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)

Tracking Information
First Submitted Date ICMJE	September 19, 2012
First Posted Date ICMJE	September 24, 2012
Results First Submitted Date	March 11, 2016
Results First Posted Date	April 12, 2016
Last Update Posted Date	May 1, 2017
Actual Study Start Date ICMJE	October 10, 2012
Actual Primary Completion Date	March 18, 2015 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: March 11, 2016)	Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to 30 Days Post-treatment Follow-up. [ Time Frame: Up to 30 days post-treatment ]; 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.
Original Primary Outcome Measures ICMJE; (submitted: September 21, 2012)	The occurrence of CDAD from start of study treatment up to 30 days post-treatment follow-up in HSCT subjects. [ Time Frame: 30 days post-treatment ]; CDAD is defined as: Diarrhea: (change in bowel habits with >3 unformed bowel movements in a 24 hour period) and; 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
Change History	Complete list of historical versions of study NCT01691248 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: March 11, 2016)	Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to 60 Days Post-treatment. [ Time Frame: Up to 60 days post-treatment ]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.; Percentage of Participants With Occurrence of CDAD From Start of Study Treatment up to Day 70 of Study. [ Time Frame: Up to Day 70 of study ]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.
Original Secondary Outcome Measures ICMJE; (submitted: September 21, 2012)	Occurrence of CDAD from start of study treatment up to 60 days post-treatment [ Time Frame: Up to 60 days post-treatment ]
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Safety and Efficacy of Fidaxomicin Versus Placebo for Prophylaxis Against Clostridium Difficile-Associated Diarrhea in Adults Undergoing Hematopoietic Stem Cell Transplantation (MK-5119-001)
Official Title ICMJE	DEFLECT-1: A Phase 3b Multi-Center, Double-Blind, Randomized, Placebo Controlled Study to Demonstrate the Safety and Efficacy of Fidaxomicin for Prophylaxis Against Clostridium Difficile-Associated Diarrhea in Adults Undergoing Hematopoietic Stem Cell Transplantation
Brief Summary	The objective of this study is to demonstrate the efficacy and safety of Fidaxomicin versus placebo for prophylaxis against Clostridium difficile-Associated Diarrhea (CDAD) in adult participants undergoing hematopoietic stem cell transplantation (HSCT). The primary hypothesis is that Fidaxomicin is superior to placebo in preventing CDAD in participants undergoing HSCT.
Detailed Description	Not Provided
Study Type ICMJE	Interventional
Study Phase	Phase 3
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Prevention
Condition ICMJE	Clostridium Difficile-Associated Diarrhea (CDAD)
Intervention ICMJE	Drug: fidaxomicin Fidaxomicin 200 mg tablet once daily from the start (+/- 2 days) of condition (prior to transplantation) or at the time of Fluoroquinolone initiation. Study drug treatment will continue until 7 days after either neutrophil engraftment or the completion of any Fluoroquinolone antibiotic regimen (whichever occurs later). Study drug treatment will stop at onset of CDAD or no longer than 40 days of duration, even if other antibiotics are still administered or neutrophil engraftment extends beyond 40 days. Other Names: DIFICID DIFICLIR OPT-80 PAR-101; Drug: Placebo Placebo tablet once daily from the start (+/- 2 days) of condition (prior to transplantation) or at the time of Fluoroquinolone initiation. Treatment will continue until 7 days after either neutrophil engraftment or the completion of any Fluoroquinolone antibiotic regimen (whichever occurs later). Treatment will stop at onset of CDAD or no longer than 40 days of duration, even if other antibiotics are still administered or neutrophil engraftment extends beyond 40 days.
Study Arms	Active Comparator: Fidaxomicin 200 mg Fidaxomicin tablet once daily for no longer than 40 days Intervention: Drug: fidaxomicin; Placebo Comparator: Placebo Placebo tablet once daily for no longer than 40 days Intervention: Drug: Placebo
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: April 27, 2015)	611
Original Estimated Enrollment ICMJE; (submitted: September 21, 2012)	340
Actual Study Completion Date	April 16, 2015
Actual Primary Completion Date	March 18, 2015 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	Inclusion Criteria: Male or female 18 years of age or older.; Females of childbearing potential must be using an adequate and reliable method of contraception (e.g., abstinence, barrier with additional spermicide foam or jelly, intrauterine device, hormonal contraception). Males and females must agree to avoid conception during treatment and for four weeks following the end of study treatment.; Is undergoing HSCT with planned Fluoroquinolone prophylaxis.; Informed consent is provided. Exclusion Criteria: Ongoing active CDAD infection (as evidenced by clinical signs of diarrhea along with the presence of either toxin A and/or B [or their respective genes, tcdA and/or tcdB] of C. difficile in the stool) or current treatment for CDAD.; Undergoing cord blood transplants.; Has fulminant colitis, toxic megacolon, or ileus.; A history of inflammatory bowel disease (ulcerative colitis or Crohn's disease).; Women who are pregnant or are actively breast feeding (all women of childbearing potential must have a negative pregnancy test result prior to dosing study drug).; Use of any drugs potentially useful in the treatment of CDAD (e.g. oral Vancomycin, Metronidazole, oral Bacitracin, Fusidic Acid, Rifaximin, and Nitazoxanide).; Any other condition that, in the opinion of the investigator, would jeopardize the safety or rights of the participant in the study, would make it unlikely for the participant to complete the study, or would confound the results of the study.; Participation in other clinical research studies utilizing an investigational agent within one month prior to screening and during the study treatment period.
Sex/Gender	Sexes Eligible for Study: All
Ages	18 Years and older (Adult, Senior)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Not Provided
Removed Location Countries	Canada, United States
Administrative Information
NCT Number ICMJE	NCT01691248
Other Study ID Numbers ICMJE	5119-001; OPT-80-302 ( Other Identifier: Optimerpharma Study Number )
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Plan to Share IPD: Yes Plan Description: http://www.merck.com/clinical-trials/pdf/Merck%20Procedure%20on%20Clinical%20Trial%20Data%20Access%20Final_Updated%20July_9_2014.pdf http://engagezone.msd.com/ds_documentation.php
Responsible Party	Optimer Pharmaceuticals LLC
Study Sponsor ICMJE	Optimer Pharmaceuticals LLC
Collaborators ICMJE	Not Provided
Investigators ICMJE	Study Director: Medical Director Merck Sharp & Dohme Corp.
PRS Account	Optimer Pharmaceuticals LLC
Verification Date	March 2017
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP