Trial Outcomes & Findings for Pharmacokinetics of Intravenous Difelikefalin in Chinese Adult Subjects on Haemodialysis (NCT NCT05885763)

NCT ID: NCT05885763

Last Updated: 2025-02-14

Results Overview

Cmax = Maximum (peak) observed plasma concentration

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 30 participants
• Primary outcome timeframe: 1 week
• Results posted on: 2025-02-14

Participant Flow

Participant milestones

Measure	1-week, Single Arm, Open Label Treatment Phase Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pharmacokinetics of Intravenous Difelikefalin in Chinese Adult Subjects on Haemodialysis

Baseline characteristics by cohort

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	30 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Race/Ethnicity, Customized Chinese	30 Participants n=5 Participants
Region of Enrollment China	30 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 week

Cmax = Maximum (peak) observed plasma concentration

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Evaluation of the PK Profile of Difelikefalin - Cmax - Dose 1	3.70 ng/mL Geometric Coefficient of Variation 33.1

PRIMARY outcome

Timeframe: 1 week

Tmax = Time to reach maximum observed plasma concentration

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Evaluation of the PK Profile of Difelikefalin - Tmax - Dose 1	0.08 Hours Interval 0.05 to 0.98

PRIMARY outcome

Timeframe: 1 week

AUC0-t = Area under the concentration-versus-time curve (AUC) from time zero to time "t"

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Evaluation of the PK Profile of Difelikefalin - AUC0-t - Dose 1	48.3 h*ng/mL Geometric Coefficient of Variation 27.6

PRIMARY outcome

Timeframe: 1 week

AUCinf = AUC from time zero to infinity

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
AUCinf - Dose 1	63.2 h*ng/mL Geometric Coefficient of Variation 43.9

PRIMARY outcome

Timeframe: 1 week

AUCextrap(%) = percentage of AUCinf based on extrapolation

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
AUCextrap(%) - Dose 1	23.5 percentage Geometric Coefficient of Variation 49.3

PRIMARY outcome

Timeframe: 1 week

t½ = elimination half-life

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
t½ - Dose 1	22.2 Hours Geometric Coefficient of Variation 36.4

PRIMARY outcome

Timeframe: 1 week

Clearance = the volume of blood or plasma that can be freed of a specified constituent in a specified time by its excretion into the urine through the kidneys

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Clearance - Dose 1	0.510 L/H Geometric Coefficient of Variation 43.6

PRIMARY outcome

Timeframe: 1 week

Vz = volume of distribution

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Vz - Dose 1	16.3 L Geometric Coefficient of Variation 20.4

PRIMARY outcome

Timeframe: 1 week

Cmax = Maximum (peak) observed plasma concentration

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=29 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Evaluation of the PK Profile of Difelikefalin - Cmax - Dose 3	4.23 ng/mL Geometric Coefficient of Variation 23.8

PRIMARY outcome

Timeframe: 1 week

Tmax = Time to reach maximum observed plasma concentration

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=29 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Evaluation of the PK Profile of Difelikefalin - Tmax - Dose 3	0.08 Hours Interval 0.05 to 0.57

PRIMARY outcome

Timeframe: 1 week

AUC0-t = Area under the concentration-versus-time curve (AUC) from time zero to time "t"

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=29 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Evaluation of the PK Profile of Difelikefalin - AUC0-t - Dose 3	65.6 h*ng/mL Geometric Coefficient of Variation 33.8

PRIMARY outcome

Timeframe: 1 week

AUCinf = AUC from time zero to infinity

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=29 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
AUCinf - Dose 3	77.6 h*ng/mL Geometric Coefficient of Variation 48.0

PRIMARY outcome

Timeframe: 1 week

AUCextrap(%) = percentage of AUCinf based on extrapolation

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=29 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
AUCextrap(%) - Dose 3	15.1 percentage Geometric Coefficient of Variation 61.4

PRIMARY outcome

Timeframe: 1 week

t½ = elimination half-life

Outcome measures

Measure	1-week, Single Arm, Open Label Treatment Phase n=29 Participants Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
t½ - Dose 3	25.0 Hours Geometric Coefficient of Variation 37.7

Adverse Events

1-week, Single Arm, Open Label Treatment Phase

Serious events: 2 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 participants at risk Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Skin and subcutaneous tissue disorders Abscess limb	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Nervous system disorders Syncope	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.

Other adverse events

Measure	1-week, Single Arm, Open Label Treatment Phase n=30 participants at risk Difelikefalin Injection: Participants receive Difelikefalin three times a week (0,5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis.
Nervous system disorders Paraesthesia	10.0% 3/30 • Number of events 3 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Nervous system disorders Dizziness	6.7% 2/30 • Number of events 2 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Metabolism and nutrition disorders Hyperkalaemia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Metabolism and nutrition disorders Hyperlipidaemia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Metabolism and nutrition disorders Hypoalbuminaemia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Metabolism and nutrition disorders Hypocalcaemia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Metabolism and nutrition disorders Hypoglycaemia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Gastrointestinal disorders Constipation	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Gastrointestinal disorders Diarrhoea	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Gastrointestinal disorders Vomiting	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
General disorders Asthenia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Infections and infestations Nasopharyngitis	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Investigations Blood bilirubin increased	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Psychiatric disorders Insomnia	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Respiratory, thoracic and mediastinal disorders Throat irritation	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.
Vascular disorders Hypotension	3.3% 1/30 • Number of events 1 • Adverse Events (AEs) reported until the last study visit (up to 5 weeks) and Serious AEs (SAEs) until 30 days after the last study visit (up to 10 weeks). The SAE reporting period begins at the time the ICF is signed by the subject. The SAE reporting period ends 30 days following the last study visit. After last study visit, SAEs that comes to the attention of the Investigator must be reported to the CRO/Sponsor and will be documented in the safety database of the Sponsor only and not in eCRF. A rationale for the assessment of a causal relationship must be provided by the Investigator.

Additional Information

Milica Enoiu

CSL Vifor

Phone: +41 58 851 82 64

Email: milica.enoiu@viforpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place