Trial Outcomes & Findings for Givinostat and Metabolites Pharmacokinetics in Urine and Plasma (Part 3) (NCT NCT05860114)

NCT ID: NCT05860114

Last Updated: 2025-01-24

Results Overview

Cmax is the Maximum Observed Plasma Concentration. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 22 blood samples were collected as follows: \- 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Days 1, for the determination of Givinostat and its metabolites plasma concentrations using a validated LC-MS/MS analytical method. Summary Statistics for the main PK parameters, like Cmax, Following Single-Dose Administration of Givinostat (Day 1) are reported.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1.

Results posted on

2025-01-24

Participant Flow

A total of 11 subjects were screened for participation. Eight (8) subjects (2 women and 6 men) were admitted to study Part 3.

Participant milestones

Participant milestones
Measure
Givinostat (Single-dose and Multiple-dose)
On Day 1 and Day 13, Givinostat 50 mg as oral suspension was administered as a single dose, in the morning. On Days 5-12 Givinostat 50 mg as oral suspension was administered twice-daily, in the morning and in the evening, as a multiple dose. Givinostat: Drug: ITF2357 Givinostat 10mg/mL Dose: 10mg/mL; Dosage form: oral suspension
Overall Study
STARTED
8
Overall Study
Safety Analysis Population
8
Overall Study
Single-dose PK Population
8
Overall Study
Multiple-dose PK Population
8
Overall Study
COMPLETED
8
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Givinostat and Metabolites Pharmacokinetics in Urine and Plasma (Part 3)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Givinostat (Single-dose or Multiple-dose)
n=8 Participants
On day 1, givinostat 50 mg as oral suspension was administered as a single dose, in the morning. On Days 5-12 givinostat 50 mg as oral suspension was administered twice-daily, in the morning and in the evening, as a multiple dose. On day 13 givinostat 50 mg as oral suspension was administered as a single dose, in the morning. Givinostat: Drug: ITF2357 Givinostat 10mg/mL Dose: 10mg/mL; Dosage form: oral suspension
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
8 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
30 years
STANDARD_DEVIATION 6.4 • n=5 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
Sex: Female, Male
Male
6 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
7 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
Portugal
8 participants
n=5 Participants

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose Pharmacokinetic Analysis Population.

Cmax is the Maximum Observed Plasma Concentration. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 22 blood samples were collected as follows: \- 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Days 1, for the determination of Givinostat and its metabolites plasma concentrations using a validated LC-MS/MS analytical method. Summary Statistics for the main PK parameters, like Cmax, Following Single-Dose Administration of Givinostat (Day 1) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma Pharmacokinetic (PK) Parameters of Givinostat, Following First Single-dose Administration of Givinostat: Cmax
48.06 ng/mL
Geometric Coefficient of Variation 25.8
7.34 ng/mL
Geometric Coefficient of Variation 45.7
132.20 ng/mL
Geometric Coefficient of Variation 40.2
88.77 ng/mL
Geometric Coefficient of Variation 16.4
15.38 ng/mL
Geometric Coefficient of Variation 13.8

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose Pharmacokinetic Analysis Population.

Tmax is the Time to Maximum Observed Concentration.The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 22 blood samples were collected as follows: \- 22 blood samples at predose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Days 1, for the determination of Givinostat and its metabolites plasma concentrations. Summary Statistics for the main PK parameters, like tmax, Following Single-Dose Administration of Givinostat (Day 1) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose Administration of Givinostat: Tmax
1.50 hours
Interval 1.0 to 3.5
5.50 hours
Interval 5.0 to 12.0
3.50 hours
Interval 2.0 to 4.0
9.00 hours
Interval 8.0 to 12.0
10.00 hours
Interval 8.0 to 12.0

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1 and on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1 and Day 13 in the morning), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Pharmacokinetic Analysis Population.

