Treatment of Iron Overload Requiring Chelation Therapy
This study is not yet open for participant recruitment.
Verified January 2014 by Shire Development LLC
Information provided by (Responsible Party):
Shire Development LLC
First received: August 20, 2013
Last updated: January 6, 2014
Last verified: January 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 ]
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||August 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).
Please refer to this study by its ClinicalTrials.gov identifier: NCT01927913
|Contact: Shire Call Center
Shire Development LLC
||Ali Taher, MD, PhD, FRCP
||American University of Beirut Medical Center
No publications provided
||Shire Development LLC
History of Changes
|Other Study ID Numbers:
||SPD602-204, FBS0701 (SSP-004184)
|Study First Received:
||August 20, 2013
||January 6, 2014
||United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on March 13, 2014
Iron Metabolism Disorders
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action