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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.

Primary Purpose

Myelodysplastic Syndrome, Thalassemia

Status

Completed

Phase

Phase 4

Locations

Russian Federation

Study Type

Interventional

Intervention

Deferasirox

About this trial

This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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
Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.

Exclusion Criteria:

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.

Other protocol-defined inclusion/exclusion criteria may apply

Sites / Locations

Novartis Investigative Site
Novartis Investigative Site
Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

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

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.

Full Information

NCT ID

NCT01250951

First Posted

November 11, 2010

Last Updated

December 8, 2016

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT01250951

Brief Title

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.

Official Title

1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

Study Type

Interventional

2. Study Status

Record Verification Date

December 2011

Overall Recruitment Status

Completed

Study Start Date

December 2009 (undefined)

Primary Completion Date

July 2011 (Actual)

Study Completion Date

September 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelodysplastic Syndrome, Thalassemia

Keywords

Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

111 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Deferasirox

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Deferasirox

Other Intervention Name(s)

ICL670

Primary Outcome Measure Information:

Title

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

Time Frame

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

Secondary Outcome Measure Information:

Title

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

Time Frame

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

Title

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

Time Frame

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

Title

Time Frame

From the start of study up to 1 year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents. Exclusion Criteria: 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. Other protocol-defined inclusion/exclusion criteria may apply

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Novartis Pharmaceuticals

Organizational Affiliation

Novartis Pharmaceuticals

Official's Role

Study Director

Facility Information:

Facility Name

Novartis Investigative Site

City

Moscow

Country

Russian Federation

Facility Name

Novartis Investigative Site

City

Saint-Petersburg

Country

Russian Federation

Facility Name

Novartis Investigative Site

City

St. Petersburg

Country

Russian Federation

12. IPD Sharing Statement

Citations:

Citation

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.

Results Reference

result

Links:

URL

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

Description

Deferasirox

URL

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

Description

Thalassemia

URL

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

Description

Myelodisplastic Syndrome

URL

http://elibrary.ru/item.asp?id=24872220.

Description

Related Info

Learn more about this trial

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.

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