Ctrough is the Pre-dose Plasma Concentration. On Day 1 and Day 13, Givinostat 50 mg as oral suspension was administered as a single dose, in the morning, following an overnight fasting of at least 8 hours and subjects remained fasted until at least 4 hours post-dose. From Day 5 to Day 12 Givinostat 50 mg as oral suspension was administered twice daily. A total of 48 blood samples were collected: * 22 during single-dose treatment (Day 1, at pre-dose and at the timepoints indicated in the timeframe post-dose) and * 26 during the multiple dose treatment (Days 5-12 plus single-dose treatment at Day 13). Of these 26 samples: 22 as per Day 1, and 4 before the morning dose of IMP on Days 9, 10, 11 and 12. PK plasma parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-Dose Administration of Givinostat: Ctrough
single dose (Day 1)
6.75 ng/mL
Geometric Coefficient of Variation 22.3
4.14 ng/mL
Geometric Coefficient of Variation 42.6
28.47 ng/mL
Geometric Coefficient of Variation 53.3
81.58 ng/mL
Geometric Coefficient of Variation 14.9
14.75 ng/mL
Geometric Coefficient of Variation 11.1
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-Dose Administration of Givinostat: Ctrough
multiple dose (Day 13)
13.16 ng/mL
Geometric Coefficient of Variation 28.7
10.18 ng/mL
Geometric Coefficient of Variation 50.8
56.12 ng/mL
Geometric Coefficient of Variation 60.6
279.96 ng/mL
Geometric Coefficient of Variation 15.6
50.11 ng/mL
Geometric Coefficient of Variation 18.8

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1 and on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

AUC0-inf is the AUC from Time Zero to Infinity. On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose, in the morning, following an overnight fasting of at least 8 hours and subjects remained fasted until at least 4 hours post-dose. From Day 5 to Day 12 givinostat 50 mg as oral suspension was administered twice daily. A total of 48 blood samples were collected: * 22 during single-dose treatment (Day 1, at pre-dose and at the timepoints indicated in the timeframe post-dose) and * 26 during the multiple dose treatment (Days 5-12 plus single-dose treatment at Day 13). Of these 26 samples: 22 as per Day 1, and 4 before the morning dose of IMP on Days 9, 10, 11 and 12. PK plasma parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: AUC0-inf.
Single-dose (Day 1)
305.72 h*ng/mL
Geometric Coefficient of Variation 21.0
99.01 h*ng/mL
Geometric Coefficient of Variation 49.1
1239.34 h*ng/mL
Geometric Coefficient of Variation 47.8
2431.31 h*ng/mL
Geometric Coefficient of Variation 12.1
457.95 h*ng/mL
Geometric Coefficient of Variation 13.4
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: AUC0-inf.
Multiple-dose (Day 13)
562.42 h*ng/mL
Geometric Coefficient of Variation 17.5
337.71 h*ng/mL
Geometric Coefficient of Variation 53.2
2754.89 h*ng/mL
Geometric Coefficient of Variation 77.8
9526.56 h*ng/mL
Geometric Coefficient of Variation 30.2
1715.62 h*ng/mL
Geometric Coefficient of Variation 23.0

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1 and on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1 and Day 13) and multiple-dose administration (From Day 5 to Day 12), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

AUC0-t is the AUC from Time Zero to Last Sampling Time with Quantifiable. On Days 1 and 13, Givinostat 50 mg as oral suspension was administered as a single dose, in the morning, following an overnight fasting of at least 8 h and subjects remained fasted until at least 4 h post-dose. From Day 5 to Day 12 Givinostat 50 mg as oral suspension was administered twice daily. 48 blood samples were collected in total: * 22 during single-dose treatment (Day 1, at pre-dose and at the timepoints indicated in the timeframe post-dose), * 26 during the multiple dose treatment (Days 5-12 plus single-dose treatment at Day 13). Of these 26 samples: 22 as per Day 1, and 4 before the morning dose of IMP on Days 9, 10, 11 and 12. PK plasma parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: AUC0-t.
Single-dose (Day 1)
300.45 h*ng/mL
Geometric Coefficient of Variation 21.3
93.30 h*ng/mL
Geometric Coefficient of Variation 51.0
1223.60 h*ng/mL
Geometric Coefficient of Variation 47.5
2345.60 h*ng/mL
Geometric Coefficient of Variation 12.7
439.59 h*ng/mL
Geometric Coefficient of Variation 13.0
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: AUC0-t.
Multiple-dose (Day 13)
554.78 h*ng/mL
Geometric Coefficient of Variation 17.5
328.36 h*ng/mL
Geometric Coefficient of Variation 53.9
2609.03 h*ng/mL
Geometric Coefficient of Variation 68.2
9262.28 h*ng/mL
Geometric Coefficient of Variation 27.4
1681.26 h*ng/mL
Geometric Coefficient of Variation 21.8

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose Pharmacokinetic Analysis Population.

