Long-term Study of Ferriprox® in Patients with Sickle Cell Disease

• Research type

  Research Study

• Full title

  Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients with Sickle Cell Disease or Other Anemias

• IRAS ID

  185878

• Contact name

  Dianne Zarobiak

• Contact email

  dzarobia@apopharma.com

• Sponsor organisation

  ApoPharma Inc.

• Eudract number

  2014-005685-30

• Clinicaltrials.gov Identifier

  NCT02443545

• Clinicaltrials.gov Identifier

  045724, IND number

• Duration of Study in the UK

  2 years, 6 months, 30 days

• Research summary

  Sickle cell disease is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Rather than being round and flexible, some of the red blood cells sickle, which is their shape, is
  like a crescent. The abnormal blood cells have a shorter lifespan and are not replaced as quickly as normal; this leads to a shortage of red blood cell, called anaemia. Symptoms of anaemia include tiredness and breathlessness; especially after exercise.

  Some patients with sickle cell disease or some other anaemias may require regular blood transfusions to replace their sickle red blood cells with healthy ones. Such transfusions are lifesaving, but over time, they cause a condition called iron overload, a buildup of iron in blood and organs which can lead to serious medical problems. Deferiprone (brand name Ferriprox®) is a drug that acts to remove extra iron from the body.

  The main study (LA38-0411) is investigating the safety and efficacy of deferiprone in sickle cell patients. The reason for the extension study (LA38-EXT) is to collect long-term safety information about the use of deferiprone.

  In the main study LA38-0411, patients were randomized (assigned by chance) to either deferiprone or deferoxamine, for 1 year. In this extension, everyone will get deferiprone for 2 years.

  Deferiprone is taken orally (by mouth). Patients have the choice to take in tablet or liquid form.The daily dosage depends on the patient’s weight, the level of iron overload, and how much extra iron the patient continues to receive during the study through blood transfusions.

  LA38-EXT is expected to be carried out at the same treatment centers (close to 30) in North America, South America, Europe, Egypt, and the Middle East that are carrying out LA38-0411. About 300 patients are expected to take part in the first study, and everyone who completes that study is eligible to enroll in the extension study.

• REC name

  HSC REC B

• REC reference

  15/NI/0188

• Date of REC Opinion

  18 Sep 2015

• REC opinion

  Favourable Opinion