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Open-label Study of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Undergoing Treatment Programs in Comparison With Control Group

Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients



Sponsors

Lead Sponsor

 Novartis Pharmaceuticals


Source

Novartis

Oversight Info

Has Dmc

Yes

Is Fda Regulated Drug

No

Is Fda Regulated Device

No


Brief Summary

To evaluate Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.

Overall Status

Completed

Start Date

2014-06-23

Completion Date

2016-10-17

Primary Completion Date

2016-10-17

Phase

Phase 4

Study Type

Interventional

Primary Outcome

Measure

Time Frame

Change of serum ferritin, iron transferrin saturation, serum total iron-binding capacity (TIBC)
Screening, 4,8,12,16,20,24,28,32,36,40,44,48,52 weeks

Secondary Outcome

Measure

Time Frame

Adverse events
52 weeks
Change of proteinuria in urinalysis over a period of 1 year treatment.
1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks
Change from baseline in creatinine clearance over a period of 1 year treatment.
1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks
Change from baseline of serum creatinine, bilirubin, ALT, AST, glucose over a period of 1 year treatment.
1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks

Enrollment

14

Condition

 Aplastic Anemia

Intervention

Intervention Type

Drug

Intervention Name

 ICL670A and standard immunosupressive therapy (Cyclosporine A)

Description

In the investigational arm all patients recieve a standard immunosuppressant (Cyclosporine A). The starting dose of Exjade will be 20 mg/kg/day with up- and down-titration steps of 5-10 mg/kg/day if necessary, depending on serum ferritin, tolerability and comorbidities. Doses above 30 mg/kg are discouraged as such experience is limited in aplastic anemia. Patients are to take Exjade once daily at least 30 minutes before meals, on the same time each day. A combination of 250 mg and 500 mg tablets may be used to provide a specific dose level. The duration of treatment in this study is 12 months. The investigator muct emphasize complaince and will instruct the patients to take the Exjade exactly as prescribe.

Arm Group Label

Exjade treatment arm

Other Name

Deferasirox and standard immunosupressive therapy


Intervention Type

Drug

Intervention Name

 Immunosupressive therapy (Cyclosporine A)

Description

Comparative group of patients with aplastic anemia and transfusion-dependent iron overload is undergoing treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A)

Arm Group Label

Conventional treatment arm



Eligibility

Criteria

Inclusion Criteria: - Main diagnosis: aplastic anemia - Absence of severe and/or uncontrolled comorbidities - Confirmed iron overload (serum ferritin ≥ 1000 mkg/L) - Serum creatinine is not higher than the upper limit of normal for the given age - Absence of severe proteinuria. Protein/Creatinine ratio should be < 0.5 mg/mg - Liver enzymes are < 5 ULN - Completion of a scheduled cycle of immunosuppressive treatment program, with no severe infectious or generalized hemorrhagic complications - WHO (ECOG) performance status ≤ 2 Exclusion Criteria: - No signed informed consent form - Patient is under 18 years old - Severe concomitant condition - Severe infectious and generalized haemorrhagic complication following regular planned cycle of programmed immune suppressive treatment. - History of increased sensitivity to active substance and any other ingredient of the medicinal product. - Creatinine clearance (CC) < 60 ml/min and/or creatinine concentration in blood serum is 2 or more times higher than upper limit of age normal by results of 2 tests at Visits 1 and 2. - Severe liver disorders (class C by Child-Pugh scale). - Patients with aplastic anaemia in which chelator treatment will be ineffective due to rapid progression of the disease. - Significant proteinuria basing on protein creatinine ratio > 1.0 mg/ml in urine sample from second urination at Visits 1 and 2 (or as an alternative in 2 of 3 urine samples at screening); - Rare hereditary disorders related to galactose intolerance, severe deficit of lactase or glucose-galactose malabsorption; - Pregnancy, lactation; - Level of liver enzymes higher than 5 upper limits of age normal at Visits 1 and 2.

Gender

All

Minimum Age

18 Years

Maximum Age

N/A

Healthy Volunteers

No


Overall Official

Last Name

Role

Affiliation

Novartis Pharmaceuticals
Study Director
Novartis Pharmaceuticals

Location

Facility

Novartis Investigative Site
Moscow 125167 Russian Federation

Location Countries

Country

Russian Federation


Verification Date

2017-08-01

Lastchanged Date

N/A

Firstreceived Date

N/A

Responsible Party

Responsible Party Type

Sponsor


Keyword

 Aplastic anemia, deferasirox, renal function

Has Expanded Access

No

Condition Browse

 Anemia,  Anemia, Aplastic,  Iron Overload

Number Of Arms

2

Intervention Browse

Mesh Term

Cyclosporins

Cyclosporine

Immunosuppressive Agents

Deferasirox



Arm Group

Arm Group Label

Conventional treatment arm

Arm Group Type

Active Comparator

Description

10 Adult (aged above 18) transfusion-dependent patients with AA and serum ferritin < 1000 mg/L undergoing treatment programs of immunosuppressive treatment (Cyclosporine A)


Arm Group Label

Exjade treatment arm

Arm Group Type

Experimental

Description

15 transfusion-dependent adult (aged above 18) patients with AA and serum ferritin ≥ 1000 mg/L undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) and Exjade



Firstreceived Results Date

N/A

Patient Data

Sharing Ipd

Undecided

Ipd Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com


Firstreceived Results Disposition Date

N/A

Study Design Info

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary Purpose

Treatment

Masking

None (Open Label)


Study First Submitted

March 5, 2013

Study First Submitted Qc

March 25, 2013

Study First Posted

March 26, 2013

Last Update Submitted

August 25, 2017

Last Update Submitted Qc

August 25, 2017

Last Update Posted

August 28, 2017


ClinicalTrials.gov processed this data on August 29, 2018

Conditions

Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov, conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions

Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied. Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase

Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions that study is seeking to answer:

In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.

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