Trial Outcomes & Findings for Comparison of Cadazolid Versus Vancomycin in Children With Clostridium Difficile-associated Diarrhea (CDAD) (NCT NCT03105479)

Results Overview

This is the percentage of participants in part B reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive). percentage of subjects with a clinical cure

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

1 participants

Primary outcome timeframe

Day 10 (End of Treatment) + 2 days

Results posted on

2025-07-08

Participant Flow

Due to early termination of the study after sponsor's decision to discontinue the development of cadazolid, only one patient was enrolled at one site in the US.

Participant milestones

Participant milestones
Measure	Part A / Cohort A Subjects from 12 years to 18 years old (exclusive) are to be treated with cadazolid 250 mg twice daily for 10 days. The dose may be adjusted based on the pharmacokinetic (PK) and safety data reviewed for the first 3 subjects.	Part A / Cohort B Subjects from 6 years to 12 years old (exclusive) are to be treated with cadazolid for 10 days (dose determined based on the PK and safety data from cohort A reviewed by the Independent Data Monitoring Committee (IDMC)).	Part A / Cohort C Subjects from 2 years to 6 years old (exclusive) are to be treated with cadazolid for 10 days. (dose determined based on the PK and safety data from cohort B reviewed by the IDMC).	Part A/ Cohort D Subjects from 3 months to 2 years old (exclusive) are to be treated with cadazolid for 10 days (dose determined based on the PK and safety data from cohort B reviewed by the IDMC).	Part A/ Cohort E Subjects from birth to 3 months old (exclusive) are to be treated with cadazolid for 10 days (dose determined based on the PK and safety data from cohort B reviewed by the IDMC). .	Part B / Cadazolid Subjects from birth to 18 years old (exclusive) are to be treated with cadazolid for 10 days, at the dose defined in the corresponding age cohort in Part A.	Part B / Vancomycin Subjects from birth to 18 years old (exclusive)are to be treated with vancomycin for 10 days either as capsules (for subjects able to swallow) or oral solution (for the others) .
Overall Study STARTED	1	0	0	0	0	0	0
Overall Study COMPLETED	1	0	0	0	0	0	0
Overall Study NOT COMPLETED	0	0	0	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part A / Cohort A n=1 Participants Subjects from 12 years to 18 years old (exclusive) will receive cadazolid 500 mg per day for 10 days. The dose may be adjusted based on the pharmacokinetic (PK) and safety data reviewed for the first 3 subjects.
Age, Customized Adolescents (12 years to < 18 years)	1 Participants n=1 Participants
Age, Customized Children (2 years to < 12 years	0 Participants n=1 Participants
Age, Customized infants and toddlers (3 months to < 2 years	0 Participants n=1 Participants
Sex: Female, Male Female	1 Participants n=1 Participants
Sex: Female, Male Male	0 Participants n=1 Participants

PRIMARY outcome

Timeframe: Day 10 (End of Treatment) + 2 days

Population: Due to the premature study termination, no subject was enrolled into Part B and consequently the percentage of subjects with a clinical cure could not be reported.

This is the percentage of participants in part B reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive). percentage of subjects with a clinical cure

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 10 (End of Treatment)

Population: Due to the premature study termination, cadazolid concentrations were obtained from only one subject and pharmacokineitc plasma profile was not analyzed because of lack of meaningful data.

Blood samples are collected at different timepoints on Day 10 for the determination of cadazolid Cmax after 10 days of treatment.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 10 (End of Treatment)

Population: Due to the premature study termination, cadazolid concentrations were obtained from only one subject and pharmacokineitc plasma profile was not analyzed because of lack of meaningful data.

Blood samples are collected at different timepoints to determine the time when the maximal plasma concentration of cadazolid is reached.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 10 (End of Treatment)

Population: Due to the premature study termination, cadazolid concentrations were obtained from only one subject and pharmacokineitc plasma profile was not analyzed because of lack of meaningful data.

Blood samples are collected at different timepoints for the determination of the cadazolid AUC over one dosing interval (0-12h) on Day 10.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 10 (End of Treatment)

Population: Due to premature termination, fecal sample was collected from only one subject, consequently mean values cannot be provided and no statistical analyses can be performed.

A fecal sample is collected as the end-of-treatment visit in all participants in Part A.

Outcome measures

Outcome measures
Measure	Part B n=1 Participants Due to early termination of the study, no subject was enrolled into Part B.	Part B / Cadazolid No subject was enrolled in cohort B due to early study termination.
Fecal Concentrations of Cadazolid During Part A	4520 mcg/g	—

SECONDARY outcome

Timeframe: Day 10 (End of Treatment) + 2 days

Population: Due to premature termination, data were collected from only one subject, consequently percentage of participants with clinical cure cannot be calculated and no statistical analyses can be performed

This is the percentage of participants in Part A reported as with a clinical cure. Clinical Cure is defined as: • \<3 unformed bowel movement (UBM) per day (or no water diarrhea if subjects \< 2 years of age), for at least 2 consecutive days between first dose of study treatment up to end of treatment (EOT) (inclusive) AND • Subject remains well up to EOT + 2 days (inclusive) based on investigator judgment AND • No need for additional antimicrobial treatment active against Clostridium difficile-associated diarrhea (CDAD) between first dose of study treatment up to EOT + 2 days (inclusive).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 40 (on average)

Population: Due to the premature termination of the study and consequent lack of meaningful data, no analyses were performed.

