Pasireotide Therapy in Patients with Nelson's Syndrome Version 1.1

• Research type

  Research Study

• Full title

  An Open Label, Longitudinal Study of the Effects of Subcutaneous Acute and Chronic Pasireotide (SOM230) Therapy on Adrenocorticotrophic Hormone and Tumour Volume in Patients with Nelson's Syndrome.

• IRAS ID

  7118

• Contact name

  j newellprice

• Sponsor organisation

  Sheffield Teaching Hospitals NHS Foundation Trust

• Eudract number

  2009-014457-33

• ISRCTN Number

  N/A

• Research summary

  Nelson's syndrome, an expanding pituitary tumour, occurs in up to 30% of adults after bilateral adrenalectomy for Cushing's disease, for which no medical treatment exists. Plasma Adrenocorticotrophic hormone (ACTH) levels in these patients remain high, they are characteristically deeply pigmented, and may experience neurological effects as a consequence of the tumour. It is not known whether the tumour growth is due to the lack of cortisol feedback after adrenalectomy or whether the pituitary cells were pre-programmed to develop into a tumour. There is a real need for an effective medical management for Nelson??s syndrome. This is especially true given the increasing data on the somewhat disappointing long-term outcome of transsphenoidal surgery, and the increasing use of laparoscopic bilateral adrenalectomy for failures of pituitary surgery or even as primary therapy for Cushing's disease. Therefore, it is likely that there will be increasing numbers of patients attending endocrine centres worldwide with Nelson??s syndrome following bilateral adrenalectomy as part of their management for Cushing??s disease. In view of this it is important to investigate all potential avenues for the treatment of Nelson??s syndrome and translate any benefits to patients. This study, designed and initiated by the investigators, will assess if pasireotide reduces ACTH levels and tumour volume in patients with Nelson's syndrome. Patients will be recruited for a period of 32 weeks and receive 4 weeks of pasireotide twice daily and then 24 weeks of pasireotide long acting release therapy every 4 weeks. Over the 32 week protocol patients will make 12 visits for serial ACTH blood measurements and have 2 MRI scans to assess tumour volume.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  10/H1005/53

• Date of REC Opinion

  30 Sep 2010

• REC opinion

  Further Information Favourable Opinion