Trial Outcomes & Findings for Comparison of Deferiprone Delayed Release Tablets and Deferiprone Oral Solution in Healthy Volunteers (NCT NCT02442310)
NCT ID: NCT02442310
Last Updated: 2016-05-13
Results Overview
Maximum measured serum concentration. Blood samples will be collected pre-dose and over a 24-hour interval post-dose
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
20 participants
Primary outcome timeframe
24-hour interval
Results posted on
2016-05-13
Participant Flow
Participant milestones
| Measure |
Healthy Volunteers
Subjects were randomized to receive the following four treatments in different orders, with a 7-day washout period between treatments:
* Deferiprone delayed release tablets under fed conditions.
* Deferiprone delayed release tablets under fasting conditions.
* Deferiprone delayed release tablets administered as half-tablets, under fed conditions.
* Deferiprone oral solution under fasting conditions
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Healthy Volunteers
Subjects were randomized to receive the following four treatments in different orders, with a 7-day washout period between treatments:
* Deferiprone delayed release tablets under fed conditions.
* Deferiprone delayed release tablets under fasting conditions.
* Deferiprone delayed release tablets administered as half-tablets, under fed conditions.
* Deferiprone oral solution under fasting conditions
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Comparison of Deferiprone Delayed Release Tablets and Deferiprone Oral Solution in Healthy Volunteers
Baseline characteristics by cohort
| Measure |
Healthy Volunteers
n=20 Participants
Subjects were randomized to receive the following four treatments in different orders, with a 7-day washout period between treatments:
* Deferiprone delayed release tablets under fed conditions.
* Deferiprone delayed release tablets under fasting conditions.
* Deferiprone delayed release tablets administered as half-tablets, under fed conditions.
* Deferiprone oral solution under fasting conditions
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=99 Participants
|
|
Region of Enrollment
Canada
|
20 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 24-hour intervalPopulation: The pharmacokinetics population included all subjects who provided evaluable data for at least one of the comparisons of interest
Maximum measured serum concentration. Blood samples will be collected pre-dose and over a 24-hour interval post-dose
Outcome measures
| Measure |
Delayed Release, Fed Conditions
n=18 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation administered following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, administered following a 10-hour fast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release Half-tablets
n=18 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Oral Solution, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone oral solution, administered following a 10-hour fast
Deferiprone oral solution: Deferiprone 100 mg/mL oral solution
|
|---|---|---|---|---|
|
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Cmax for serum deferiprone
|
8.05 μg/mL
Standard Deviation 2.85
|
8.21 μg/mL
Standard Deviation 2.18
|
7.43 μg/mL
Standard Deviation 2.00
|
16.71 μg/mL
Standard Deviation 4.54
|
|
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Cmax for serum deferiprone 3-O-glucuronide
|
15.77 μg/mL
Standard Deviation 3.80
|
19.27 μg/mL
Standard Deviation 3.73
|
15.40 μg/mL
Standard Deviation 4.33
|
21.87 μg/mL
Standard Deviation 4.34
|
PRIMARY outcome
Timeframe: 24-hour intervalPopulation: The pharmacokinetics population included all subjects who provided evaluable data for at least one of the comparisons of interest
Time to maximum observed serum concentration. Blood samples will be collected pre-dose and over a 24-hour interval post-dose
Outcome measures
| Measure |
Delayed Release, Fed Conditions
n=18 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation administered following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, administered following a 10-hour fast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release Half-tablets
n=18 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Oral Solution, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone oral solution, administered following a 10-hour fast
Deferiprone oral solution: Deferiprone 100 mg/mL oral solution
|
|---|---|---|---|---|
|
Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Tmax for serum deferiprone
|
3.93 Hour
Standard Deviation 1.77
|
2.05 Hour
Standard Deviation 0.60
|
3.49 Hour
Standard Deviation 1.68
|
0.52 Hour
Standard Deviation 0.16
|
|
Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Tmax for serum deferiprone 3-O-glucuronide
|
4.63 Hour
Standard Deviation 1.48
|
3.03 Hour
Standard Deviation 0.52
|
4.75 Hour
Standard Deviation 1.87
|
1.87 Hour
Standard Deviation 0.34
|
PRIMARY outcome
Timeframe: 24-hour intervalPopulation: The pharmacokinetics population included all subjects who provided evaluable data for at least one of the comparisons of interest
Area under the serum concentration time curve extrapolated to infinity. Blood samples will be collected pre-dose and over a 24-hour interval post-dose
Outcome measures
| Measure |
Delayed Release, Fed Conditions
n=17 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation administered following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, administered following a 10-hour fast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release Half-tablets
n=17 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Oral Solution, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone oral solution, administered following a 10-hour fast
Deferiprone oral solution: Deferiprone 100 mg/mL oral solution
|
|---|---|---|---|---|
|
AUC0-∞for Serum Deferiprone and Deferiprone 3-O-glucuronide
AUC0-∞ for serum deferiprone
|
36.70 ug*h/mL
Standard Deviation 9.37
|
35.77 ug*h/mL
Standard Deviation 8.74
|
37.18 ug*h/mL
Standard Deviation 9.72
|
40.61 ug*h/mL
Standard Deviation 10.72
|
|
AUC0-∞for Serum Deferiprone and Deferiprone 3-O-glucuronide
AUC0-∞ for serum deferiprone 3-O-glucuronide
|
97.52 ug*h/mL
Standard Deviation 11.47
|
100.44 ug*h/mL
Standard Deviation 13.49
|
98.23 ug*h/mL
Standard Deviation 10.64
|
101.34 ug*h/mL
Standard Deviation 13.99
|
SECONDARY outcome
Timeframe: Throughout the trial, from the time of the first dose until the last study visit (Day 30 or early termination)Population: The safety population included all subjects who received at least one of the investigational products under study.
