Trial Outcomes & Findings for Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients (NCT NCT00529152)

Results Overview

Number of Adverse Events over 24 weeks

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

100 participants

Primary outcome timeframe

24 Weeks

Results posted on

2009-09-02

Participant Flow

Participant milestones

Participant milestones
Measure	Ferriprox Oral Solution All subjects were administered Ferriprox Oral Solution three times daily for a total daily dose of either 50, 75 or 100 mg/kg/day. Subjects were initiated at a dose of 50 mg/kg/day, but this dose could be increased after two weeks of therapy depending on the subjects' individual needs.
Overall Study STARTED	100
Overall Study COMPLETED	95
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Ferriprox Oral Solution All subjects were administered Ferriprox Oral Solution three times daily for a total daily dose of either 50, 75 or 100 mg/kg/day. Subjects were initiated at a dose of 50 mg/kg/day, but this dose could be increased after two weeks of therapy depending on the subjects' individual needs.
Overall Study Adverse Event	2
Overall Study Withdrawal by Subject	2
Overall Study Lost to Follow-up	1

Baseline Characteristics

Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ferriprox Oral Solution n=100 Participants All subjects were administered Ferriprox Oral Solution three times daily for a total daily dose of either 50, 75 or 100 mg/kg/day. Subjects were initiated at a dose of 50 mg/kg/day, but this dose could be increased after two weeks of therapy depending on the subjects' individual needs.
Age Continuous	5.1 years STANDARD_DEVIATION 2.4 • n=5 Participants
Sex: Female, Male Female	46 Participants n=5 Participants
Sex: Female, Male Male	54 Participants n=5 Participants
Region of Enrollment Egypt	76 participants n=5 Participants
Region of Enrollment Malaysia	11 participants n=5 Participants
Region of Enrollment Indonesia	13 participants n=5 Participants
serum ferritin	2531.7 ug/L STANDARD_DEVIATION 1463.0 • n=5 Participants

PRIMARY outcome

Timeframe: 24 Weeks

Population: All subjects enrolled (100), had at least one dose of Ferriprox oral solution, all were included in safety analysis

Number of Adverse Events over 24 weeks

Outcome measures

Outcome measures
Measure	Ferriprox Oral Solution n=100 Participants All subjects were administered Ferriprox Oral Solution three times daily for a total daily dose of either 50, 75 or 100 mg/kg/day. Subjects were initiated at a dose of 50 mg/kg/day, but this dose could be increased after two weeks of therapy depending on the subjects' individual needs.
Occurrence of Adverse Events	212 Adverse Events

SECONDARY outcome

Timeframe: Baseline and 24 weeks

Population: 99 subjects had at least one post baseline measurement of serum ferritin concentration and were eligible for the efficacy analyses in the Intent to Treat population (all subjects). The Last Measurement Carried Forward methodology was used to populate any missing serum ferritin value.

The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study

Outcome measures

Outcome measures
Measure	Ferriprox Oral Solution n=99 Participants All subjects were administered Ferriprox Oral Solution three times daily for a total daily dose of either 50, 75 or 100 mg/kg/day. Subjects were initiated at a dose of 50 mg/kg/day, but this dose could be increased after two weeks of therapy depending on the subjects' individual needs.
Change in Serum Ferritin Concentration From Baseline.	-355.5 ug/L Standard Deviation 978.1

Adverse Events

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dian Shaw, Manager Clinical Research

ApoPharma

Phone: 416-401-7283

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Sponsor has the right to review manuscript at least 60 days prior to submission and posters/abstracts at least 30 days. If review indicates potentially patentable or confidential information would be disclosed, submission may be delayed an extra 30 days to allow filing the necessary patent applications and to co-operate with the sponsor to protect confidential information. Investigator will delay single center publications until after disclosure of pooled data (all sites) by Sponsor.
Publication restrictions are in place

Restriction type: OTHER