NCT02218372

Brief Summary

The purpose of this study was to investigate the clinical response to fidaxomicin oral suspension or tablets and vancomycin oral liquid or capsules in pediatric participants with Clostridium difficile-associated diarrhea (CDAD). It also investigated the recurrence/sustained clinical response to and safety of fidaxomicin and vancomycin, as well as acceptance of the fidaxomicin oral suspension formulation.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
148

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Jan 2015

Typical duration for phase_3

Geographic Reach
10 countries

44 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 11, 2014

Completed
7 days until next milestone

First Posted

Study publicly available on registry

August 18, 2014

Completed
5 months until next milestone

Study Start

First participant enrolled

January 9, 2015

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 7, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 7, 2018

Completed
9 months until next milestone

Results Posted

Study results publicly available

December 7, 2018

Completed
Last Updated

November 26, 2024

Status Verified

November 1, 2024

Enrollment Period

3.2 years

First QC Date

August 11, 2014

Results QC Date

October 23, 2018

Last Update Submit

November 11, 2024

Conditions

Keywords

Clostridium difficile-associated Diarrhea (CDAD)vancomycinClostridium difficile infectionfidaxomicin

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With Confirmed Clinical Response (CCR) at End of Treatment (EOT) +2 Days

    Initial clinical response (ICR) for ages from birth to \< 2 years was defined as absence of watery diarrhea for 2 consecutive treatment days, remaining well until study drug discontinuation. ICR for ages ≥ 2 years to \< 18 years was defined as improvement in number and character of bowel movements as determined by \< 3 unformed bowel movements (UBMs) per day for 2 consecutive treatment days, remaining well until study drug discontinuation. CCR was defined for both age groups as not requiring further CDAD therapy within 2 days after study drug completion, and was reported with a positive (Yes) or negative (No) outcome. Resolution of diarrhea was assessed during interviews of participant/parent/legal guardian, supplemented by review of personal records (if hospitalized) and checked for presence of watery diarrhea (ages from birth to \< 2 years) or number of UBMs (for ages ≥ 2 years to \< 18 years).

    Up to day 12

Secondary Outcomes (22)

  • Percentage of Participants With Sustained Clinical Response (SCR) at EOT +9 Days

    Up to day 19

  • Percentage of Participants With Global Cure (GC) at EOT +9 Days

    Up to day 19

  • Percentage of Participants With Recurrence of CDAD at EOT +9 Days

    Up to day 19

  • Percentage of Participants With SCR at EOT +16 Days

    Up to day 26

  • Percentage of Participants With GC at EOT +16 Days

    Up to day 26

  • +17 more secondary outcomes

Study Arms (2)

Fidaxomicin

EXPERIMENTAL

Participants from birth to \< 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to \< 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.

Drug: Fidaxomicin oral suspensionDrug: Fidaxomicin tablets

Vancomycin

ACTIVE COMPARATOR

Participants from birth to \< 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to \< 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.

Drug: Vancomycin oral liquidDrug: Vancomycin capsules

Interventions

Participants from birth to \< 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days.

Also known as: Dificid, Dificlir
Fidaxomicin

Participants aged ≥ 6 years to \< 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.

Also known as: Dificlir, Dificid
Fidaxomicin

Participants from birth to \< 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days.

Vancomycin

Participants aged ≥ 6 years to \< 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.

Vancomycin

Eligibility Criteria

AgeUp to 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Subject is diagnosed with CDAD according to local diagnostic criteria. As a minimum there must be positive detection, within 72 hours prior to randomization, of either toxin A and/or toxin B in stool or positive detection of toxigenic C. difficile in stool and:
  • Subject from Birth to \< 2 years: watery diarrhea in the 24 hours prior to screening.
  • Subject ≥ 2 years to \< 18 years: ≥ 3 unformed bowel movements in the 24 hours prior to screening.
  • Male and female subjects aged from birth to \< 18 years: Note that in the United States of America subjects can only be included if aged ≥ 6 months to \< 18 years.
  • For subjects \< 5 years: Negative rotavirus test.
  • Female subject of childbearing potential:
  • must have a negative urine pregnancy test at Screening, and
  • must abstain from sexual activity for the duration of the study, or
  • must use two forms of birth control (at least one of which must be a barrier method) starting at Screening and throughout the study period and for 28 days after the final study drug administration.
  • Female subject must not be breastfeeding at Screening or during the study period, and for 28 days after the final study drug administration.
  • Female subject must not donate ova starting at Screening and throughout the study period, and for 28 days after the final study drug administration.

