SPD602 Ph2 Study to assess Liver Iron Concentration
Research type
Research Study
Full title
A 48-week, Open-label, 2-arm, Parallel-group, Randomised Exploratory Study to Assess Liver Iron Concentration measured by FerriScan® (R2) Magnetic Resonance Imaging in ß-thalassemia Subjects Administered SPD602 (SSP-004184AQ)or Exjade® (deferasirox)for treatment of Chronic Transfusional Iron Overload
IRAS ID
135240
Contact name
Kate Ryan
Contact email
Sponsor organisation
Shire Development LLC and International Affiliates
Eudract number
2013-000743-33
Clinicaltrials.gov Identifier
Research summary
β-thalassemia is a blood disorder that reduces the production of haemoglobin. Haemoglobin is the iron-containing protein in the red blood cells that carries oxygen to cells throughout the body. Therefore people who suffer from β-thalassemia require regular blood transfusions to maintain their haemoglobin levels and to suppress endogenous production of red blood cells due to chronic rupturing of their red blood cells. While the blood transfusions will result in the effective management of the β-thalassemia, it would inevitably lead to an iron overload. Studies in patients who received in excess of 20 blood transfusions suggested that they were at an increased risk of developing signs or symptoms of an iron overload.
As human beings lack an effective mechanism to excrete the excess iron due to the blood transfusions, iron chelation treatments are used. The main goal of this treatment would be to eliminate the excess tissue iron and to compensate for the iron intake associated with ongoing transfusions. There are currently three iron chelating agents approved for the treatment of transfusional iron overload; however each has properties that diminish its optimal performance. There is currently no marketed chelator that demonstrates improved safety, tolerability and ease of use.
SSP-004184AQ is a new iron chelator being developed for the treatment of chronic iron overload in patients with transfusion-dependent blood disorders. Data taken from non-clinical and preliminary data suggests that SSP-004184 has the potential for improved safety and tolerability in comparison to the current standard of care treatment.
This study is an open-label, 2-arm, parallel-group, randomized multicentre study to evaluate the effectiveness and safety of treatment with SSP-004184AQ in comparison with the standard of care in adult patients with β-thalassemia who have transfusional iron overload.
REC name
North West - Haydock Research Ethics Committee
REC reference
13/NW/0841
Date of REC Opinion
3 Feb 2014
REC opinion
Further Information Favourable Opinion