AUC0-τ is the area under the plasma concentration versus time curve. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 22 blood samples were collected as follows: \- 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Days 1, for the determination of Givinostat and its metabolites plasma concentrations. Summary Statistics for the main PK parameters, like AUC0-τ, Following Single-Dose Administration of Givinostat (Day 1) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose Administration of Givinostat: AUC0-τ
245.73 h*ng/mL
Geometric Coefficient of Variation 23.2
52.48 h*ng/mL
Geometric Coefficient of Variation 42.2
806.71 h*ng/mL
Geometric Coefficient of Variation 40.6
681.56 h*ng/mL
Geometric Coefficient of Variation 25.8
99.71 h*ng/mL
Geometric Coefficient of Variation 23.1

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1 and on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1 and Day 13) and multiple-dose administration (From Day 5 to Day 12), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

Area under the curve (AUC) is used to describe the total exposure to a drug. The total AUC (AUC0-∞) is the area under the curve from time 0 extrapolated to infinite time. %AUCextrap is the Residual Area or Percentage of Extrapolated Part of AUC0-∞. On Days 1 and 13, givinostat 50 mg as oral suspension was administered as a single dose, in the morning, following an overnight fasting of at least 8 h and subjects remained fasted until at least 4 h post-dose. From Day 5 to Day 12 Givinostat 50 mg as oral suspension was administered twice daily. A total of 48 samples were collected as already described. PK plasma parameters following single oral dose administration of givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: %AUCextrap
Single-dose (Day 1)
1.69 percentage of AUC0-∞
Geometric Coefficient of Variation 21.7
5.39 percentage of AUC0-∞
Geometric Coefficient of Variation 39.8
0.93 percentage of AUC0-∞
Geometric Coefficient of Variation 97.7
3.46 percentage of AUC0-∞
Geometric Coefficient of Variation 20.9
3.96 percentage of AUC0-∞
Geometric Coefficient of Variation 16.3
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: %AUCextrap
Multiple-dose (Day 13)
1.33 percentage of AUC0-∞
Geometric Coefficient of Variation 20.6
2.56 percentage of AUC0-∞
Geometric Coefficient of Variation 45.2
2.13 percentage of AUC0-∞
Geometric Coefficient of Variation 220.0
1.84 percentage of AUC0-∞
Geometric Coefficient of Variation 112.1
1.53 percentage of AUC0-∞
Geometric Coefficient of Variation 82.8

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1 and on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1 and Day 13) and multiple-dose administration (From Day 5 to Day 12), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

λz is the Apparent Terminal Elimination Rate Constant. On Day 1 and Day 13, Givinostat 50 mg as oral suspension was administered as a single dose, in the morning, following an overnight fasting of at least 8 hours and subjects remained fasted until at least 4 hours post-dose. From Day 5 to Day 12 Givinostat 50 mg as oral suspension was administered twice daily. A total of 48 blood samples were collected: * 22 during single-dose treatment (Day 1, at pre-dose and at the timepoints indicated in the timeframe post-dose) and * 26 during the multiple dose treatment (Days 5-12 plus single-dose treatment at Day 13). Of these 26 samples: 22 as per Day 1, and 4 before the morning dose of IMP on Days 9, 10, 11 and 12. PK plasma parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: λz
Multiple-dose (Day 13)
0.067 1/hour
Geometric Coefficient of Variation 24.8
0.064 1/hour
Geometric Coefficient of Variation 22.5
0.037 1/hour
Geometric Coefficient of Variation 71.6
0.047 1/hour
Geometric Coefficient of Variation 49.8
0.052 1/hour
Geometric Coefficient of Variation 31.5
Plasma PK Parameters of Givinostat, Following First Single-dose and Following Multiple-dose Administration of Givinostat: λz
Single-dose (Day 1)
0.097 1/hour
Geometric Coefficient of Variation 15.6
0.094 1/hour
Geometric Coefficient of Variation 33.0
0.062 1/hour
Geometric Coefficient of Variation 34.2
0.060 1/hour
Geometric Coefficient of Variation 15.2
0.055 1/hour
Geometric Coefficient of Variation 27.8

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose Pharmacokinetic Analysis Population.

t1/2 is the Apparent Terminal Half-Life. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 22 blood samples were collected as follows: \- 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Days 1, for the determination of Givinostat and its metabolites plasma concentrations. Summary Statistics for the main PK parameters, like t1/2, Following Single-Dose Administration of Givinostat (Day 1) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose Administration of Givinostat: t1/2
7.16 hours
Geometric Coefficient of Variation 15.7
7.40 hours
Geometric Coefficient of Variation 32.8
11.21 hours
Geometric Coefficient of Variation 34.4
11.65 hours
Geometric Coefficient of Variation 15.4
12.57 hours
Geometric Coefficient of Variation 27.8

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 1.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose Pharmacokinetic Analysis Population.

