Phase 2 study ATR-101 for the Treatment of Cushing’s Syndrome
Research type
Research Study
Full title
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ATR-101 for the Treatment of Cushing’s Syndrome
IRAS ID
211482
Contact name
Khashaiar Nikookam
Contact email
Sponsor organisation
Millendo Therapeutics, Inc.
Eudract number
2016-002240-17
Clinicaltrials.gov Identifier
129885, IND Number
Duration of Study in the UK
1 years, 1 months, days
Research summary
Cushing’s syndrome is caused by long-term excessive levels of glucocorticoids (hormone) in the body, resulting in weight gain, high blood pressure, skin thinning, and weak bones. Cushing’s syndrome can be known as ‘exogenous’ when the disease has been caused by long-term use of steroid medication, or ‘endogenous’ when the disease has been caused by a tumour in the pituitary gland causing the adrenal glands to produce excessive glucocorticoids. Endogenous Cushing’s syndrome is rare, with an incidence of 2-3 per million per year.
Surgery to remove the tumour is first line of treatment for endogenous Cushing’s syndrome, however the success rate depends on the size of tumour and recurrence rates are high. If surgery fails, radiotherapy can be used to destroy the tumour however success rates are variable and it can take years to take effect. Medication can also be taken to block production of glucocorticoids. There is an unmet need for the use of a treatment for a broader subset of Cushing’s syndrome patients with less side effects.
Millendo Therapeutics, Inc. have developed a new drug, ATR-101, that has been designed to inhibit ACAT1, an enzyme that is highly expressed in the adrenal glands, and inhibiting cholesterol ester (a substrate for steroid synthesis) production.
This study is being done to find out the efficacy and safety of ATR-101 when taken orally in adults with endogenous Cushing’s syndrome.
All patients will receive escalating doses of ATR-101 orally daily for 6 weeks in the first part of the study, then patients will be randomly assigned to receive either ATR-101 or placebo (looks like the study drug but with no active ingredient) orally daily for 4 weeks in the second part of the study. Neither the patient nor the study doctor will know which medication is being given in the second part. It is anticipated that approximately 16 patients will be enrolled worldwide.REC name
London - Riverside Research Ethics Committee
REC reference
17/LO/0473
Date of REC Opinion
16 May 2017
REC opinion
Further Information Favourable Opinion