Nelson Syndrome Clinical Trial
Official 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
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 preprogrammed 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 longterm outcome of
transsphenoidal surgery, and the increasing use of aparoscopic 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.
As above ;
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00958841 -
Study of Pasireotide in Patients With Rare Tumors of Neuroendocrine Origin
|
Phase 2 |