Vd/F is the Apparent volume of distribution. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 22 blood samples were collected as follows: \- 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Days 1, for the determination of Givinostat and its metabolites plasma concentrations. Summary Statistics for the main PK parameters, like Vd/F, Following Single-Dose Administration of Givinostat (Day 1) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following First Single-dose Administration of Givinostat: Vd/F
1690.43 Liters
Geometric Coefficient of Variation 21.7
0000 Liters
Geometric Coefficient of Variation 0000
0000 Liters
Geometric Coefficient of Variation 0000
0000 Liters
Geometric Coefficient of Variation 0000
0000 Liters
Geometric Coefficient of Variation 0000

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1 and Day 13) and multiple-dose administration (from day 5 to Day 12), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

Cmax,ss is the Maximum Observed Plasma Concentration at steady state. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 26 blood samples were collected as follows: * 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Day 13, for the determination of Givinostat and its metabolites plasma concentrations. * 4 blood samples before the morning dose of on Days 9, 10, 11 and 12, for the determination of pre-dose plasma concentration of Givinostat and its metabolites. Summary Statistics for Cmax,ss, Following Multiple-Dose of Givinostat (Days 5-12 plus single-dose at Day 13, with values calculated at Day 13) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following Multiple-dose Administration of Givinostat: Cmax,ss
75.50 ng/mL
Geometric Coefficient of Variation 27.0
20.52 ng/mL
Geometric Coefficient of Variation 38.6
217.29 ng/mL
Geometric Coefficient of Variation 38.1
306.60 ng/mL
Geometric Coefficient of Variation 15.2
55.37 ng/mL
Geometric Coefficient of Variation 20.3

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Multiple-Dose Pharmacokinetic Analysis Population.

Tmax,ss is the Time of occurrence of Cmax,ss. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported in this platform). A total of 26 blood samples were collected as follows: * 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Day 13, for the determination of Givinostat and its metabolites plasma concentrations. * 4 blood samples before the morning dose of on Days 9, 10, 11 and 12, for the determination of pre-dose plasma concentration of Givinostat and its metabolites. Summary Statistics for Tmax,ss, Following Multiple-Dose of Givinostat (Days 5-12 plus single-dose at Day 13, with values calculated at Day 13) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following Multiple-dose Administration of Givinostat: Tmax,ss
1.50 hours
Interval 1.5 to 3.0
5.50 hours
Interval 5.0 to 6.0
3.50 hours
Interval 2.05 to 4.0
10.00 hours
Interval 4.0 to 12.0
9.00 hours
Interval 6.0 to 10.0

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Multiple-Dose Pharmacokinetic Analysis Population.

AUC0-τ,ss is the AUC during a Dosing Interval at steady state. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 26 blood samples were collected as follows: * 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field the administration of Givinostat (post-dose), on Day 13, for the determination of Givinostat and its metabolites plasma concentrations. * 4 blood samples before the morning dose of on Days 9, 10, 11 and 12, for the determination of pre-dose plasma concentration of Givinostat and its metabolites. Summary Statistics for AUC0-τ,ss, Following Multiple-Dose of Givinostat (Days 5-12 plus single-dose at Day 13, with values calculated at Day 13) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following Multiple-dose Administration of Givinostat: AUC0-τ,ss
408.55 h*ng/mL
Geometric Coefficient of Variation 16.2
179.63 h*ng/mL
Geometric Coefficient of Variation 41.9
1518.21 h*ng/mL
Geometric Coefficient of Variation 50.5
3395.41 h*ng/mL
Geometric Coefficient of Variation 15.8
586.72 h*ng/mL
Geometric Coefficient of Variation 19.3

PRIMARY outcome

Timeframe: At 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 15, 24, 36, 48, 60, 72, 84 and 96 hours post dose on Day 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Multiple-Dose Pharmacokinetic Analysis Population.

