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This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

NCT ID: NCT01250951

Last Updated: 2016-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

111 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-12-31

Study Completion Date

2011-09-30

Brief Summary

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

Detailed Description

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Conditions

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Myelodysplastic Syndrome Thalassemia

Keywords

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Iron Overload hemotranfusion deferasirox ferritin Rare anemia Transfusional Iron Overload

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Deferasirox

Group Type EXPERIMENTAL

Deferasirox

Intervention Type DRUG

Interventions

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Deferasirox

Intervention Type DRUG

Other Intervention Names

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ICL670

Eligibility Criteria

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Inclusion Criteria

1. Age ≥ 2 years
2. 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)
3. ECOG Performance Status ≤ 2
4. Transfusion overload confirmed with ferritin level \>1000 µg/l.
5. No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
6. Serum creatine level \> ULN
7. No proteinuria
8. Liver enzymes level \< 5 ULN.
9. No pregnancy or lactation
10. Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.

Exclusion Criteria

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

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Moscow, , Russia

Site Status

Novartis Investigative Site

Saint Petersburg, , Russia

Site Status

Countries

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Russia

References

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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.

Reference Type RESULT

Related Links

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http://www.nlm.nih.gov/medlineplus/druginfo/meds/a606002.html

Deferasirox

http://www.nlm.nih.gov/medlineplus/thalassemia.html

Thalassemia

http://vsearch.nlm.nih.gov/vivisimo/cgi-bin/query-meta?v%3aproject=medlineplus&v%3asources=medlineplus-bundle&query=myelodysplastic%20syndrome&

Myelodisplastic Syndrome

Other Identifiers

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CICL670ARU01

Identifier Type: -

Identifier Source: org_study_id