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.

Myelodysplastic Syndrome Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01250951
• Other study ID #: CICL670ARU01
• Status: Completed
• Phase: Phase 4
• First received: November 11, 2010
• Last updated: December 8, 2016
• Start date: December 2009
• Est. completion date: September 2011

Study information

• Verified date: December 2011
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Russia: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 111
• Est. completion date: September 2011
• Est. primary completion date: July 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years and older

Eligibility

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.

Other protocol-defined inclusion/exclusion criteria may apply

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Iron Overload
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome
• Thalassemia

Intervention

Drug:
• Deferasirox

Locations

Country	Name	City	State
Russian Federation	Novartis Investigative Site	Moscow
Russian Federation	Novartis Investigative Site	Saint-Petersburg
Russian Federation	Novartis Investigative Site	St. Petersburg

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Russian Federation, 

References & Publications (1)

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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	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.	No
Secondary	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).	No
Secondary	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	Yes

External Links

•   Link
•   Link
•   Deferasirox
•   Myelodisplastic Syndrome
•   Results for CICL670ARU01 on the Novartis Clinical Trial Results Website
•   Thalassemia
•   Transfusional Iron Overload