablet once daily for no longer than 40 days
Placebo tablet once daily for no longer than 40 days
Overall Number of Participants Analyzed 301 299
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
29.2
(24.1 to 34.4)
31.1
(25.9 to 36.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3091
Comments 1-sided Wald test for a difference in proportions using an unpooled estimate of variance.
Method One-sided Wald p-value
Comments [Not Specified]
Method of Estimation Estimation Parameter Percentage difference
Estimated Value 1.9
Confidence Interval (2-Sided) 95%
-5.5 to 9.2
Estimation Comments Placebo minus Fidaxomicin
Time Frame Non-serious adverse events (AEs) up to 32 days post treatment. Serious AEs (SAEs) up to 62 days post treatment.
Adverse Event Reporting Description Participants who received at least one dose of study drug. One participant randomized to Fidaxomicin, received placebo instead.
 
Arm/Group Title Fidaxomicin Placebo
Hide Arm/Group Description 200 mg Fidaxomicin tablet once daily for no longer than 40 days Placebo tablet once daily for no longer than 40 days
All-Cause Mortality
Fidaxomicin Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Fidaxomicin Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   98/300 (32.67%)      92/300 (30.67%)    
Blood and lymphatic system disorders     
Anaemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Febrile neutropenia  1  17/300 (5.67%)  18 14/300 (4.67%)  14
Pancytopenia  1  1/300 (0.33%)  1 2/300 (0.67%)  2
Splenic haemorrhage  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Cardiac disorders     
Atrial fibrillation  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Cardiac arrest  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Cardiac failure acute  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Cardiac failure congestive  1  2/300 (0.67%)  2 1/300 (0.33%)  3
Pericarditis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Sinus tachycardia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Eye disorders     
Blindness  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Gastrointestinal disorders     
Abdominal distension  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Abdominal pain  1  1/300 (0.33%)  1 2/300 (0.67%)  3
Ascites  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Diarrhoea  1  5/300 (1.67%)  5 2/300 (0.67%)  2
Enterocolitis  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Gastrointestinal haemorrhage  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Gastrooesophageal reflux disease  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Haematochezia  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Nausea  1  5/300 (1.67%)  5 2/300 (0.67%)  2
Neutropenic colitis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Oesophagitis  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Stomatitis  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Vomiting  1  7/300 (2.33%)  7 2/300 (0.67%)  2
General disorders     
Adverse drug reaction  1  2/300 (0.67%)  2 0/300 (0.00%)  0
Asthenia  1  2/300 (0.67%)  2 0/300 (0.00%)  0
Chest pain  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Chills  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Inflammation  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Mucosal inflammation  1  0/300 (0.00%)  0 3/300 (1.00%)  3
Multi-organ failure  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Oedema peripheral  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pyrexia  1  5/300 (1.67%)  5 10/300 (3.33%)  10
Systemic inflammatory response syndrome  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Hepatobiliary disorders     
Cholecystitis acute  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Hepatic failure  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Hepatic function abnormal  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Portal hypertension  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Venoocclusive liver disease  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Immune system disorders     
Acute graft versus host disease  1  3/300 (1.00%)  3 1/300 (0.33%)  1
Acute graft versus host disease in intestine  1  0/300 (0.00%)  0 2/300 (0.67%)  2
Acute graft versus host disease in skin  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Engraftment syndrome  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Graft versus host disease  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Graft versus host disease in intestine  1  2/300 (0.67%)  2 3/300 (1.00%)  3
Graft versus host disease in lung  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Graft versus host disease in skin  1  2/300 (0.67%)  2 1/300 (0.33%)  1
