Clinical trial NCT01250951: 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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Myelodysplastic Syndrome
Thalassemia
Keywords: Iron Overload
hemotranfusion
deferasirox
ferritin
Rare anemia
Transfusional Iron Overload
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-12-01
Enrollment: 111
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
timeFrame: Baseline assessment is followed by monthly assessments for up to 1 year
Secondary outcomes:
measure: changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination
timeFrame: Baseline assessment is followed by monthly assessments for up to 1 year.
measure: 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
timeFrame: at baseline and 1 at year (at the end of study).
measure: 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.
timeFrame: From the start of study up to 1 year
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: Novartis Investigative Site
city: Saint-Petersburg
country: Russian Federation
facility: Novartis Investigative Site
city: St. Petersburg
country: Russian Federation
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
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.