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

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Fidaxomicin	Placebo
Arm/Group Description	200 mg Fidaxomicin tablet once dail...	Placebo tablet once daily for no lo...
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.

Baseline Characteristics

Arm/Group Title		Fidaxomicin	Placebo	Total
Arm/Group Description		200 mg Fidaxomicin tablet once dail...	Placebo tablet once daily for no lo...	Total of all reporting groups
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
Baseline Analysis Population Description		...
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%

Outcome Measures

1.Primary Outcome

Title	Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to 30 Days Post-treatment Follow-up.
Description	CDAD is defined as follows: Diarrhea: (change in bowel habits with >...
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

Outcome Measure Data

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
Arm/Group Description:	200 mg Fidaxomicin tablet once dail...	Placebo tablet once daily for no lo...
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)

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.
Description	CDAD is defined as follows: Diarrhea: (change in bowel habits with >...
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

Outcome Measure Data

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
Arm/Group Description:	200 mg Fidaxomicin tablet once dail...	Placebo tablet once daily for no lo...
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)

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.
Description	CDAD is defined as follows: Diarrhea: (change in bowel habits with >...
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

Outcome Measure Data

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
Arm/Group Description:	200 mg Fidaxomicin tablet once dail...	Placebo tablet once daily for no lo...
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)

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

Adverse Events

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
Arm/Group Description	200 mg Fidaxomicin tablet once dail...		Placebo tablet once daily for no lo...
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	--/--		--/--
Serious Adverse Events 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
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

Limitations and Caveats

[Not Specified]

More Information

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

EMail: ClinicalTrialsDisclosure@merck.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Mullane KM, Winston DJ, Nooka A, Morris MI, Stiff P, Dugan MJ, Holland H, Gregg K, Adachi JA, Pergam SA, Alexander BD, Dubberke ER, Broyde N, Gorbach SL, Sears PS. A Randomized, Placebo-Controlled Trial of Fidaxomicin for Prophylaxis of Clostridium difficile Associated Diarrhea in Adults undergoing Hematopoietic Stem Cell Transplantation. Clin Infect Dis. 2018 Jun 9. doi: 10.1093/cid/ciy484. [Epub ahead of print]

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

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