Infections and infestations     
Appendicitis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Aspergillosis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
BK virus infection  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Bacteraemia  1  3/300 (1.00%)  3 2/300 (0.67%)  3
Bacterial infection  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Bronchopneumonia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Candidiasis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Cellulitis  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Cellulitis gangrenous  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Cholecystitis infective  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Clostridium difficile colitis  1  1/300 (0.33%)  1 2/300 (0.67%)  2
Cystitis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Cytomegalovirus infection  1  4/300 (1.33%)  4 2/300 (0.67%)  2
Cytomegalovirus viraemia  1  2/300 (0.67%)  2 1/300 (0.33%)  1
Device related infection  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Diverticulitis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Enterobacter bacteraemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Enterobacter sepsis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Enterococcal infection  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Enterococcal sepsis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Escherichia bacteraemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Escherichia infection  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Escherichia sepsis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Gastroenteritis norovirus  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Gastroenteritis viral  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Human herpesvirus 6 infection  1  0/300 (0.00%)  0 2/300 (0.67%)  2
Metapneumovirus infection  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Pneumonia  1  4/300 (1.33%)  4 3/300 (1.00%)  3
Pneumonia bacterial  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Pneumonia cytomegaloviral  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pneumonia fungal  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pseudomonal bacteraemia  1  2/300 (0.67%)  2 1/300 (0.33%)  1
Pyelonephritis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Sepsis  1  3/300 (1.00%)  3 4/300 (1.33%)  4
Septic shock  1  1/300 (0.33%)  1 5/300 (1.67%)  5
Staphylococcal bacteraemia  1  0/300 (0.00%)  0 4/300 (1.33%)  4
Staphylococcal infection  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Systemic candida  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Urinary tract infection enterococcal  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Viraemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Viral haemorrhagic cystitis  1  2/300 (0.67%)  2 0/300 (0.00%)  0
Injury, poisoning and procedural complications     
Accidental overdose  1  3/300 (1.00%)  3 0/300 (0.00%)  0
Craniocerebral injury  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Drug dispensing error  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Fall  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Medication error  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Transfusion reaction  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Transplant failure  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Investigations     
Alanine aminotransferase increased  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Aspartate aminotransferase increased  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Blood albumin decreased  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Liver function test abnormal  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Metabolism and nutrition disorders     
Alkalosis  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Decreased appetite  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Dehydration  1  2/300 (0.67%)  2 0/300 (0.00%)  0
Failure to thrive  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Fluid imbalance  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Hyperkalaemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Hypokalaemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Hypovolaemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Tumour lysis syndrome  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Musculoskeletal and connective tissue disorders     
