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Failure to meet the schedule of patient recruitment
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Evaluated 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.
The secondary endpoints that were originally planned for this study were not analyzed as the study ended prematurely.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Serum ferritin level ≥ 1,000 μg/l | Experimental | Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study |
|
| Serum ferritin level < 1,000 μg/l | Experimental | Transfusion-dependent adult patients with AA and serum ferritin < 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ICL670 | Drug | ICL670 was supplied in registered packages as 250mg or 500mg dispersible tablets. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Serum Ferritin Values | Change from baseline was be summarized descriptively for all on-treatment study visits. Changes to the planned statistical analysis were related to significant withdrawal of patients from the Per-Protocol Analysis Set due to a large number of patients who discontinued the study (lack of assessments of iron exchange parameters at visits) and deviations from the Protocol affecting the assessment of efficacy parameters. Because of that, the additional efficacy analysis in the Per-Protocol Analysis Set was not performed. | Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52 |
| Change in Transferrin Saturation With Iron (TSI) Values | Mean percentage change from baseline in transferrin saturation with iron was summarized descriptively for all on-treatment study visits. | Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52 |
| Change in Serum Total Iron-binding Capacity (TIBC) | Mean change from baseline in serum total iron-binding capacity was summarized descriptively for all on-treatment study visits. | Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Moscow | 125167 | Russia |
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
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The study enrolled adult transfusion-dependent patients with Aplastic Anaemia (AA) on programmed immune suppressive treatment.
Estimated number of patients was 25; the actual number of patients enrolled in the study and included in efficacy analysis was 15.
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| ID | Title | Description |
|---|---|---|
| FG000 | Serum Ferritin Level ≥ 1,000 μg/l | Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study |
| FG001 | Serum Ferritin Level < 1,000 μg/l | Transfusion-dependent adult patients with AA and serum ferritin < 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety Analysis Set included all enrolled patients received at least one dose of the investigational product, as well as all patients from the comparison group.
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| ID | Title | Description |
|---|---|---|
| BG000 | Serum Ferritin Level ≥ 1,000 μg/l | Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study |
| BG001 | Serum Ferritin Level < 1,000 μg/l |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Serum Ferritin Values | Change from baseline was be summarized descriptively for all on-treatment study visits. Changes to the planned statistical analysis were related to significant withdrawal of patients from the Per-Protocol Analysis Set due to a large number of patients who discontinued the study (lack of assessments of iron exchange parameters at visits) and deviations from the Protocol affecting the assessment of efficacy parameters. Because of that, the additional efficacy analysis in the Per-Protocol Analysis Set was not performed. | Full Analysis Set included all patients of the Safety Analysis Set with available baseline data & data obtained at follow-up visits &/or final visit, for evaluation of at least 1 efficacy parameter. Statistical analysis was not performed due to large number of patients discontinuing & thus lack of assessments of iron exchange parameters at visits. | Posted | Mean | Standard Deviation | ng/ml | Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52 |
|
Adverse events and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 376 days.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Serum Ferritin Level ≥ 1,000 μg/l | Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA ver. 20.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | novartis.email@novartis.com |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| D002614 | Chelating Agents |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
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| Chelation | Drug | Main group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment (immunosupressant - Cyclosporine A) and received chelation with ICL670 (deferasirox). |
|
| No chelation | Drug | Comparative group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A) |
|
| Decision to perform 2nd course of trtmnt |
|
Transfusion-dependent adult patients with AA and serum ferritin < 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product |
| BG002 | Total | Total of all reporting groups |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | Participants |
|
| OG000 |
| Serum Ferritin Level ≥ 1,000 μg/l |
Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study |
| OG001 | Serum Ferritin Level < 1,000 μg/l | Transfusion-dependent adult patients with AA and serum ferritin < 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product |
|
|
| Primary | Change in Transferrin Saturation With Iron (TSI) Values | Mean percentage change from baseline in transferrin saturation with iron was summarized descriptively for all on-treatment study visits. | Full Analysis Set (FAS) included all patients of the Safety Analysis Set with the available baseline data, as well as data obtained at follow-up visits and/or the final visit, for evaluation of at least one efficacy parameter. | Posted | Mean | Standard Deviation | Percentage of saturation | Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52 |
|
|
|
| Primary | Change in Serum Total Iron-binding Capacity (TIBC) | Mean change from baseline in serum total iron-binding capacity was summarized descriptively for all on-treatment study visits. | Full Analysis Set (FAS) included all patients of the Safety Analysis Set with the available baseline data, as well as data obtained at follow-up visits and/or the final visit, for evaluation of at least one efficacy parameter. | Posted | Mean | Standard Deviation | μmol/l | Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52 |
|
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| 0 |
| 10 |
| 0 |
| 10 |
| 4 |
| 10 |
| EG001 | Serum Ferritin Level < 1,000 μg/l | Transfusion-dependent adult patients with AA and serum ferritin < 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product | 0 | 5 | 0 | 5 | 0 | 5 |
| Blood creatine increased | Investigations | MedDRA ver. 20.0 | Systematic Assessment |
|
| Weakness | General disorders | MedDRA ver. 20.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA ver. 20.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA ver. 20.0 | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | MedDRA ver. 20.0 | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety
| D001855 | Bone Marrow Diseases |
| D001555 |
| Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D064449 | Sequestering Agents |
| D045504 | Molecular Mechanisms of Pharmacological Action |
| D020228 | Pharmacologic Actions |
| D020164 | Chemical Actions and Uses |
| D020313 | Specialty Uses of Chemicals |
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