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.
This study has been completed.
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01250951
First received: November 11, 2010
Last updated: December 1, 2011
Last verified: December 2011
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Purpose
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
| Condition | Intervention | Phase |
|---|---|---|
|
Myelodysplastic Syndrome Thalassemia |
Drug: Deferasirox |
Phase 4 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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. |
Resource links provided by NLM:
MedlinePlus related topics:
Anemia
Blood Transfusion and Donation
Iron
Myelodysplastic Syndromes
Thalassemia
Drug Information available for:
Deferasirox
U.S. FDA Resources
Further study details as provided by Novartis:
Primary Outcome Measures:
- 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 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- 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. ] [ Designated as safety issue: No ]
- 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). ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: Yes ]
| Enrollment: | 111 |
| Study Start Date: | December 2009 |
| Study Completion Date: | September 2011 |
| Primary Completion Date: | July 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Deferasirox |
Drug: Deferasirox
Other Name: ICL670
|
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01250951
Locations
| Russian Federation | |
| Novartis Investigative Site | |
| Moscow, Russian Federation | |
| Novartis Investigative Site | |
| Saint-Petersburg, Russian Federation | |
| Novartis Investigative Site | |
| St. Petersburg, Russian Federation | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
Additional Information:
No publications provided
| Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT01250951 History of Changes |
| Other Study ID Numbers: | CICL670ARU01 |
| Study First Received: | November 11, 2010 |
| Last Updated: | December 1, 2011 |
| Health Authority: | Russia: Ministry of Health |
Keywords provided by Novartis:
|
Iron Overload hemotranfusion deferasirox |
ferritin Rare anemia Transfusional Iron Overload |
Additional relevant MeSH terms:
|
Anemia Myelodysplastic Syndromes Preleukemia Thalassemia Iron Overload Hematologic Diseases Bone Marrow Diseases Precancerous Conditions Neoplasms Anemia, Hemolytic, Congenital |
Anemia, Hemolytic Hemoglobinopathies Genetic Diseases, Inborn Iron Metabolism Disorders Metabolic Diseases Deferasirox Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on June 18, 2013