This is the percentage of participants in Parts A and B reported as with sustained clinical cure. Sustained clinical cure is defined as • Clinical Cure and no Recurrence until 30 days after the last study drug intake (end of study).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 40 (on average)

Population: Due to the premature termination of the study and consequent lack of meaningful data, no analyses were performed.

This is the percentage of participants in Parts A and B assessed as having a recurrence out of subjects meeting the criteria for Clinical Cure. Recurrence is defined as: • Clinical Cure AND New episode of diarrhea with ≥ 3 UBMs (or watery diarrhea if subject \< 2 years) on any day between EOT + 3 days and end of study AND • Stool test showing positive C. difficile (as defined in Inclusion Criterion 4), AND •Antimicrobial treatment active against CDAD started between EOT + 3 days and end of study.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 40 (on average)

Population: Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B

This it the time (in days) elapsed between the last dose of study drug and the onset day of new episode of diarrhea reported as Kaplan-Meier estimates (KM estimates)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 10

Population: Due to premature termination, data were collected from only one subject, consequently KM estimates cannot be calculated and no statistical analyses can be performed

This is the time (in days) elapsed between the first dose of study treatment and the resolution of diarrhea and reported as Kaplan-Meier estimates (KM estimates). The date of resolution of Diarrhea (ROD) is defined as the date of the first day of the 2 consecutive days on treatment with \< 3 UBM (or no watery diarrhea for subjects \< 2 years of age). Time to ROD is the time (in days) elapsed between the first dose of study treatment and the ROD

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 10

Population: Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B

Number of participants who prematurely discontinued the study treatment due to an adverse event

Outcome measures

Outcome measures
Measure	Part B n=1 Participants Due to early termination of the study, no subject was enrolled into Part B.	Part B / Cadazolid No subject was enrolled in cohort B due to early study termination.
Adverse Events Leading to Premature Discontinuation of Study Treatment	0 Participants	—

SECONDARY outcome

Timeframe: Day 17 (on average)

Population: Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B

Number of participants with any marked abnormalities in laboratory parameters up to 7 days after end of treatment

Outcome measures

Outcome measures
Measure	Part B n=1 Participants Due to early termination of the study, no subject was enrolled into Part B.	Part B / Cadazolid No subject was enrolled in cohort B due to early study termination.
Marked Abnormalities in Clinical Laboratory Parameters	0 Participants	—

SECONDARY outcome

Timeframe: Day 17 (on average)

Population: Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B

Number of subjects with any treatment-emergent abnormalities in vital signs (up to 7 days after end of treatment)

Outcome measures

Outcome measures
Measure	Part B n=1 Participants Due to early termination of the study, no subject was enrolled into Part B.	Part B / Cadazolid No subject was enrolled in cohort B due to early study termination.
Marked Abnormalities in Vital Signs	0 Participants	—

SECONDARY outcome

Timeframe: Day 17 (on average)

Population: Due to premature termination, no subject was enrolled in Part B. Consequently, no data can be reported during Part B

Number of subjects with any TEAEs. A TEAE is any adverse event temporally associated with the use of study treatment (from study treatment initiation until 7 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.

Outcome measures

Outcome measures
Measure	Part B n=1 Participants Due to early termination of the study, no subject was enrolled into Part B.	Part B / Cadazolid No subject was enrolled in cohort B due to early study termination.
Treatment-emergent Adverse Events (TEAES)	0 Participants	—

Adverse Events

Overall Study (Part A and Part B)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Overall Study (Part A and Part B) n=1 participants at risk Only one subject was included in cohort A; no subject was enrolled in the other cohorts of Part A or Part B due to early study termination.
Respiratory, thoracic and mediastinal disorders Sore throat	100.0% 1/1 • Number of events 1 • From study treatment initiation up to Day 37 (i.e., 27 days after the end of treatment) All adverse events (AE) which occurred at any time during the treatment period (10 days with cadazolid) and during the follow-up period (about 30 days) are reported. All AEs reported below occurred during the follow-up period.
Nervous system disorders Headache	100.0% 1/1 • Number of events 1 • From study treatment initiation up to Day 37 (i.e., 27 days after the end of treatment) All adverse events (AE) which occurred at any time during the treatment period (10 days with cadazolid) and during the follow-up period (about 30 days) are reported. All AEs reported below occurred during the follow-up period.
Gastrointestinal disorders Vomiting	100.0% 1/1 • Number of events 1 • From study treatment initiation up to Day 37 (i.e., 27 days after the end of treatment) All adverse events (AE) which occurred at any time during the treatment period (10 days with cadazolid) and during the follow-up period (about 30 days) are reported. All AEs reported below occurred during the follow-up period.

Additional Information

clinical trial disclosure desk

Actelion Pharmaceuticals Ltd

Phone: 0041615656565

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Any study-related publication written independently by investigators must be submitted to Actelion for review at least 30 days prior to submission for publication or presentation. Upon review, Actelion may provide comments, and may also request alterations and/or deletions for the sole purpose of protecting its confidential information and/or patent rights.
Publication restrictions are in place

Restriction type: OTHER