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Clinical Trials/NCT06726863
NCT06726863
Completed
Phase 1

A Phase 1, Randomized, Open-label Study to Characterize the Pharmacokinetics, Pharmacodynamics, and Safety of Vamifeport After Multiple Oral Administration of One Immediate-release Formulation and After Single and Multiple Oral Administration of Two Prolonged-release Formulations in Healthy Adult Subjects

CSL Behring1 site in 1 country22 target enrollmentNovember 22, 2024

Overview

Phase
Phase 1
Intervention
Vamifeport IRF
Conditions
Healthy Volunteers
Sponsor
CSL Behring
Enrollment
22
Locations
1
Primary Endpoint
Plasma concentration-time course profiles of vamifeport
Status
Completed
Last Updated
10 months ago

Overview

Brief Summary

This is a phase 1, single-center, randomized, open-label study to characterize the pharmacokinetics (PK), pharmacodynamics (PD), and safety of vamifeport after multiple oral administrations of one immediate-release (IR) formulation and after single and multiple oral administrations of two prolonged-release (PR) formulation in healthy adult participants.

Registry
clinicaltrials.gov
Start Date
November 22, 2024
End Date
February 1, 2025
Last Updated
10 months ago
Study Type
Interventional
Study Design
Sequential
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Aged greater than or equal to (\>=) 18 to less than or equal to (\<=) 60 years when providing written informed consent.
  • Healthy, as determined by the investigator based on review of defined assessments during Screening.
  • Body weight between 50 and 100 kilograms (kg) (inclusive) and body mass index within the range 18.0 to 32.0 kg per meter squared (kg/m2) (inclusive) at Screening and Day -1.

Exclusion Criteria

  • Any clinically relevant abnormal 12-lead ECG finding at Screening or Day -1 (as deemed by the investigator).
  • Serum ferritin of \< 30 nanograms per milliliter (ng/mL) or \> 300 ng/mL for assigned male at birth (AMAB) subjects or \<16 ng/mL or \> 300 ng/mL for assigned female at birth (AFAB) subjects at Screening or Day -
  • Hemoglobin \< 13 grams per deciliter (g/dL) (8.1 millimoles per liter \[mmol/L\]) for AMAB subjects or 12 g/dL (7.5 mmol/L) for AFAB subjects at Screening or Day -
  • Blood draw or donation of blood (\>= 450 mL) within 3 months before Screening, plasma from 2 weeks before Screening, or platelets from 6 weeks before Screening.

Arms & Interventions

Treatment Period 1: Vamifeport IR Formulation Dose Level 1

Participants will receive multiple doses of Vamifeport Immediate-release (IR) formulation at Dose level 1.

Intervention: Vamifeport IRF

Treatment Period 2: Vamifeport PR1 Dose Level 2

Participants will receive multiple doses of Vamifeport Prolonged-release formulation 1 (PR1) at Dose level 2.

Intervention: Vamifeport PR1

Treatment Period 2: Vamifeport PR2 Dose Level 2

Participants will receive multiple doses of Vamifeport Prolonged-release formulation 2 (PR2) at Dose level 2.

Intervention: Vamifeport PR2

Treatment Period 3: Vamifeport PR1 Dose Level 3

Participants will receive a single dose of Vamifeport PR1 at Dose level 3.

Intervention: Vamifeport PR1

Treatment Period 3: Vamifeport PR2 Dose Level 3

Participants will receive a single dose of Vamifeport PR2 at Dose level 3.

Intervention: Vamifeport PR2

Treatment Period 4: Vamifeport PR1 Dose Level 3

Participants will receive a single dose of Vamifeport PR1 at Dose level 3.

Intervention: Vamifeport PR1

Treatment Period 4: Vamifeport PR2 Dose Level 3

Participants will receive a single dose of Vamifeport PR2 at Dose level 3.