Number of subjects with AEs, by frequency, severity, time to onset, duration, and relatedness to study product. AEs will include clinically significant changes from baseline in vital signs, 12-lead ECG, physical examinations, and laboratory tests.
Outcome measures
| Measure |
Delayed Release, Fed Conditions
n=18 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation administered following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release, Fasting Conditions
n=18 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, administered following a 10-hour fast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release Half-tablets
n=19 Participants
A single 1200 mg dose of deferiprone delayed release tablet formulation, following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Oral Solution, Fasting Conditions
n=17 Participants
A single 1200 mg dose of deferiprone oral solution, administered following a 10-hour fast
Deferiprone oral solution: Deferiprone 100 mg/mL oral solution
|
|---|---|---|---|---|
|
Number of Subjects With Adverse Events (AEs)
|
3 participants
|
4 participants
|
5 participants
|
6 participants
|
Adverse Events
Delayed Release, Fed Conditions
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Delayed Release, Fasting Conditions
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Delayed Release Half-tablets
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Oral Solution, Fasting Conditions
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Delayed Release, Fed Conditions
n=18 participants at risk
A single 1200 mg dose of deferiprone delayed release tablet formulation administered following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release, Fasting Conditions
n=18 participants at risk
A single 1200 mg dose of deferiprone delayed release tablet formulation, administered following a 10-hour fast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Delayed Release Half-tablets
n=19 participants at risk
A single 1200 mg dose of deferiprone delayed release tablet formulation, following a high-fat breakfast
Deferiprone delayed release tablet formulation: Deferiprone 600 mg delayed release tablet formulation
|
Oral Solution, Fasting Conditions
n=17 participants at risk
A single 1200 mg dose of deferiprone oral solution, administered following a 10-hour fast
Deferiprone oral solution: Deferiprone 100 mg/mL oral solution
|
|---|---|---|---|---|
|
Injury, poisoning and procedural complications
Vessel Puncture Site Haematoma
|
11.1%
2/18 • Number of events 2
|
5.6%
1/18 • Number of events 1
|
0.00%
0/19
|
5.9%
1/17 • Number of events 1
|
|
Injury, poisoning and procedural complications
Face Injury
|
5.6%
1/18 • Number of events 1
|
0.00%
0/18
|
0.00%
0/19
|
0.00%
0/17
|
|
Injury, poisoning and procedural complications
Vessel Puncture Site Bruise
|
0.00%
0/18
|
0.00%
0/18
|
0.00%
0/19
|
5.9%
1/17 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
5.9%
1/17 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
5.9%
1/17 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
5.9%
1/17 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
0.00%
0/17
|
|
Nervous system disorders
Headache
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
5.9%
1/17 • Number of events 1
|
|
Nervous system disorders
Somnolence
|
0.00%
0/18
|
0.00%
0/18
|
10.5%
2/19 • Number of events 2
|
0.00%
0/17
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
5.9%
1/17 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
0.00%
0/18
|
5.6%
1/18 • Number of events 1
|
5.3%
1/19 • Number of events 1
|
0.00%
0/17
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
0.00%
0/17
|
|
Investigations
Lipase Increased
|
0.00%
0/18
|
5.6%
1/18 • Number of events 1
|
5.3%
1/19 • Number of events 1
|
0.00%
0/17
|
|
Investigations
Amylase Increased
|
0.00%
0/18
|
0.00%
0/18
|
5.3%
1/19 • Number of events 1
|
0.00%
0/17
|
|
General disorders
Paresthesia Mucosal
|
0.00%
0/18
|
0.00%
0/18
|
0.00%
0/19
|
5.9%
1/17 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/18
|
5.6%
1/18 • Number of events 1
|
0.00%
0/19
|
0.00%
0/17
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor retained title to and the right to publish all documentation, records, raw data, specimens or other work product generated in connection with the trial. Such publications shall not be made without the prior written consent of Sponsor. Neither Party will use the other Party's name in connection with any publication or promotion without the other Party's prior written consent. However, Sponsor has the right to publish appropriate information in order to satisfy regulatory requirements.
- Publication restrictions are in place
Restriction type: OTHER