CLss/F is the Apparent total body clearance at steady state. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). A total of 26 blood samples were collected as follows: * 22 blood samples at pre-dose and at the timepoints indicated in the timeframe field after the administration of Givinostat (post-dose), on Day 13, for the determination of Givinostat and its metabolites plasma concentrations. * 4 blood samples before the morning dose of on Days 9, 10, 11 and 12, for the determination of pre-dose plasma concentration of Givinostat and its metabolites. Summary Statistics for CLss/F, Following Multiple-Dose of Givinostat (Day 13) are reported.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Plasma PK Parameters of Givinostat, Following Multiple-dose Administration of Givinostat: CLss/F.
122.38 L/h
Geometric Coefficient of Variation 16.2
0000 L/h
Geometric Coefficient of Variation 0000
0000 L/h
Geometric Coefficient of Variation 0000
00000 L/h
Geometric Coefficient of Variation 00000
00000 L/h
Geometric Coefficient of Variation 00000

PRIMARY outcome

Timeframe: 0-12, 12-24, 24-36, 36-48, 48-72 and 72-96 hours post-dose on days 1 and 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

Rmax is the Maximum urinary excretion rate. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). Urine was collected, beside on Day -1 (baseline), in the following intervals, on Days 1 and 13, for the determination of the amounts of Givinostat and its metabolites excreted in urine: 0h-12h, 12h-24h, 24h-36h, 36h-48h, 48h-72h and 72h-96h post-dose. Givinostat and its metabolites urine levels were measured using a validated LC-MS/MS analytical method. PK urine parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: Rmax.
single-dose (Day 1)
0.051 mg/h
Geometric Coefficient of Variation 50.8
0.015 mg/h
Geometric Coefficient of Variation 25.9
0.006 mg/h
Geometric Coefficient of Variation 44.6
0.276 mg/h
Geometric Coefficient of Variation 25.9
0.107 mg/h
Geometric Coefficient of Variation 23.6
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: Rmax.
multiple-dose (Day 13)
0.059 mg/h
Geometric Coefficient of Variation 39.8
0.043 mg/h
Geometric Coefficient of Variation 30.6
0.009 mg/h
Geometric Coefficient of Variation 83.0
0.692 mg/h
Geometric Coefficient of Variation 23.7
0.319 mg/h
Geometric Coefficient of Variation 28.3

PRIMARY outcome

Timeframe: 0-12, 12-24, 24-36, 36-48, 48-72 and 72-96 hours post-dose on days 1 and 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

tumax is the Time to Rmax. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). Urine was collected, beside on Day -1 (baseline), in the following intervals, on Days 1 and 13, for the determination of the amounts of Givinostat and its metabolites excreted in urine: 0h-12h, 12h-24h, 24h-36h, 36h-48h, 48h-72h and 72h-96h post-dose. Givinostat and its metabolites urine levels were measured using a validated LC-MS/MS analytical method. PK urine parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: Tumax
single-dose (Day 1)
6.00 hours
Interval 6.0 to 6.0
6.00 hours
Interval 6.0 to 18.0
6.00 hours
Interval 6.0 to 6.0
6.00 hours
Interval 6.0 to 18.0
12.00 hours
Interval 6.0 to 18.0
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: Tumax
multiple-dose (Day 13)
6.00 hours
Interval 6.0 to 6.0
6.00 hours
Interval 6.0 to 6.0
6.00 hours
Interval 6.0 to 6.0
12.00 hours
Interval 6.0 to 18.0
18.00 hours
Interval 6.0 to 18.0

PRIMARY outcome

Timeframe: 0-12, 12-24, 24-36, 36-48, 48-72 and 72-96 hours post-dose on days 1 and 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

AmtCUM is the Cumulative amount of drug excreted in urine. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). Urine was collected, beside on Day -1 (baseline), in the following intervals, on Days 1 and 13, for the determination of the amounts of Givinostat and its metabolites excreted in urine: 0h-12h, 12h-24h, 24h-36h, 36h-48h, 48h-72h and 72h-96h post-dose. Givinostat and its metabolites urine levels were measured using a validated LC-MS/MS analytical method. PK urine parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: AmtCUM
multiple-dose (Day 13)
1.037 mg
Geometric Coefficient of Variation 32.6
1.201 mg
Geometric Coefficient of Variation 39.8
0.184 mg
Geometric Coefficient of Variation 85.9
26.627 mg
Geometric Coefficient of Variation 20.2
12.208 mg
Geometric Coefficient of Variation 18.3
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: AmtCUM
single-dose (Day 1)
0.782 mg
Geometric Coefficient of Variation 39.2
0.417 mg
Geometric Coefficient of Variation 24.9
0.093 mg
Geometric Coefficient of Variation 49.6
9.179 mg
Geometric Coefficient of Variation 15.6
3.985 mg
Geometric Coefficient of Variation 18.6

