Treatment of Iron Overload Requiring Chelation Therapy
This study has been withdrawn prior to enrollment.
(This study was withdrawn until the evaluation of the nonclinical rat findings is complete.)
Information provided by (Responsible Party):
First received: August 20, 2013
Last updated: August 7, 2014
Last verified: August 2014
The purpose of this open-label study is to assess liver iron concentration using MRI imaging in subjects with beta-thalassemia when administered with either SPD602 or deferasirox for the treatment of chronic transfusional iron overload.
Transfusional Iron Overload
Iron Metabolism Disorders
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
||A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in B-thalassemia Subjects Administered SPD602 (SSP-004184AQ) or Exjade® (Deferasirox) for Treatment of Chronic Transfusional Iron Overload
Primary Outcome Measures:
- Change in Liver Iron Concentration (LIC) from Baseline Utilizing R2 Magnetic Resonance Imaging (MRI) [ Time Frame: 48 Weeks ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Response Rate Utilizing R2* MRI [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
- Change in Cardiac Iron Concentration (CIC) from baseline Utilizing T2* MRI [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
- Change in Serum Ferritin Levels from Baseline [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
- Change in Pancreas Iron Concentration (PIC) from Baseline Utilizing R2* MRI [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
- Left Ventricular Ejection Fraction (LVEF) Utilizing MRI [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
- Gastrointestinal Symptom Rating Scale [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
- Total Neuropathy Score nurse (TNSn) [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||October 2015 (Final data collection date for primary outcome measure)
32, 50 or 75 mg/kg/day BID, capsule
Other Name: SSP-004184AQ
Active Comparator: Deferasirox
Per approved country specific label
Other Name: Exjade®
|Ages Eligible for Study:
||18 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
These criteria are to be met at the Screening Visit (Visit 1), the Washout Visit (Visit 2), and the Baseline Visit (Visit 3), if reassessed.
- An understanding, ability, and willingness to fully comply with study procedures and restrictions.
- Ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent as applicable to participate in the study before completing any study-related procedures.
- Subjects 18 years of age or older at the time of signing consent.
Female subjects should be one of the following:
- Post-menopausal (12 consecutive months of spontaneous amenorrhea)
- Surgically sterile, or
- Females of child-bearing potential must have a negative serum Beta-HCG pregnancy test at the Screening Visit (Visit 1) and a negative urine pregnancy test at the Baseline Visit (Visit 3). Females of child-bearing potential must agree to abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception.
- Subjects with beta-thalassemia who have received at least 100mL/kg of packed red blood cells (or >20 transfusion units) and who have iron overload (transfusional hemosiderosis) requiring chronic treatment with an iron chelator.
- Serum ferritin >500µg/L at the Screening Visit (Visit 1).
Baseline LIC (last MRI assessment prior to Day 1) greater than or equal to 2.0mg and less than 30.0mg iron per g (equivalent dry weight, liver) determined by FerriScan® R2 MRI.
8. Mean of the previous 3 pre-transfusion hemoglobin concentrations greater than or equal to 7.5g/dL assessed at the Screening Visit (Visit 1) (1 value from clinical laboratory tests taken at the Screening Visit [Visit 1] and the previous 2 historical values available).
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01927913
||Ali Taher, MD, PhD, FRCP
||American University of Beirut Medical Center
No publications provided
History of Changes
|Other Study ID Numbers:
||SPD602-204, FBS0701 (SSP-004184), 2013-000743-33
|Study First Received:
||August 20, 2013
||August 7, 2014
||United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on October 19, 2014
Iron Metabolism Disorders
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action