NCT01250951

Brief Summary

This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
111

participants targeted

Target at P50-P75 for phase_4

Timeline
Completed

Started Dec 2009

Geographic Reach
1 country

2 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

December 1, 2009

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

November 11, 2010

Completed
20 days until next milestone

First Posted

Study publicly available on registry

December 1, 2010

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2011

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2011

Completed
Last Updated

December 12, 2016

Status Verified

December 1, 2011

Enrollment Period

1.6 years

First QC Date

November 11, 2010

Last Update Submit

December 8, 2016

Conditions

Myelodysplastic SyndromeThalassemia

Keywords

Iron OverloadhemotranfusiondeferasiroxferritinRare anemiaTransfusional Iron Overload

Outcome Measures

Primary Outcomes (1)

  • changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade

    Baseline assessment is followed by monthly assessments for up to 1 year

Secondary Outcomes (3)

  • changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination

    Baseline assessment is followed by monthly assessments for up to 1 year.

  • changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade

    at baseline and 1 at year (at the end of study).

  • Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.

    From the start of study up to 1 year

Study Arms (1)

Deferasirox

EXPERIMENTAL
Drug: Deferasirox

Interventions

DeferasiroxDRUG
Also known as: ICL670
Deferasirox

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Age ≥ 2 years
  • Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)
  • ECOG Performance Status ≤ 2
  • Transfusion overload confirmed with ferritin level \>1000 µg/l.
  • No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
  • Serum creatine level \> ULN
  • No proteinuria
  • Liver enzymes level \< 5 ULN.
  • No pregnancy or lactation

You may not qualify if:

  • Age \< 2 years
  • No iron overload (Ferritin level \<1000 µg/l).
  • Primary iron overload (hereditary hemochromatosis)
  • Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)
  • Elevated serum creatinine \> ULN or/and proteinuria
  • Liver enzymes level \>5 ULN.
  • Pregnancy or lactation.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Novartis Investigative Site

Moscow, Russia

Location

Novartis Investigative Site

Saint Petersburg, Russia

Location

Related Publications (1)

  • Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. Epub 2009 Dec 8. Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. Epub 2009 Nov 30.

    RESULT

Related Links

MeSH Terms

Conditions

Myelodysplastic SyndromesThalassemiaIron Overload

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesIron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

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

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 11, 2010

First Posted

December 1, 2010

Study Start

December 1, 2009

Primary Completion

July 1, 2011

Study Completion

September 1, 2011

Last Updated

December 12, 2016

Record last verified: 2011-12

Locations

RU(2)