Back pain  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Chondrocalcinosis pyrophosphate  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pathological fracture  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Rhabdomyolysis  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Acute lymphocytic leukaemia recurrent  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Acute myeloid leukaemia recurrent  1  2/300 (0.67%)  2 1/300 (0.33%)  1
Adenocarcinoma pancreas  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Cancer pain  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Central nervous system lymphoma  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Diffuse large B-cell lymphoma recurrent  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Leukaemia  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Leukaemia recurrent  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Lymphoma  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Myelodysplastic syndrome  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Non-Hodgkin's lymphoma  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Non-Hodgkin's lymphoma recurrent  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Peripheral T-cell lymphoma unspecified recurrent  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Post transplant lymphoproliferative disorder  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Nervous system disorders     
Autonomic nervous system imbalance  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Autonomic neuropathy  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Cerebrovascular accident  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Encephalopathy  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Haemorrhage intracranial  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Headache  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Hypoaesthesia  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Leukoencephalopathy  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Posterior reversible encephalopathy syndrome  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Subarachnoid haemorrhage  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Syncope  1  2/300 (0.67%)  2 1/300 (0.33%)  1
Psychiatric disorders     
Anxiety  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Delirium  1  2/300 (0.67%)  2 0/300 (0.00%)  0
Mental status changes  1  2/300 (0.67%)  2 2/300 (0.67%)  2
Renal and urinary disorders     
Haematuria  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Renal failure  1  0/300 (0.00%)  0 2/300 (0.67%)  2
Renal failure acute  1  8/300 (2.67%)  9 1/300 (0.33%)  1
Renal failure chronic  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Renal impairment  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Respiratory, thoracic and mediastinal disorders     
Acute respiratory distress syndrome  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Acute respiratory failure  1  0/300 (0.00%)  0 3/300 (1.00%)  6
Dyspnoea  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Hypoxia  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Organising pneumonia  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pleural effusion  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pulmonary alveolar haemorrhage  1  1/300 (0.33%)  1 2/300 (0.67%)  2
Pulmonary embolism  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Pulmonary oedema  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Respiratory distress  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Respiratory failure  1  2/300 (0.67%)  2 1/300 (0.33%)  1
Skin and subcutaneous tissue disorders     
Purpura  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Rash  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Vascular disorders     
Deep vein thrombosis  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Haemodynamic instability  1  0/300 (0.00%)  0 1/300 (0.33%)  1
Hypotension  1  1/300 (0.33%)  1 1/300 (0.33%)  1
Jugular vein thrombosis  1  2/300 (0.67%)  2 0/300 (0.00%)  0
Shock  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Venoocclusive disease  1  1/300 (0.33%)  1 0/300 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Fidaxomicin Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   297/300 (99.00%)      298/300 (99.33%)    
Blood and lymphatic system disorders     
Anaemia  1  40/300 (13.33%)  63 36/300 (12.00%)  52
Febrile neutropenia  1  128/300 (42.67%)  131 98/300 (32.67%)  107
Neutropenia  1  31/300 (10.33%)  63 40/300 (13.33%)  65
Thrombocytopenia  1  54/300 (18.00%)  77 46/300 (15.33%)  77
Cardiac disorders     
Sinus tachycardia  1  13/300 (4.33%)  14 16/300 (5.33%)  16
Tachycardia  1  30/300 (10.00%)  39 43/300 (14.33%)  59
Gastrointestinal disorders     
Abdominal distension  1  16/300 (5.33%)  17 12/300 (4.00%)  14
Abdominal pain  1  62/300 (20.67%)  71 63/300 (21.00%)  83
