| Eligibility |
Inclusion Criteria:
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:
1. Post-menopausal (12 consecutive months of spontaneous amenorrhea)
2. Surgically sterile, or
3. 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).
Exclusion Criteria:
- Severe iron overload including: (a) cardiac T2* MRI less than 10.0ms; or (b) LIC by
FerriScan® R2 MRI greater than or equal to 30.0mg/g liver (dw) as assessed at the
Screening Visit (Visit 1).
- Iron overload from causes other than transfusional siderosis.
- Subjects with thalassemia intermedia
- Unable to undergo MRI assessment.
- Cardiac LVEF less than 50% at baseline testing by MRI.
- Subjects with documented liver failure (presence of portal hypertension, hepatic
edema, ascites, cirrhosis), Child-Pugh C hepatic impairment, or biliary disorder.
- Platelet count <100 x 109/L at the Screening Visit (Visit 1).
- Absolute neutrophils counts of <1500mm3 at the Screening Visit (Visit 1).
- Evidence of renal insufficiency eg, creatinine clearance <60mL/min or serum creatinine
>1.5x ULN at the Screening Visit (Visit 1).
- Clinically significant laboratory assessments at the Screening Visit (Visit 1).
- Significant proteinuria: urinary protein/creatinine ratio >1.0 in a non-first void
urine sample at the Screening Visit (Visit 1).
- Current or recurrent disease that could affect the action, absorption, or disposition
of the investigational product, or clinical or laboratory assessments.
- Current or relevant history of physical or psychiatric illness, any medical disorder
that may require treatment or make the subject unlikely to fully complete the study,
or any condition, including pregnancy, that presents undue risk from the
investigational product or procedures.
- Current use of any medication contraindicated in the deferasirox prescribing
information/SmPC.
- Known or suspected intolerance or hypersensitivity to SSP-004184AQ, deferasirox,
closely-related compounds, or any of the stated ingredients in either medication.
- Known history of alcohol or other substance abuse within the last year.
- Within 30 days prior to the Baseline Visit (Visit 3):
1. Have used an investigational product
2. Have been enrolled in a clinical study (including vaccine studies) that, in the
investigator's opinion, may impact this study.
- History of malignancy within the past 5 years, with the exception of basal cell or
squamous cell skin carcinoma or cervical carcinoma in situ or completely resected
colon carcinoma in situ.
- Insufficient venous access that precludes prescribed blood draws for safety laboratory
assessments.
- Pregnant or lactating females.
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