PRIMARY outcome

Timeframe: 0-12, 12-24, 24-36, 36-48, 48-72 and 72-96 hours post-dose on days 1 and 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

AURC0-t is the Area under the urine excretion curve from time zero to last measurable observed excretion rate. The unit of measure is mg because the value measured is an amount. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). Urine was collected, beside on Day -1 (baseline), in the following intervals, on Days 1 and 13, for the determination of the amounts of Givinostat and its metabolites excreted in urine: 0h-12h, 12h-24h, 24h-36h, 36h-48h, 48h-72h and 72h-96h post-dose. Givinostat and its metabolites urine levels were measured using a validated LC-MS/MS analytical method. PK urine parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: AURC0-t
single-dose (Day 1)
0.532 mg
Geometric Coefficient of Variation 31.1
0.354 mg
Geometric Coefficient of Variation 27.4
0.063 mg
Geometric Coefficient of Variation 55.3
8.019 mg
Geometric Coefficient of Variation 16.6
3.571 mg
Geometric Coefficient of Variation 19.9
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: AURC0-t
multiple-dose (Day 13)
0.773 mg
Geometric Coefficient of Variation 30.6
1.027 mg
Geometric Coefficient of Variation 41.1
0.142 mg
Geometric Coefficient of Variation 89.0
23.836 mg
Geometric Coefficient of Variation 19.6
10.961 mg
Geometric Coefficient of Variation 18.2

PRIMARY outcome

Timeframe: 0-12, 12-24, 24-36, 36-48, 48-72 and 72-96 hours post-dose on days 1 and 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the Givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

REC% is the Percentage of drug recovered in urine. The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). Urine was collected, beside on Day -1 (baseline), in the following intervals, on Days 1 and 13, for the determination of the amounts of Givinostat and its metabolites excreted in urine: 0h-12h, 12h-24h, 24h-36h, 36h-48h, 48h-72h and 72h-96h post-dose. Givinostat and its metabolites urine levels were measured using a validated LC-MS/MS analytical method. PK urine parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: REC%
single-dose (Day 1)
1.565 Percentage of drug
Geometric Coefficient of Variation 39.1
0.868 Percentage of drug
Geometric Coefficient of Variation 24.9
0.193 Percentage of drug
Geometric Coefficient of Variation 49.8
30.072 Percentage of drug
Geometric Coefficient of Variation 15.6
18.852 Percentage of drug
Geometric Coefficient of Variation 18.6
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: REC%
multiple-dose (Day 13)
2.073 Percentage of drug
Geometric Coefficient of Variation 32.6
2.496 Percentage of drug
Geometric Coefficient of Variation 39.8
0.380 Percentage of drug
Geometric Coefficient of Variation 86.3
87.228 Percentage of drug
Geometric Coefficient of Variation 20.2
57.755 Percentage of drug
Geometric Coefficient of Variation 18.3

PRIMARY outcome

Timeframe: 0-12, 12-24, 24-36, 36-48, 48-72 and 72-96 hours post-dose on days 1 and 13.

Population: Eight (8) subjects were enrolled in Part 3 of the study and provided evaluable pharmacokinetic data for the givinostat single-dose administration (Day 1) and multiple-dose administration (Day 13), without deviations affecting pharmacokinetic interpretation. They constitute the Part 3 Single-Dose and Multiple-Dose Pharmacokinetic Analysis Population, respectively.

