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A Study of ATR-101 for the Treatment of Endogenous Cushing's Syndrome

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ClinicalTrials.gov Identifier: NCT03053271
Recruitment Status : Recruiting
First Posted : February 15, 2017
Last Update Posted : October 25, 2018
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc.

Brief Summary:
This is a Phase 2 multicenter, randomized, double-blind, placebo controlled study of ATR-101 to evaluate the efficacy and safety of orally-administered ATR-101 in adults with endogenous Cushing's syndrome. Following wash-out (if needed), all eligible subjects will enter an open-label intra-subject dose-escalation period of 8 weeks' duration, followed either by a double-blind randomized withdrawal period of 4 weeks' duration (if the subject meets randomization criteria) or by an additional open label dosing period of 4 weeks' duration (if the subject does not meet randomization criteria).It is anticipated that the overall duration of the study per subject will range from approximately 16-22 weeks.

Condition or disease Intervention/treatment Phase
Cushing Syndrome Drug: ATR-101 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ATR-101 for the Treatment of Cushing's Syndrome
Actual Study Start Date : February 16, 2017
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : November 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: ATR-101
During the 4-week randomized withdrawal period, eligible subjects will receive ATR-101 at the same dose level being used at the completion of the open-label dose-escalation period.
Drug: ATR-101
During the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks at the same dose level being used at the completion of the open-label dose-escalation period.

Placebo Comparator: Placebo
During the 4-week randomized withdrawal period, eligible subjects will receive a placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period.
Drug: Placebo
During the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks with placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period.




Primary Outcome Measures :
  1. The proportion of subjects with either a normal 24-hr urinary free cortisol (UFC) or a reduction in 24-hr UFC of ≥ 50% relative to their baseline value [ Time Frame: Through Day 85 ]

Secondary Outcome Measures :
  1. The proportion of subjects with a normal 24-hr UFC [ Time Frame: Through Day 85 ]
  2. The proportion of subjects with a reduction in 24-hr UFC of ≥ 50% relative to their baseline value [ Time Frame: Through Day 85 ]
  3. The proportion of subjects with a normal 24-hr UFC [ Time Frame: Through Day 57 and Day 85 ]
  4. The proportion of subjects with a reduction in 24-hr UFC of ≥ 50% relative to their baseline value [ Time Frame: Through Day 57 and Day 85 ]
  5. The change in the 24-hr UFC [ Time Frame: From Day 57 to Day 85 ]
  6. The change in the 24-hr UFC [ Time Frame: Through Day 57 and Day 85 ]
  7. The proportion of subjects with a normal late night salivary cortisol [ Time Frame: Through Day 85 ]
  8. The proportion of subjects with a normal late night salivary cortisol [ Time Frame: Through Day 57 and Day 85 ]
  9. The change in the late night salivary cortisol [ Time Frame: From Day 57 to Day 85 ]
  10. The change in the late night salivary cortisol [ Time Frame: Through Day 57 and Day 85 ]
  11. The change from baseline in blood hormone levels [ Time Frame: Through study completion, up to 22 weeks ]


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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed diagnosis of endogenous Cushing's syndrome
  • Baseline UFC 1.3 to 10 × upper limit of normal (ULN)
  • If previous pituitary surgery, participants must be at least 3 months since surgery at the time of screening
  • BMI between 18 and 60 kg/m2, inclusive

Exclusion Criteria:

  • Pseudo-Cushing's syndrome, cyclic Cushing's syndrome or current iatrogenic Cushing's syndrome
  • Candidates for surgical treatment of Cushing's syndrome, unless surgery is not anticipated to occur during the study
  • Normal late night salivary cortisol or 24-hr urine free cortisol
  • Radiotherapy of the pituitary within 6 months

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03053271


Contacts
Contact: Marian Ijzerman, PhD 734-845-9300 ijzerman@millendo.com
Contact: Marianne Plaunt, PhD 734-249-6030 plaunt@millendo.com

Locations
United States, Alabama
University of Alabama - Birmingham Recruiting
Birmingham, Alabama, United States, 35294
Contact: Tammy Perkins Wilson    205-934-4112    tlperkins@uabmc.edu   
Principal Investigator: T. Brooks Vaughan III, MD         
United States, Arizona
St Joseph's Hospital and Medical Center Not yet recruiting
Phoenix, Arizona, United States, 85013
Contact: Brittany Maykowski    602-406-6274    brittany.maykowski@dignityhealth.org   
Principal Investigator: Kevin Yuen, MD         
United States, California
Keck School of Medicine at USC Medical Center Not yet recruiting
Los Angeles, California, United States, 90033
Contact: Monica Chui    323-865-1225    monica.chiu@med.usc.edu   
Principal Investigator: John Carmichael, MD         
United States, Massachusetts
Massachusetts General Hospital Not yet recruiting
Boston, Massachusetts, United States, 02114
Contact: Karen Liebert    617-726-7473    pulaski@helix.mgh.harvard.edu   
Principal Investigator: Beverly Biller, MD         
United States, Minnesota
Mayo Clinic Active, not recruiting
Rochester, Minnesota, United States, 55905
United States, Ohio
The Cleveland Clinic Foundation Recruiting
Cleveland, Ohio, United States, 44195
Contact: Ana Surckla, CRC    216-444-7955    asurckla@ccf.org   
Principal Investigator: Laurence Kennedy, MD         
Ohio State University Recruiting
Columbus, Ohio, United States, 43203
Contact: Angela Howell, LPN    614-688-3785    angela.howell@osumc.edu   
Principal Investigator: Lawrence Kirschner, MD         
United States, Oregon
Oregon Health and Sciences University Recruiting
Portland, Oregon, United States, 97239
Contact: Jessica Williams, CRC    503-494-9546    nelsjess@ohsu.edu   
Principal Investigator: Maria Fleseriu, MD         
United States, Wisconsin
Medical College of Wisconsin Active, not recruiting
Milwaukee, Wisconsin, United States, 53051
United Kingdom
Royal Bournemouth Hospital Recruiting
Bournemouth, Dorset, United Kingdom, BH7 7DW
Contact: Becci Watling    +44 120 270 4344    becci.watling@rbch.nhs.uk   
Principal Investigator: Tristan Richardson, MD         
King George Hospital Recruiting
Ilford, Essex, United Kingdom, IG3 8YB
Contact: Elisa Visentin       elisa.visentin2@bhrhospitals.nhs.uk   
Principal Investigator: Khashaiar Nikookam, MD         
Salford Royal NHS Foundation Trust Recruiting
Manchester, Salford, United Kingdom, M6 8HD
Contact: Nicola Proudfoot    +44 161 206 5709    nicola.proudfoot@srft.nhs.uk   
Principal Investigator: Tara Kearney, MD         
The James Cook University Hospital Recruiting
Middlesbrough, United Kingdom, TS4 3 BW
Contact: Sue Winship    44 1642 854140    suewinship@nhs.net   
Principal Investigator: Arutchelvam Vijayaraman, MD         
Sponsors and Collaborators
Millendo Therapeutics, Inc.
Investigators
Principal Investigator: James Findling, MD Medical College of Wisconsin

Responsible Party: Millendo Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03053271     History of Changes
Other Study ID Numbers: ATR-101-301
First Posted: February 15, 2017    Key Record Dates
Last Update Posted: October 25, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Millendo Therapeutics, Inc.:
Endogenous Cushing's Syndrome
ATR-101
CS

Additional relevant MeSH terms:
Syndrome
Cushing Syndrome
Disease
Pathologic Processes
Adrenocortical Hyperfunction
Adrenal Gland Diseases
Endocrine System Diseases