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.

• ClinicalTrials.gov Identifier: NCT01250951
• Recruitment Status: Completed
• First Posted: December 1, 2010
• Last Update Posted: December 12, 2016
• Sponsor: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals )

Tracking Information

• First Submitted Date: November 11, 2010
• First Posted Date: December 1, 2010
• Last Update Posted Date: December 12, 2016
• Study Start Date: December 2009
• Actual Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: November 29, 2010): 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 ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 29, 2010)

• 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. ]
• 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). ]
• 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. [ Time Frame: From the start of study up to 1 year ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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.
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Myelodysplastic Syndrome
• Thalassemia

Intervention

Drug: Deferasirox

Other Name: ICL670

Study Arms

Experimental: Deferasirox

Intervention: Drug: Deferasirox

• Publications *: 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. doi: 10.3324/haematol.2009.014696. Epub 2009 Nov 30.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: November 29, 2010): 111
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: September 2011
• Actual Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Russian Federation

Administrative Information

• NCT Number: NCT01250951
• Other Study ID Numbers: CICL670ARU01
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Novartis ( Novartis Pharmaceuticals )
• Original Responsible Party: External Affairs, Novartis Pharmaceuticals
• Current Study Sponsor: Novartis Pharmaceuticals
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Novartis Pharmaceuticals; Novartis Pharmaceuticals

• PRS Account: Novartis
• Verification Date: December 2011