Abdominal pain upper  1  20/300 (6.67%)  20 16/300 (5.33%)  24
Constipation  1  55/300 (18.33%)  59 88/300 (29.33%)  107
Diarrhoea  1  211/300 (70.33%)  286 220/300 (73.33%)  340
Dry mouth  1  22/300 (7.33%)  24 32/300 (10.67%)  34
Dyspepsia  1  44/300 (14.67%)  47 33/300 (11.00%)  35
Gastrooesophageal reflux disease  1  15/300 (5.00%)  15 18/300 (6.00%)  19
Haemorrhoids  1  18/300 (6.00%)  18 26/300 (8.67%)  27
Nausea  1  184/300 (61.33%)  252 201/300 (67.00%)  284
Oesophagitis  1  29/300 (9.67%)  29 24/300 (8.00%)  25
Stomatitis  1  68/300 (22.67%)  89 75/300 (25.00%)  103
Vomiting  1  119/300 (39.67%)  174 123/300 (41.00%)  172
General disorders     
Asthenia  1  22/300 (7.33%)  31 38/300 (12.67%)  47
Chills  1  28/300 (9.33%)  33 31/300 (10.33%)  39
Fatigue  1  117/300 (39.00%)  138 118/300 (39.33%)  149
Mucosal inflammation  1  107/300 (35.67%)  128 106/300 (35.33%)  134
Oedema peripheral  1  78/300 (26.00%)  99 63/300 (21.00%)  74
Pyrexia  1  69/300 (23.00%)  83 91/300 (30.33%)  103
Infections and infestations     
Oral candidiasis  1  17/300 (5.67%)  17 15/300 (5.00%)  15
Investigations     
Aspartate aminotransferase increased  1  12/300 (4.00%)  15 17/300 (5.67%)  24
Blood creatinine increased  1  13/300 (4.33%)  16 21/300 (7.00%)  23
Haemoglobin decreased  1  7/300 (2.33%)  11 16/300 (5.33%)  33
Platelet count decreased  1  17/300 (5.67%)  26 21/300 (7.00%)  31
Metabolism and nutrition disorders     
Decreased appetite  1  97/300 (32.33%)  117 106/300 (35.33%)  127
Fluid overload  1  30/300 (10.00%)  30 29/300 (9.67%)  31
Hyperglycaemia  1  13/300 (4.33%)  17 21/300 (7.00%)  27
Hypocalcaemia  1  21/300 (7.00%)  25 13/300 (4.33%)  18
Hypokalaemia  1  45/300 (15.00%)  53 44/300 (14.67%)  52
Hypomagnesaemia  1  51/300 (17.00%)  68 51/300 (17.00%)  55
Hypophosphataemia  1  37/300 (12.33%)  39 27/300 (9.00%)  28
Musculoskeletal and connective tissue disorders     
Arthralgia  1  13/300 (4.33%)  21 25/300 (8.33%)  29
Back pain  1  33/300 (11.00%)  37 40/300 (13.33%)  46
Musculoskeletal pain  1  10/300 (3.33%)  10 16/300 (5.33%)  20
Pain in extremity  1  28/300 (9.33%)  34 29/300 (9.67%)  31
Nervous system disorders     
Dizziness  1  44/300 (14.67%)  53 40/300 (13.33%)  43
Dysgeusia  1  31/300 (10.33%)  37 33/300 (11.00%)  36
Headache  1  75/300 (25.00%)  91 87/300 (29.00%)  105
Neuropathy peripheral  1  22/300 (7.33%)  24 7/300 (2.33%)  7
Tremor  1  17/300 (5.67%)  17 10/300 (3.33%)  11
Psychiatric disorders     
Anxiety  1  47/300 (15.67%)  54 39/300 (13.00%)  42
Confusional state  1  16/300 (5.33%)  16 9/300 (3.00%)  10
Insomnia  1  58/300 (19.33%)  61 62/300 (20.67%)  67
Renal and urinary disorders     
Dysuria  1  21/300 (7.00%)  22 23/300 (7.67%)  25
Haematuria  1  12/300 (4.00%)  13 16/300 (5.33%)  16
Renal failure acute  1  22/300 (7.33%)  22 23/300 (7.67%)  30
Respiratory, thoracic and mediastinal disorders     
Cough  1  43/300 (14.33%)  48 54/300 (18.00%)  60
Dyspnoea  1  39/300 (13.00%)  45 30/300 (10.00%)  34
Dyspnoea exertional  1  11/300 (3.67%)  12 20/300 (6.67%)  27
Epistaxis  1  19/300 (6.33%)  21 24/300 (8.00%)  29
Hiccups  1  15/300 (5.00%)  15 19/300 (6.33%)  19
Nasal congestion  1  12/300 (4.00%)  13 18/300 (6.00%)  18
Oropharyngeal pain  1  43/300 (14.33%)  47 42/300 (14.00%)  49
Skin and subcutaneous tissue disorders     
Alopecia  1  17/300 (5.67%)  19 12/300 (4.00%)  13
Dry skin  1  20/300 (6.67%)  21 23/300 (7.67%)  26
Erythema  1  18/300 (6.00%)  19 10/300 (3.33%)  11
Pruritus  1  32/300 (10.67%)  38 32/300 (10.67%)  36
Rash  1  52/300 (17.33%)  63 46/300 (15.33%)  55
Rash generalised  1  7/300 (2.33%)  8 18/300 (6.00%)  20
Vascular disorders     
Hypertension  1  35/300 (11.67%)  38 40/300 (13.33%)  50
Hypotension  1  43/300 (14.33%)  53 48/300 (16.00%)  65
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
Any publication or other public presentation of results from this study requires prior review and written approval of the Sponsor. Draft abstracts, manuscripts, and materials for presentation at scientific meetings should be provided to the Sponsor at least 90 working days prior to abstract or other relevant submission deadlines.
Results Point of Contact
Name/Title: Senior Vice President, Global Clinical Development
Organization: Merck Sharp & Dohme Corp.
Phone: 1-800-672-6372
Responsible Party: Optimer Pharmaceuticals LLC
ClinicalTrials.gov Identifier: NCT01691248     History of Changes
Other Study ID Numbers: 5119-001
OPT-80-302 ( Other Identifier: Optimerpharma Study Number )
First Submitted: September 19, 2012
First Posted: September 24, 2012
Results First Submitted: March 11, 2016
Results First Posted: April 12, 2016
Last Update Posted: September 18, 2018