Clostridium Difficile-associated Diarrhea
Conditions
Keywords
Clostridium difficile-associated diarrhea, CDAD, Pediatric
Brief summary
The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of fidaxomicin in pediatric subjects with Clostridium difficile-associated diarrhea (CDAD).
Interventions
DRUGfidaxomicin
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.
Sponsors
Optimer Pharmaceuticals LLC, a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
* Male or female 6 months to 17 years 11 months of age, inclusive; * Female subjects of childbearing potential must use adequate contraception * Diagnosed with CDAD
Exclusion criteria
* Concurrent use of oral vancomycin or metronidazole or any other effective treatments for CDAD * Fulminant colitis * History of inflammatory bowel disease * Pregnant or breast-feeding * Need for concurrent use of some P-glycoprotein inhibitors during therapy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Investigate Concentrations of Fidaxomicin in Fecal Samples. | End of Therapy; Day 10-11 | End of therapy fecal levels of fidaxomicin (mean) |
| Investigate Concentrations of the Main Metabolite OP-1118 in Fecal Samples. | End of Therapy; Day 10-11 | End of therapy fecal levels of OP-1118 (mean) |
| Number of Participants With Adverse Events. | Enrollment through end of study (Day 38-41) | Number of participants with adverse events, as categorized by MedDRA. |
| Investigate Concentrations of Fidaxomicin in Plasma Samples. | 3-5 hours after administration | 3-5 hour plasma levels of fidaxomicin (mean) |
| Investigate Concentrations of the Main Metabolite OP-1118 in Plasma Samples. | 3-5 hours after administration | 3-5 hour plasma levels of OP-1118 (mean) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate the Clinical Outcome by Assessment of Sustained Clinical Response. | 28 days post-treatment | Positive clinical response without recurrence through the follow-up period |
| Evaluate the Clinical Outcome by Assessment of Clinical Response. | Day 10 | Positive clinical response defined as resolution of diarrhea |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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. | 38 |
| Total | 38 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 3 |
| Overall Study | Lack of Efficacy | 1 |
| Overall Study | recurrence | 9 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Fidaxomicin |
|---|---|
| Age, Continuous | 99.4 months STANDARD_DEVIATION 68.9 |
| Sex: Female, Male Female | 16 Participants |
| Sex: Female, Male Male | 22 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 17 / 38 |
| serious Total, serious adverse events | 9 / 38 |
Outcome results
Primary
Investigate Concentrations of Fidaxomicin in Fecal Samples.
End of therapy fecal levels of fidaxomicin (mean)
Time frame: End of Therapy; Day 10-11
Population: Treated subjects with evaluable fecal data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Fidaxomicin | Investigate Concentrations of Fidaxomicin in Fecal Samples. | 3227.93 microgram/g | Standard Deviation 2668.08 |
Primary
Investigate Concentrations of Fidaxomicin in Plasma Samples.
3-5 hour plasma levels of fidaxomicin (mean)
Time frame: 3-5 hours after administration
Population: Treated subjects with evaluable plasma pharmacokinetic data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Fidaxomicin | Investigate Concentrations of Fidaxomicin in Plasma Samples. | 13.363 ng/mL | Standard Deviation 15.472 |
Primary
Investigate Concentrations of the Main Metabolite OP-1118 in Fecal Samples.
End of therapy fecal levels of OP-1118 (mean)
Time frame: End of Therapy; Day 10-11
Population: Treated subjects with evaluable fecal data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Fidaxomicin | Investigate Concentrations of the Main Metabolite OP-1118 in Fecal Samples. | 865.49 microgram/g | Standard Deviation 614.15 |
Primary
Investigate Concentrations of the Main Metabolite OP-1118 in Plasma Samples.
3-5 hour plasma levels of OP-1118 (mean)
Time frame: 3-5 hours after administration
Population: Treated subjects with evaluable plasma pharmacokinetic data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Fidaxomicin | Investigate Concentrations of the Main Metabolite OP-1118 in Plasma Samples. | 60.016 ng/mL | Standard Deviation 152.196 |
Primary
Number of Participants With Adverse Events.
Number of participants with adverse events, as categorized by MedDRA.
Time frame: Enrollment through end of study (Day 38-41)
Population: Subjects receiving any amount of study drug
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Fidaxomicin | Number of Participants With Adverse Events. | 28 participants |
Secondary
Evaluate the Clinical Outcome by Assessment of Clinical Response.
Positive clinical response defined as resolution of diarrhea
Time frame: Day 10
Population: Treated subjects with positive toxin assay result within 24 hours of enrollment
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Fidaxomicin | Evaluate the Clinical Outcome by Assessment of Clinical Response. | 92.1 percentage of subjects |
Secondary
Evaluate the Clinical Outcome by Assessment of Sustained Clinical Response.
Positive clinical response without recurrence through the follow-up period
Time frame: 28 days post-treatment
Population: Treated subjects with positive toxin assay result within 24 hours of enrollment
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Fidaxomicin | Evaluate the Clinical Outcome by Assessment of Sustained Clinical Response. | 65.8 percentage of participants |