Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, beta-thalassemia and Diamond-Blackfan anemia
Conditions
Interventions
Product Name: SSP-004184AQ 50 mg Capsule
Product Code: SSP-004184AQ 50 mg
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: deferitazole
CAS Number: 1173095-59-5
Current Sponsor code: SSP-004184
Sponsors
Shire Development LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: For all subjects: 1. Subjects and/or parents willing and able to sign the approved informed consent/and assent (based on institutional guidelines). 2. An understanding, ability, and willingness to fully comply with study procedures and restrictions. 3. Has either completed a previous SSP-004184AQ study or prematurely discontinued (with agreement from the Investigator and Shire physician) from 1 of the SSP-004184AQ studies. 4. If applicable, female subjects should be either: • Post-menopausal (12 consecutive months of spontaneous amenorrhea), or • Surgically sterile, or • Females of child-bearing potential must have a negative urine pregnancy test at the Qualification and Enrollment Visit prior to dosing on Day 1. 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 For subjects that did not transition directly from their feeder protocol and have initiated treatment with a chelator other than SSP-004184AQ: 5. Willing to discontinue all existing iron chelation therapies for a minimum period of 1 to 5 days prior to first dose of SSP-004184AQ. 6. (Note: This inclusion criterion is also applicable for subjects that were receiving chelators other than SSP-004184AQ in their feeder protocol.) 7. Serum ferritin >500 ng/mL at the Qualification and Enrollment Visit. 8. Liver iron concentration (LIC) =2.0 mg iron per g (equivalent dry weight, liver) determined by FerriScan® R2 MRI at the Qualification and Enrollment Visit (Day -28 to Day -8). (Note: Younger subjects for whom MRI is not feasible will be considered iron overloaded on the basis of serum ferritin only.) 9. Mean of the previous 3 pre-transfusion hemoglobin concentrations =7.5 g/dL Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1320 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 150
Exclusion criteria
Exclusion criteria: For all subjects: 1. Unwilling to remain off all other existing chelation therapies during SSP-004184AQ dosing and for up to 24 hours from last dose. 2. A history of non-compliance in a prior FerroKin/Shire-sponsored SSP-004184AQ study (excluding dose suspensions that were medically warranted). 3. Cardiac MRI T2* 200 IU/L at the Qualification and Enrollment Visit. OR For subjects 180 IU/L at the Qualification and Enrollment Visit. 12. For subjects =18 years old: Evidence of severe renal insufficiency, eg, serum creatinine 1.5X above the upper limit of normal or proteinuria greater than 1 gm per day or a calculated glomerular filtration rate <40 mL/min. OR For subjects <18 years old: Evidence of significant renal insufficiency, eg, serum creatinine above the upper limit of normal or proteinuria greater than 1 gm per day. 13. Use of any investigational agent within the 30 days prior to receiving the first dose of SSP-004184AQ. 14. Cardiac left ventricular ejection fraction below the locally determined normal range in the 12 months prior to Screening by echocardiography or MRI or <50% at Baseline testing by MRI (Echocardiograph is acceptable for LVEF if MRI information is not available.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Measure the extent and durability of SSP-004184AQ (SPD602)treatment effects; continue to assess the safety, tolerability and efficacy of SSP-004184AQ in subjects who received SSP-004184AQ in prior study; allow access and assess response to SSP-004184AQ in subjects randomised to another chelating therapy in a prior SSP-004184AQ study. Pharmacokinetic parameters and their relationship to selected efficacy and safety parameters will also be explored. Further details regarding the pharmacokinetic analysis will be provided in the pharmacokinetic analysis plan. ;Secondary Objective: Not applicable;Primary end point(s): Primary as per objectives;Timepoint(s) of evaluation of this end point: 3 years | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): No;Timepoint(s) of evaluation of this end point: No | — |
Countries
Canada, Egypt, Italy, Lebanon, Thailand, Turkey, United Kingdom, United States
Contacts
Public ContactRachel Duncan
Shire Pharmaceutical Development Ltd
Outcome results
None listed