NCT00529152

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Aug 2007

Shorter than P25 for phase_3

Geographic Reach
3 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2007

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

September 12, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 14, 2007

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2008

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

August 12, 2009

Completed
Last Updated

September 2, 2009

Status Verified

August 1, 2009

Enrollment Period

11 months

First QC Date

September 12, 2007

Results QC Date

June 26, 2009

Last Update Submit

August 25, 2009

Conditions

Iron Overload

Keywords

Iron Overload

Outcome Measures

Primary Outcomes (1)

  • Occurrence of Adverse Events

    Number of Adverse Events over 24 weeks

    24 Weeks

Secondary Outcomes (1)

  • Change in Serum Ferritin Concentration From Baseline.

    Baseline and 24 weeks

Study Arms (1)

A

OTHER

Ferriprox Oral Solution single treatment

Drug: Deferiprone

Interventions

DeferiproneDRUG

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.

Also known as: Ferriprox Oral Solution
A

Eligibility Criteria

AgeUp to 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Abo El Reish Hospital, Cairo University

Cairo, Egypt

Location

Children Hospital, Ain Shams University

Cairo, Egypt

Location

Cipto Mangunkusumo National Hospital

Jakarta, 10010, Indonesia

Location

University of Malaya Medical Center

Kuala Lumpur, 50603, Malaysia

Location

Related Publications (1)

  • ElAlfy MS, Sari TT, Lee CL, Tricta F, El-Beshlawy A. The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol. 2010 Nov;32(8):601-5. doi: 10.1097/MPH.0b013e3181ec0f13.

    PMID: 20921906DERIVED

MeSH Terms

Conditions

Iron Overload

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Iron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

PyridonesPyridinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Results Point of Contact

Title
Dian Shaw, Manager Clinical Research
Organization
ApoPharma
dshaw@apotex.com
416-401-7283

Study Officials

  • Prof. Mohsen S El Alfy, MD

    Children Hospital, Ain Shams University, Cairo, Egypt

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

September 12, 2007

First Posted

September 14, 2007

Study Start

August 1, 2007

Primary Completion

July 1, 2008

Study Completion

July 1, 2008

Last Updated

September 2, 2009

Results First Posted

August 12, 2009

Record last verified: 2009-08

Locations

MY(1)ID(1)EG(2)