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A study of pentosan polysulfate sodium in sickle cell disease patients

  • Research type

    Research Study

  • Full title

    A randomized, double-blind, placebo-controlled study to assess the safety and activity of pentosan polysulfate sodium on microvascular blood flow, vascular endothelial injury and vasoocclusive pain in patients with sickle cell disease.

  • Sponsor organisation

    TRF Pharma Inc

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  • Identifier


  • Research summary

    Sickle cell disease is an inherited condition associated with episodes of severe pain which are due to the blockage of small blood vessels. This blockage is due to a combination of abnormal red blood cells, abnormal blood vessel walls and increased adhesion molecules, which increase the stickiness of red cells. Pentosan polysulfate sodium (PPS) has been shown to block these adhesion molecules and decrease the stickiness of sickle blood, in laboratory and animal models. It is already licenced for the treatment of pain in interstitial cystitis and has few side effects. It is an oral drug taken once a day. The aims of this trial are to assess the safety and tolerability of PPS, to assess how long PPS stays in the body and to assess the effect of PPS on the blood flow in small blood vessels and on the incidence of painful episodes in patients with Sickle Cell Disease. Patients 18 years or older, with HbSS or HbS-B0thalassaemia and who have had2-10 documented pain crises in the last year will be eligible for the trial. This trial is a randomised double blind trial, which will treat half of the patients with PPS and half with a placebo. The study consists of a 3 week screening period, 12 weeks of treatment and a 4 week follow up. It will involve10 clinic visits and at these visits blood tests will be taken and a laser Doppler test will be performed. This is an ultrasound of blood flow in the forearm. Participants will have to make a phone call to an electronic diary every day during the study.

  • REC name

    London - Central Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    14 Oct 2008

  • REC opinion

    Further Information Favourable Opinion