Thalassemia syndromes;
Myelodysplastic syndrome (MDS) or myelofibrosis;
Diamond Blackfan anemia;
Primary bone marrow failure;
Baseline LIC >30 mg/g dw (measured by MRI);
Unable or unwilling to undergo a 7 day washout period if currently being treated with deferiprone or deferoxamine or deferasirox;
Previous discontinuation of treatment with deferiprone or deferoxamine due to adverse events;
History or presence of hypersensitivity or idiosyncratic reaction to deferiprone or deferoxamine;
Treated with hydroxyurea within 30 days;
History of malignancy;
Evidence of abnormal liver function (serum ALT level(s) > 5 times upper limit of normal at screening or creatinine levels >2 times upper limit of normal at screening);
A serious, unstable illness, as judged by the Investigator, during the past 3 months before screening/baseline visit including but not limited to: hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease;
Clinically significant abnormal 12-lead ECG findings;
Cardiac MRI T2* <10ms;
Myocardial infarction, cardiac arrest or cardiac failure within 1 year before screening/baseline visit;
Unable to undergo MRI
Presence of metallic objects such as artificial joints, inner ear (cochlear) implants, brain aneurysm clips, pacemakers, and metallic foreign bodies in the eye or other body areas that would prevent use of MRI imaging

NCT02041299_exc_T1
Condition
Thalassemia syndromes
NCT02041299_exc_T2
Condition
Myelodysplastic syndrome (MDS)
NCT02041299_exc_T3
Condition
myelofibrosis
NCT02041299_exc_T4
Condition
Diamond Blackfan anemia
NCT02041299_exc_T5
Condition
Primary bone marrow failure
NCT02041299_exc_T6
Measurement
LIC
NCT02041299_exc_T7
Value
>30 mg/g
NCT02041299_exc_T8
Temporal
Baseline
NCT02041299_exc_T9
Procedure
MRI
NCT02041299_exc_T10
Mood
measured by
NCT02041299_exc_T12
Procedure
discontinuation of treatment
NCT02041299_exc_T13
Condition
deferiprone
NCT02041299_exc_T14
Condition
deferoxamine
NCT02041299_exc_T15
Scope
deferiprone or deferoxamine
NCT02041299_exc_T16
Condition
hypersensitivity
NCT02041299_exc_T17
Condition
idiosyncratic reaction
NCT02041299_exc_T18
Drug
deferiprone
NCT02041299_exc_T19
Drug
deferoxamine
NCT02041299_exc_T20
Scope
deferiprone or deferoxamine
NCT02041299_exc_T21
Scope
hypersensitivity or idiosyncratic reaction
NCT02041299_exc_T22
Drug
hydroxyurea
NCT02041299_exc_T23
Temporal
within 30 days
NCT02041299_exc_T24
Condition
malignancy
NCT02041299_exc_T25
Value
abnormal
NCT02041299_exc_T26
Measurement
liver function
NCT02041299_exc_T27
Measurement
serum ALT level(s)
NCT02041299_exc_T28
Value
> 5 times upper limit of normal
NCT02041299_exc_T29
Temporal
at screening
NCT02041299_exc_T30
Measurement
creatinine levels
NCT02041299_exc_T31
Value
>2 times upper limit of normal
NCT02041299_exc_T32
Temporal
at screening
NCT02041299_exc_T33
Scope
serum ALT level(s) > 5 times upper limit of normal at screening or creatinine levels >2 times upper limit of normal at screening
NCT02041299_exc_T34
Scope
abnormal liver function
NCT02041299_exc_T35
Condition
unstable illness
NCT02041299_exc_T36
Qualifier
serious
NCT02041299_exc_T37
Qualifier
as judged by the Investigator
NCT02041299_exc_T38
Temporal
during the past 3 months before screening/baseline visit
NCT02041299_exc_T39
Condition
immunologic disease
NCT02041299_exc_T40
Condition
or immunol inicall
NCT02041299_exc_T41
Condition
gic, neurologi inicall
NCT02041299_exc_T42
Condition
lar, endocrino inicall
NCT02041299_exc_T43
Condition
, cardiovas inicall
NCT02041299_exc_T44
Condition
rologic, respirato inicall
NCT02041299_exc_T45
Condition
ro-en inicall
NCT02041299_exc_T46
Condition
nal, ga inicall
NCT02041299_exc_T47
Scope
hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease
NCT02041299_exc_T48
Qualifier
Clinically significant
NCT02041299_exc_T49
Qualifier
abnormal
NCT02041299_exc_T50
Procedure
12-lead ECG
NCT02041299_exc_T51
Condition
findings
NCT02041299_exc_T52
Measurement
Cardiac MRI T2*
NCT02041299_exc_T53
Value
<10ms
NCT02041299_exc_T54
Condition
Myocardial infarction
NCT02041299_exc_T55
Condition
cardiac arrest
NCT02041299_exc_T56
Condition
cardiac failure
NCT02041299_exc_T57
Temporal
within 1 year before screening/baseline visit
NCT02041299_exc_T58
Scope
Myocardial infarction, cardiac arrest or cardiac failure
NCT02041299_exc_T59
Condition
Unable to undergo
NCT02041299_exc_T60
Procedure
MRI