NCT01033747

Brief Summary

The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
70

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Feb 2003

Longer than P75 for phase_2

Geographic Reach
1 country

4 active sites

Status
completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2003

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2008

Completed
1.8 years until next milestone

First Submitted

Initial submission to the registry

October 16, 2009

Completed
2 months until next milestone

First Posted

Study publicly available on registry

December 16, 2009

Completed
1.7 years until next milestone

Results Posted

Study results publicly available

August 11, 2011

Completed
Last Updated

August 11, 2011

Status Verified

July 1, 2011

Enrollment Period

4.9 years

First QC Date

October 16, 2009

Results QC Date

December 21, 2010

Last Update Submit

July 18, 2011

Conditions

Liver Iron Overload

Keywords

iron overloadiron chelation therapyB-thalassemia

Outcome Measures

Primary Outcomes (1)

  • The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox

    The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.

    Baseline to 7 Years

Secondary Outcomes (1)

  • Relative Change in Serum Ferritin From Baseline to 3.5 Years

    Baseline to 3.5 years

Study Arms (2)

Deferasirox

EXPERIMENTAL

Deferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study

Drug: Deferasirox

Deferasirox Crossover

EXPERIMENTAL

Deferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study

Drug: Deferasirox

Interventions

DeferasiroxDRUG

10 mg/kg or 30 mg/kg orally daily

Deferasirox

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
  • Patients currently participating in the food-effect sub-study, according to amendment 3.
  • Ability to provide written informed consent prior to participation in this non-comparative extension study.
  • Female patients sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
  • Body weight of at least 35 kg.

You may not qualify if:

  • Pregnant or breastfeeding patients.
  • History of non-compliance to medical regimens and patients who are considered potentially unreliable.
  • Proteinuria \> 300 mg/L second void morning urine.
  • Patients with serum creatinine above the upper limit normal.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Novartis Investigative Site

Cagliari, Italy

Location

Novartis Investigative Site

Genova, Italy

Location

Novartis Investigative Site

Milan, Italy

Location

Novartis Investigative Site

Torino, Italy

Location

MeSH Terms

Conditions

Iron Overloadbeta-Thalassemia

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Iron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

BenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals
862 778-8300

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

October 16, 2009

First Posted

December 16, 2009

Study Start

February 1, 2003

Primary Completion

January 1, 2008

Study Completion

January 1, 2008

Last Updated

August 11, 2011

Results First Posted

August 11, 2011

Record last verified: 2011-07

Locations

IT(4)