Intervention: Vamifeport PR2

Outcomes

Primary Outcomes

Plasma concentration-time course profiles of vamifeport

Time Frame: Treatment Period (TP) 2: Before and after dosing on Day 4 (up to 12 hours), Day 8 (up to 48 hours), Before dosing on Day 5, 6, 7 TP 3: Before and after dosing on Day 13 (up to 48 hours) TP 4: Before and after dosing on Day 16 (up to 48 hours)

Maximum plasma concentration (Cmax) of first and last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP2: Before, and up to 48 hours after, both the first and the last dose

Time to reach Cmax (Tmax) of first and last dose vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after, both the first and the last dose

Area under the plasma concentration curve from time zero to 12 hours (AUC0-12) of first and last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after, both the first and the last dose

Trough concentration (Ctrough) of first dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: Before and up to 24 hours after the first dose in TP2

AUC from time zero to infinity (AUC0-inf) of last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after last dose

AUC from time zero to time tlast (AUC0-last) of last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after last dose

AUC from time zero to 8 hours (AUC0-8) and 24 hours (AUC0-24) of last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 8 and 24 hours after last dose

Plasma concentration at 12 hours (Conc [t=12]) of last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before and up to 12 hours after last dose

Apparent clearance (CL/F) of last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after last dose

Apparent volume of distribution at steady state (Vss/F) of last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after last dose

Accumulation ratio (Rac) of Cmax between first and last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after first and last dose

Rac(Ctrough/Conc[t=12]) between first and last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 12 hours after first and last dose

Rac (AUC0-12) between first and last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 12 hours after first and last dose

Half-life (t1/2) after last dose of vamifeport PR1 and PR2 in Treatment Period 2

Time Frame: TP 2: Before, and up to 48 hours after last dose

Cmax of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

Tmax of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

AUC0-last of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

AUC0-inf of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

AUC0-8, AUC0-12 and AUC0-24 of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 8, 12 and 24 hours after dosing, in TP3 and TP4

Plasma Concentration at 8 hours (Conc [t=8]), 12 hours (Conc [t=12]) and 24 hours (Conc [t=24]) of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 8, 12 and 24 hours after dosing, in TP3 and TP4

CL/F of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

t1/2 of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

Apparent volume of distribution (Vz/F) of vamifeport PR1 and PR2 in Treatment Period 3 and 4

Time Frame: Before and up to 48 hours after dosing, in TP3 and TP4

Secondary Outcomes

  • Percentage of participants with TEAE, AE by severity, AE related to vamifeport, and serious AE(Up to 25 days after treatment)
  • Number of participants with clinically significant change from Baseline in clinical laboratory safety tests, 12 lead Electrocardiogram (ECG), and vital signs(At baseline and up to 25 days after treatment)
  • AUC0-12 of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 12 hours after dosing, in TP3 and TP4)
  • AUC0-inf of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 48 hours after dosing, in TP3 and TP4)
  • Number of participants with treatment-emergent (TE): adverse event (AE), AE by severity, AE related to vamifeport, and serious AE(Up to 25 days after treatment)
  • AUC0-24 of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 24 hours after dosing, in TP3 and TP4)
  • Conc (t=12) of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 12 hours after dosing, in TP3 and TP4)
  • Conc (t=24) of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 24 hours after dosing, in TP3 and TP4)
  • Cmax of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 48 hours after dosing, in TP3 and TP4)
  • Tmax of vamifeport PR1 and PR2 in Treatment Period 3 and 4(Before and up to 48 hours after dosing, in TP3 and TP4)
  • Absolute values of serum iron(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Absolute values of transferrin saturation (TSAT)(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Change from baseline of serum iron(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Change from baseline of TSAT(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Maximum percentage change from baseline (Emax) of serum iron(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Emax of TSAT(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Time to Emax (TEmax) of serum iron(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • TEmax of TSAT(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Time below baseline of serum iron(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • Time below baseline of TSAT(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • AUC below baseline of serum iron(Before and up to 48 hours after dosing in TP2, TP3 and TP4)
  • AUC below baseline of TSAT(Before and up to 48 hours after dosing in TP2, TP3 and TP4)

Study Sites (1)

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