Phase 3 study of Luspatercept in patients with MDS

• Research type

  Research Study

• Full title

  A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of\nLuspatercept (ACE-536) Versus Placebo for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Subjects with Ring Sideroblasts Who Require Red Blood Cell Transfusions

• IRAS ID

  198484

• Contact name

  Ghulam Mufti

• Contact email

  ghulam.mufti@kcl.ac.uk

• Sponsor organisation

  Celgene Corporation

• Eudract number

  2015-003454-41

• Clinicaltrials.gov Identifier

  NCT02631070

• Clinicaltrials.gov Identifier

  112 562, IND Number

• Duration of Study in the UK

  3 years, 5 months, 25 days

• Research summary

  Summary of Research
  Myelodysplastic syndrome (MDS) is a bone marrow disorder which causes anaemia. The standard treatment for the loss of red blood cells is supportive treatment with erythropoiesis-stimulating agents (ESAs) and administration of red blood cell (RBC) transfusions.\n\nLuspatercept is being developed to treat a number of conditions including anaemia. Luspatercept works by increasing the production of red blood cells. Preliminary Phase 2 data suggest Luspatercept is likely to attenuate ineffective erythropoiesis and correct the anaemia that characterises MDS and could provide significant clinical benefit to patients by improving haemoglobin levels and reducing the need for regular RBC transfusions.\n\nThe main purpose of this study is to see whether people with anaemia due to MDS will experience an increase in their haemoglobin, and therefore a reduction in their dependence on blood transfusions, if they take Luspatercept. The safety of Luspatercept will be also evaluated. \n\nFollowing a 5 week screening period, 67% of eligible participants will be randomised to receive Luspatercept and 33% will receive placebo. After a 24 week primary phase treatment period, participants who benefit from the study drug and whose MDS is not worsening will enter the extension phase treatment period and will be assessed approximately every 24 weeks.\n\nParticipants will enter a post-treatment follow-up period of at least 2 years from the time they stop the study drug, which encompasses a 6 week follow-up assessment, a 12 week follow-up assessment, and additional follow-up assessments every 3 months thereafter.\n\nStudy drug will be administered subcutaneously on Day 1 of every 21-day treatment cycle during the primary phase and on Day 1 of every 21- day treatment cycle during the extension phase. Study procedures will include blood, urine, bone marrow and PK sampling, ECG and QOL questionnaires.\n\n210 participants will be enrolled worldwide. The study is sponsored by Celgene Corporation.

  Summary of Results
  Positive outcome has been achieved and outlined in the following link
  https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbRehJ-2Fi4xyo44sEgJVCl5Bdj8o1FpylFwbn362DUDJ-2Fz6KgfOIyFXwiAN4hVgMsYJgUlX4-2FlVYNlnx-2BPBz37QSQ-3DTpKz_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YIWYRqc7v7qUQlJ9uJkYVcQckW6z5TadxLRnSP3i0Nfss6henOBzZ02F0vn6TFG5qbwteWJuUtYdDH9JK-2FL7WjdgUuyd0xwgzdOMI-2FnfBYqGV-2BKrTQrIve38nnn-2FI2jqsD22bvNSfUKZrUSGdSX9nPgpSAlkcPkdtZj6oDGDXZT13m85lN2XajYe1SwMybVaX8-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7Cd673bc48e5e14a912d6608da42e2f6a6%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C637895839805568302%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=GLOiHJ4yXRMW41ulMk7GKY2Ma%2FQsVWVqHkzTsuwS62Q%3D&reserved=0

• REC name

  London - Brighton & Sussex Research Ethics Committee

• REC reference

  16/LO/0681

• Date of REC Opinion

  21 Jun 2016

• REC opinion

  Further Information Favourable Opinion