Trial Outcomes & Findings for Safety, Tolerability, and Pharmacokinetics of Fidaxomicin in Pediatric Subjects With Clostridium Difficile-associated Diarrhea (CDAD) (NCT NCT01591863)

NCT ID: NCT01591863

Last Updated: 2018-09-18

Results Overview

Number of participants with adverse events, as categorized by MedDRA.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 38 participants
• Primary outcome timeframe: Enrollment through end of study (Day 38-41)
• Results posted on: 2018-09-18

Participant Flow

Participant milestones

Measure	Fidaxomicin fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Overall Study STARTED	38
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	14

Reasons for withdrawal

Measure	Fidaxomicin fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Overall Study Adverse Event	3
Overall Study Withdrawal by Subject	1
Overall Study recurrence	9
Overall Study Lack of Efficacy	1

Baseline Characteristics

Safety, Tolerability, and Pharmacokinetics of Fidaxomicin in Pediatric Subjects With Clostridium Difficile-associated Diarrhea (CDAD)

Baseline characteristics by cohort

Measure	Fidaxomicin n=38 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Age, Continuous	99.4 months STANDARD_DEVIATION 68.9 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: Enrollment through end of study (Day 38-41)

Population: Subjects receiving any amount of study drug

Number of participants with adverse events, as categorized by MedDRA.

Outcome measures

Measure	Fidaxomicin n=38 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Number of Participants With Adverse Events.	28 participants

PRIMARY outcome

Timeframe: 3-5 hours after administration

Population: Treated subjects with evaluable plasma pharmacokinetic data

3-5 hour plasma levels of fidaxomicin (mean)

Outcome measures

Measure	Fidaxomicin n=36 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Investigate Concentrations of Fidaxomicin in Plasma Samples.	13.363 ng/mL Standard Deviation 15.472

PRIMARY outcome

Timeframe: End of Therapy; Day 10-11

Population: Treated subjects with evaluable fecal data

End of therapy fecal levels of fidaxomicin (mean)

Outcome measures

Measure	Fidaxomicin n=30 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Investigate Concentrations of Fidaxomicin in Fecal Samples.	3227.93 microgram/g Standard Deviation 2668.08

PRIMARY outcome

Timeframe: 3-5 hours after administration

Population: Treated subjects with evaluable plasma pharmacokinetic data

3-5 hour plasma levels of OP-1118 (mean)

Outcome measures

Measure	Fidaxomicin n=36 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Investigate Concentrations of the Main Metabolite OP-1118 in Plasma Samples.	60.016 ng/mL Standard Deviation 152.196

PRIMARY outcome

Timeframe: End of Therapy; Day 10-11

Population: Treated subjects with evaluable fecal data

End of therapy fecal levels of OP-1118 (mean)

Outcome measures

Measure	Fidaxomicin n=30 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Investigate Concentrations of the Main Metabolite OP-1118 in Fecal Samples.	865.49 microgram/g Standard Deviation 614.15

SECONDARY outcome

Timeframe: Day 10

Population: Treated subjects with positive toxin assay result within 24 hours of enrollment

Positive clinical response defined as resolution of diarrhea

Outcome measures

Measure	Fidaxomicin n=38 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Evaluate the Clinical Outcome by Assessment of Clinical Response.	92.1 percentage of subjects Interval 83.5 to 100.0

SECONDARY outcome

Timeframe: 28 days post-treatment

Population: Treated subjects with positive toxin assay result within 24 hours of enrollment

Positive clinical response without recurrence through the follow-up period

Outcome measures

Measure	Fidaxomicin n=38 Participants fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Evaluate the Clinical Outcome by Assessment of Sustained Clinical Response.	65.8 percentage of participants Interval 50.7 to 80.9

Adverse Events

Fidaxomicin

Serious events: 9 serious events

Other events: 17 other events

Deaths: 0 deaths

Serious adverse events

Measure	Fidaxomicin n=38 participants at risk fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Infections and infestations Clostridium difficilie colitis	7.9% 3/38 • From informed consent through 30 days after the last administration of study treatment.
Blood and lymphatic system disorders febrile neutropenia	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders Hematemesis	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders vomiting	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Infections and infestations adenovirus infection	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.
Infections and infestations clostridial infection	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.
Infections and infestations septic shock	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.
Injury, poisoning and procedural complications gastrostomy failure	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.
Respiratory, thoracic and mediastinal disorders respiratory failure	2.6% 1/38 • From informed consent through 30 days after the last administration of study treatment.

Other adverse events

Measure	Fidaxomicin n=38 participants at risk fidaxomicin: 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days. 6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Gastrointestinal disorders abdominal pain upper	7.9% 3/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders constipation	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders diarrhea	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders nausea	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders oesophagitis	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Gastrointestinal disorders vomiting	7.9% 3/38 • From informed consent through 30 days after the last administration of study treatment.
General disorders chest pain	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
General disorders pyrexia	10.5% 4/38 • From informed consent through 30 days after the last administration of study treatment.
Infections and infestations nasopharyngitis	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Metabolism and nutrition disorders dehydration	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Nervous system disorders headache	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Skin and subcutaneous tissue disorders urticaria	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.
Vascular disorders hypertension	5.3% 2/38 • From informed consent through 30 days after the last administration of study treatment.

Additional Information

Senior Vice President, Global Clinical Development

Merck Sharp & Dohme Corp.

Phone: 1-800-672-6372

Email: ClinicalTrialsDisclosure@merck.com

Results disclosure agreements

• Principal investigator is a sponsor employee 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 30 working days prior to submission deadlines. Authorship of publications resulting from this study will be based on generally accepted criteria for major medical journals.
• Publication restrictions are in place

Restriction type: OTHER