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

• Sponsor: ApoPharma (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00529152
• Collaborator: (none)
• Enrollment: 100
• Locations: 4
• Arm: 1
• Duration (Months): 11
• Patients Per Site: 25
• Patients Per Site Per Month: 2.3

Study Details

Study Description

Brief Summary

• The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia.

• The secondary objective is to assess the efficacy of Ferriprox oral solution in reducing iron overload in pediatric patients with transfusion-dependent anemia.

Condition or Disease	Intervention/Treatment	Phase
Iron Overload	Drug: Deferiprone	Phase 3

Detailed Description

This will be a multi-centre, open label, single treatment, uncontrolled study. A total of 100 iron-overloaded pediatric patients with transfusion-dependent anemia will be enrolled in the study.Eligible patients will receive Ferriprox (deferiprone) oral solution, 100 mg/mL, at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided in three (3) doses, for 24 weeks.

Study Design

• Study Type: Interventional
• Actual Enrollment: 100 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A 24-Week, Open Label, Uncontrolled Study of the Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients With Transfusion-Dependent Anemia
• Study Start Date: Aug 1, 2007
• Actual Primary Completion Date: Jul 1, 2008
• Actual Study Completion Date: Jul 1, 2008

Arms and Interventions

Arm	Intervention/Treatment
Other: A Ferriprox Oral Solution single treatment	Drug: Deferiprone; Ferriprox (deferiprone) oral solution will be given orally at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided into 3 doses, for 24 weeks.; Other Names: Ferriprox Oral Solution

Outcome Measures

Primary Outcome Measures

1. Occurrence of Adverse Events [24 Weeks]

   Number of Adverse Events over 24 weeks

Secondary Outcome Measures

1. Change in Serum Ferritin Concentration From Baseline. [Baseline and 24 weeks]

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

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 10 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Patients who are ≤ 10 years of age.

• Patients who have a confirmed diagnosis of transfusion-dependent anemia, other than Blackfan-Diamond anemia, and have chronic iron overload requiring chelation therapy.

• Patients who are in a chronic transfusion program, and who have received at least eight (8) red blood cell transfusions per year for a minimum of one year.

• Patients who are iron overloaded as assessed by serum ferritin concentration greater than 1000 µg/L.

Exclusion Criteria:

• Patients who have a diagnosis of Blackfan-Diamond anemia.

• Patients who have experienced neutropenia/agranulocytosis (absolute neutrophil count (ANC) < 1.5 x 109/L) or thrombocytopenia (platelet count < 50.0 x 109/L).

• Patients who have had previous treatment with Ferriprox and presented serious adverse reaction or intolerance requiring withdrawal of Ferriprox.

• Patients with evidence of abnormal liver function (ALT level > 3 times the upper limit of normal; entry may be delayed until values return to normal).

• Patients with evidence of renal failure, characterized by serum creatinine level > 2 times the upper limit of normal; entry may be delayed until values return to normal.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Abo El Reish Hospital, Cairo University	Cairo		Egypt
2	Children Hospital, Ain Shams University	Cairo		Egypt
3	Cipto Mangunkusumo National Hospital	Jakarta		Indonesia	10010
4	University of Malaya Medical Center	Kuala Lumpur		Malaysia	50603

Sponsors and Collaborators

• ApoPharma

Investigators

• Principal Investigator: Prof. Mohsen S El Alfy, MD, Children Hospital, Ain Shams University, Cairo, Egypt

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00529152
• Other Study ID Numbers: LA30-0307
• First Posted: Sep 14, 2007
• Last Update Posted: Sep 2, 2009
• Last Verified: Aug 1, 2009

• Keywords provided by , ,: Iron Overload
• Additional relevant MeSH terms: Iron Overload; Deferiprone

Study Results

Participant Flow

Arm/Group Title	Ferriprox Oral Solution
Arm/Group Description	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.
Period Title: Overall Study
STARTED	100
COMPLETED	95
NOT COMPLETED	5

Baseline Characteristics

Arm/Group Title	Ferriprox Oral Solution
Arm/Group Description	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 Participants	100
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	5.1 (2.4)
Sex: Female, Male (Count of Participants)
Female	46 46%
Male	54 54%
Region of Enrollment (participants) [Number]
Egypt	76 76%
Malaysia	11 11%
Indonesia	13 13%
serum ferritin (ug/L) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [ug/L]	2531.7 (1463.0)

Outcome Measures

1. Primary Outcome

Title	Occurrence of Adverse Events
Description	Number of Adverse Events over 24 weeks
Time Frame	24 Weeks

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Ferriprox Oral Solution
Arm/Group Description	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.
Measure Participants	100
Number [Adverse Events]	212

2. Secondary Outcome

Title	Change in Serum Ferritin Concentration From Baseline.
Description	The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study
Time Frame	Baseline and 24 weeks

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferriprox Oral Solution
Arm/Group Description	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.
Measure Participants	99
Mean (Standard Deviation) [ug/L]	-355.5 (978.1)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Ferriprox Oral Solution
	Comments	Change in Serum Ferritin from baseline to week 24 was compared using regression analysis; null hypothesis was defined as no change in serum ferritin from baseline to week 24
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.0005
	Comments
	Method	Regression, Linear
	Comments

Adverse Events

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.