Pasireotide LAR in patients with Cushing’s disease

• Research type

  Research Study

• Full title

  A randomized, double-blind, multicenter, phase III study to evaluate the efficacy and safety of pasireotide LAR in patients with Cushing’s disease

• IRAS ID

  81262

• Contact name

  John Newell-Price

• Sponsor organisation

  Novartis Pharmaceuticals Uk Ltd

• Eudract number

  2009-011128-70

• ISRCTN Number

  n.a

• Clinicaltrials.gov Identifier

  n.a

• Research summary

  Cushing's disease is characterized by several distinctive outwards signs such as a round (moon-shaped) face, purplish streaks across the skin, unusual build up of fatty tissue between the shoulder blades (sometimes called the "buffalo hump". The disease is caused by a small non cancerous tumour (growth of cells) in the pituitary gland, a pea-sized gland below the brain. The tumour causes the pituitary gland to release a hormone which stimulates the adrenal glands (two small organs just above the kidneys) to produce excessive levels of cortisol (a steroid hormone).Currently, there is no approved medical therapy for the treatment of Cushing's disease. The new drug, Pasireotide is expected to be the first drug licensed for the medical treatment of patients with Cushing??s disease who are not cured by surgery or who are ineligible for surgery. Pasireotide has a unique high binding affinity to somatostatin receptor subtype 5, which is predominant in the ACTH-secreting tumours that cause Cushing??s disease. By blocking this receptor, Pasireotide reduces ACTH secretion from the tumour and hence lowers cortisol levels secreted by the adrenal glands. Although this represents another, much needed, treatment option for patients, a long acting formulation of pasireotide (pasireotide LAR) has been developed to reduce the number of injections from twice a day to once a month in order to enhance convenience and improve its effectiveness.This study will evaluate the safety and efficacy of pasireotide LAR 10mg and pasireotide LAR 30mg in patients with Cushing??s disease patients for whom surgery is not an option. The primary end-point will be normalization of mUFC (after seven months of treatment), a well established therapeutic goal for the treatment of Cushing??s disease.Approximately 148 patients will be enrolled into the core study. The duration of their participation in the core study is 12 months. Patients can continue in the extension study for a further 12 months. Participants will be asked to come to the hospital at least every month and on some occasions twice a month for medical assessments.Novartis is the sponsor of this clinical trial.

• REC name

  East of England - Cambridge South Research Ethics Committee

• REC reference

  11/EE/0229

• Date of REC Opinion

  10 Aug 2011

• REC opinion

  Further Information Favourable Opinion