You may not qualify if:

  • Concurrent use of metronidazole, oral vancomycin or any other antibiotic treatments for CDAD. If the investigator feels the clinical imperative is to begin treatment before knowing the laboratory result for toxigenic C. difficile, up to four doses but no more than 24 hours of treatment with metronidazole, oral vancomycin or any other effective treatment for CDAD are allowed.
  • Subject has pseudomembranous colitis, fulminant colitis, toxic megacolon or ileus.
  • Subject has a history of inflammatory bowel disease (e.g., ulcerative colitis or Crohn's disease etc.).
  • Subject has diarrhea caused by an agent other than C. difficile (e.g. infections, infestations, drugs etc.).
  • Subject has known hypersensitivity to fidaxomicin, vancomycin or their excipients or to teicoplanin.
  • Subject has received an investigational therapy within 28 days, prior to Screening, with the exception of studies with primary treatment for cancer without novel Investigational Medicinal Product (IMP) and which do not affect the assessment of diarrhea.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (44)

Site US10015

Orange, California, 92868, United States

Location

Site US10010

Chicago, Illinois, 60637, United States

Location

Site US10004

Indianapolis, Indiana, 46202, United States

Location

Site US10025

Louisville, Kentucky, 40202, United States

Location

Site US10032

Kansas City, Missouri, 64108, United States

Location

Site US10030

Omaha, Nebraska, 68114, United States

Location

Site US10008

Stony Brook, New York, 11794-8111, United States

Location

Site US10028

Chapel Hill, North Carolina, 27599, United States

Location

Site US10027

Cincinnati, Ohio, 45229-3039, United States

Location

Site US10014

Toledo, Ohio, 43606, United States

Location

Site US10034

Johnson City, Tennessee, 37604, United States

Location

Site US10022

Memphis, Tennessee, 38105-2794, United States

Location

Site US10038

Fort Worth, Texas, 76104, United States

Location

Site US10037

Salt Lake City, Utah, 84113, United States

Location

Site BE32001

Brussels, Flemish Brabant, 1200, Belgium

Location

Site BE32003

Ghent, 9000, Belgium

Location

Site BE32002

Liège, 4020, Belgium

Location

Site CA15003

Oshawa, Ontario, L1G 2B9, Canada

Location

Site FR33002

La Tronche, 38700, France

Location

Site FR33001

Nice, 06200, France

Location

Site FR33007

Nice, 06200, France

Location

Site FR33005

Paris, 75012, France

Location

Site FR33008

Strasbourg, 67098, France

Location

Site DE49010

Essen, 45122, Germany

Location

Site DE49002

Frankfurt Am M., 60596, Germany

Location

Site DE49006

Freiburg im Breisgau, 79106, Germany

Location

Site DE49004

Mainz, 55131, Germany

Location

Site DE49001

Münster, 48149, Germany

Location

Site DE49003

Saint Augustin, 53757, Germany

Location

Site HU36006

Budapest, 1097, Hungary

Location

Site HU36004

Szeged, 6720, Hungary

Location

Site IT39004

Milan, 20122, Italy

Location

Site IT39001

Roma, 165, Italy

Location

Site PL48002

Warsaw, Masovian Voivodeship, 04-730, Poland

Location

Site PL48012

Bialystok, 15 -274, Poland

Location

Site PL48007

Bydgoszcz, 85-030, Poland

Location

Site PL48004

Dębica, 39-200, Poland

Location

Site PL48014

Rzeszów, 35-210, Poland

Location

Site PL48006

Tarnów, 33-100, Poland

Location

Site RO40005

Bucharest, Romania

Location

Site ES34002

Barcelona, 8950, Spain

Location

Site ES34007

Madrid, 28009, Spain

Location

Site ES34004

Madrid, 28046, Spain

Location

Site ES34003

Valencia, 46026, Spain

Location

Related Publications (2)

  • Wolf J, Kalocsai K, Fortuny C, Lazar S, Bosis S, Korczowski B, Petit A, Bradford D, Croos-Dabrera R, Incera E, Melis J, van Maanen R. Safety and Efficacy of Fidaxomicin and Vancomycin in Children and Adolescents with Clostridioides (Clostridium) difficile Infection: A Phase 3, Multicenter, Randomized, Single-blind Clinical Trial (SUNSHINE). Clin Infect Dis. 2020 Dec 17;71(10):2581-2588. doi: 10.1093/cid/ciz1149.

  • Borali E, De Giacomo C. Clostridium Difficile Infection in Children: A Review. J Pediatr Gastroenterol Nutr. 2016 Dec;63(6):e130-e140. doi: 10.1097/MPG.0000000000001264.

Related Links

MeSH Terms

Conditions

Clostridium Infections

Interventions

FidaxomicinVancomycin

Condition Hierarchy (Ancestors)

Gram-Positive Bacterial InfectionsBacterial InfectionsBacterial Infections and MycosesInfections

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic ChemicalsPolyketidesMacrocyclic CompoundsPolycyclic CompoundsGlycopeptidesGlycoconjugatesCarbohydratesPeptidesAmino Acids, Peptides, and Proteins

Results Point of Contact

Title
Clinical Trial Disclosure
Organization
Astellas Pharma Global Development, Inc.

Study Officials

  • Clinical Research Physician

    Astellas Pharma Europe B.V.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 11, 2014

First Posted

August 18, 2014

Study Start

January 9, 2015

Primary Completion

March 7, 2018

Study Completion

March 7, 2018

Last Updated

November 26, 2024

Results First Posted

December 7, 2018

Record last verified: 2024-11

Data Sharing

IPD Sharing
Will share

Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Shared Documents
STUDY PROTOCOL, SAP, CSR
Time Frame
Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
Access Criteria
Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.
More information

Locations