The Givinostat metabolites were: ITF2374, ITF2375, ITF2440, ITF2563 (not reported on this platform). Urine was collected, beside on Day -1 (baseline), in the following intervals, on Days 1 and 13, for the determination of the amounts of Givinostat and its metabolites excreted in urine: 0h-12h, 12h-24h, 24h-36h, 36h-48h, 48h-72h and 72h-96h post-dose. Givinostat and its metabolites urine levels were measured using a validated LC-MS/MS analytical method. PK urine parameters following single oral dose administration of Givinostat (Day 1) and following multiple oral dose administration of Givinostat (Days 5-12 plus single-dose treatment at Day 13, with values on multiple-dose treatment reported at Day 13) were assessed.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
n=8 Participants
Givinostat plasma metabolite
ITF2375
n=8 Participants
Givinostat plasma metabolite
ITF2440
n=8 Participants
Givinostat plasma metabolite
ITF2563
n=8 Participants
Givinostat plasma metabolite
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: CLr/F
single-dose (Day 1)
2.604 L/h
Geometric Coefficient of Variation 28.6
4.473 L/h
Geometric Coefficient of Variation 44.1
0.076 L/h
Geometric Coefficient of Variation 26.8
3.913 L/h
Geometric Coefficient of Variation 23.7
9.065 L/h
Geometric Coefficient of Variation 25.8
Urinary PK Parameters of Givinostat Following First Single-dose and Following Multiple-dose of Givinostat: CLr/F
multiple-dose (Day 13)
1.868 L/h
Geometric Coefficient of Variation 25.4
3.656 L/h
Geometric Coefficient of Variation 26.5
0.070 L/h
Geometric Coefficient of Variation 20.0
2.875 L/h
Geometric Coefficient of Variation 18.3
7.261 L/h
Geometric Coefficient of Variation 17.0

SECONDARY outcome

Timeframe: Throughout the study, until 10-14 days after EoS (Day 17), i.e. till Days 27-31

Population: The Safety (SAF) population consisted of all randomized patients who received at least 1 dose of the IMP. The SAF population was analyzed according to the actual treatment received and was used to present results on safety data.

An AE was considered as any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily imply a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The period of observation for the collection of medical occurrences extended from the time when the subject gave Informed Consent until the follow-up visit.

Outcome measures

Outcome measures
Measure
Givinostat
n=8 Participants
On Day 1 and Day 13, givinostat 50 mg as oral suspension was administered as a single dose in the morning. From Day 5 to Day 12, givinostat 50 mg as oral suspension was administered twice a day, in the morning and in the evening.
ITF2374
Givinostat plasma metabolite
ITF2375
Givinostat plasma metabolite
ITF2440
Givinostat plasma metabolite
ITF2563
Givinostat plasma metabolite
Incidence and Severity of Treatment Emergent Adverse Events
mild TEAE
17 number of adverse events
Incidence and Severity of Treatment Emergent Adverse Events
Total TEAE
21 number of adverse events
Incidence and Severity of Treatment Emergent Adverse Events
moderate TEAE
4 number of adverse events
Incidence and Severity of Treatment Emergent Adverse Events
severe TEAE
0 number of adverse events

Adverse Events

Total

Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Total
n=8 participants at risk
The only investigational product administered in this study part, was givinostat, therefore, all TEAEs were selected and reported upon
Blood and lymphatic system disorders
Thrombocytopenia
62.5%
5/8 • Number of events 5 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Cardiac disorders
Bundle branch block right
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Gastrointestinal disorders
Nausea
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Gastrointestinal disorders
Vomiting
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Infections and infestations
Tonsillitis
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Injury, poisoning and procedural complications
Arthropod bite
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Musculoskeletal and connective tissue disorders
Back pain
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Musculoskeletal and connective tissue disorders
Muscle contracture
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Nervous system disorders
Headache
25.0%
2/8 • Number of events 2 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Nervous system disorders
Migrane
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Nervous system disorders
Presyncope
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Respiratory, thoracic and mediastinal disorders
Epistaxis
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Skin and subcutaneous tissue disorders
Dermatitis contact
12.5%
1/8 • Number of events 1 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.
Skin and subcutaneous tissue disorders
Dry skin
37.5%
3/8 • Number of events 3 • Throughout the study and 10-14 days after the End of Study (follow-up visit), i.e. up to day 30-34, all TEAEs, considered related or not related to IMPs, were collected.
End-of-study was defined as the last study procedure on Day 17 or at early discontinuation of subjects administered at least one dose of investigational product. Early termination procedures were performed as soon as possible after subject withdrawal, within 10-14 days after last participation of the subject in the study, whenever possible.

Additional Information

Maurizio Caserini, MD

Italfarmaco SpA

Phone: +39 02 64431

Results disclosure agreements

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