Clinical trial NCT00466063: ENTRUST, a 5 Year Surveillance of Children Aged 2 to <6 Years With Transfusional Iron Overload Treated With Deferasirox
Official title: A 5 Year Observational Study (Registry) of Children Aged 2 to <6 Years at Enrollment With Transfusional Hemosiderosis Treated With Deferasirox
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Anemia
Keywords: Iron overload
children
deferasirox
Transfusion-dependent anemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2007-04-27
Enrollment: 108
Sex: ALL
Minimum age (years): 2
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 200 unselected children aged from 2 to \<6 years at enrolment with chronic iron overload due to repeated blood transfusions. The participating countries were selected on the basis of both a high incidence of young children with thalassemia or other transfusion dependent anemias and on the basis that the drug is approved and 'on the market' in the country selected.

Brief summary: This registry will evaluate long-term safety and efficacy of deferasirox in children with transfusional iron overload.


Eligibility criteria: Inclusion Criteria:

* History of transfusion-dependent anemia.
* History of iron overload

Exclusion Criteria:

* Patients with non-transfusional hemosiderosis.

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Safety - renal and hepatic function monitoring.
timeFrame: 5 years
Secondary outcomes:
measure: Adverse events
timeFrame: 5 years
measure: Longitudinal ferritin levels
timeFrame: 5 years
measure: Assessment of auditory and ophthalmologic status
timeFrame: 5 years
Other outcomes:
Locations:
city: Mobile
country: United States
facility: University of South Alabama Medical Center
state: Alabama
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Oakland
country: United States
facility: Children's Hospital Oakland
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Stanford
country: United States
facility: Stanford University Medical Center
state: California
city: Orlando
country: United States
facility: MD Anderson Cancer Center - Orlando
state: Florida
city: Tampa
country: United States
facility: St. Joseph's Children's Hospital of Tampa
state: Florida
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Boston
country: United States
facility: Children's Hospital Boston
state: Massachusetts
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Paterson
country: United States
facility: St. Joseph's Children's Hospital
state: New Jersey
city: Brooklyn
country: United States
facility: The Brooklyn Hospital Center
state: New York
city: New York
country: United States
facility: Schneider Children's Hospital
state: New York
city: Syracuse
country: United States
facility: SUNY - Upstate Medical University
state: New York
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Dayton
country: United States
facility: Children's Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: Oklahoma State University Health Sciences Center
state: Oklahoma
city: Hershey
country: United States
facility: Penn State University / Milton S. Hershey Medical Center
state: Pennsylvania
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital Memphis
state: Tennessee
city: Fort Worth
country: United States
facility: Cook's Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: