Trial Outcomes & Findings for Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease (NCT NCT01835496)

Results Overview

Cmax (maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

10-hour interval

Results posted on

2015-07-24

Participant Flow

Participant milestones

Participant milestones
Measure	Ferriprox A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Age, Categorical <=18 years	0 Participants n=39 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=39 Participants
Age, Categorical >=65 years	0 Participants n=39 Participants
Age, Continuous	33.0 years STANDARD_DEVIATION 5.9 • n=39 Participants
Sex: Female, Male Female	5 Participants n=39 Participants
Sex: Female, Male Male	3 Participants n=39 Participants
Region of Enrollment Canada	8 participants n=39 Participants

PRIMARY outcome

Timeframe: 10-hour interval

Cmax (maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.

Outcome measures

Outcome measures
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide Cmax for serum deferiprone 3-O-glucuronide	32.95 μg/mL Standard Deviation 11.85
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide Cmax for serum deferiprone	17.56 μg/mL Standard Deviation 5.76

PRIMARY outcome

Timeframe: 10-hour interval

Tmax (time to the maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. The results of the Tmax parameter are reported as the median and range (other parameters are reported as mean and standard deviation).

Outcome measures

Outcome measures
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Tmax for Deferiprone and Deferiprone 3-O-glucuronide Tmax for serum seferiprone	1.000 hr Interval 0.5 to 2.5
Tmax for Deferiprone and Deferiprone 3-O-glucuronide Tmax for serum deferiprone 3-O-glucuronide	2.750 hr Interval 1.33 to 3.0

PRIMARY outcome

Timeframe: 10-hour interval

AUC0-∞ (area under the curve, zero to infinity) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.

Outcome measures

Outcome measures
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide AUC0-∞ for serum deferiprone	43.37 µg*hr/mL Standard Deviation 5.39
AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide AUC0-∞ for serum deferiprone 3-O-glucuronide	142.7 µg*hr/mL Standard Deviation 47.02

PRIMARY outcome

Timeframe: 10-hour interval

T1/2 (apparent terminal elimination half-life) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.

Outcome measures

Outcome measures
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide T1/2 for serum deferiprone	1.458 hr Standard Deviation 0.207
T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide T1/2 for serum deferiprone 3-O-glucuronide	1.575 hr Standard Deviation 0.217

SECONDARY outcome

Timeframe: From Day 1 (Dosing) to Day 7 plus/minus 3 days (Follow-up)

Outcome measures

Outcome measures
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Frequency of Adverse Events	2 participants

SECONDARY outcome

Timeframe: From Day 1 (Dosing) to Day 30 post-dose

Outcome measures

Outcome measures
Measure	Ferriprox n=8 Participants A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Frequency of Serious Adverse Events	0 participants

Adverse Events

Ferriprox

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ferriprox n=8 participants at risk A single dose of 1500 mg of Ferriprox (three 500 mg tablets) administered under fasting conditions
Gastrointestinal disorders Abdominal Pain	12.5% 1/8 • Number of events 1 • From the time of dosing to 7 ±3 days post-dose
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	12.5% 1/8 • Number of events 1 • From the time of dosing to 7 ±3 days post-dose
Nervous system disorders Headache	12.5% 1/8 • Number of events 1 • From the time of dosing to 7 ±3 days post-dose
General disorders Fatigue	12.5% 1/8 • Number of events 1 • From the time of dosing to 7 ±3 days post-dose
General disorders Fever	12.5% 1/8 • Number of events 1 • From the time of dosing to 7 ±3 days post-dose

Additional Information

Caroline Fradette, PhD

ApoPharma Inc.

Phone: 416-401-7543

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee All unpublished information given to the PI by ApoPharma shall not be published or disclosed to a third party without the prior written consent of ApoPharma. The data generated by this study are considered confidential information and the property of ApoPharma. This confidential information may be published only in collaboration with participating personnel from ApoPharma or upon ApoPharma written consent to publish the article.
Publication restrictions are in place

Restriction type: OTHER