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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by Millendo Therapeutics, Inc.
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT03053271
First received: February 6, 2017
Last updated: May 9, 2017
Last verified: March 2017
  Purpose
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, all eligible subjects will undergo a 6-week open-label dose escalation with ATR-101. Subjects who continue to be eligible will then undergo a 4-week randomized withdrawal period. It is anticipated that the overall duration of the study per subject will range from approximately 14-20 weeks.

Condition Intervention Phase
Cushing Syndrome
Drug: ATR-101
Drug: Placebos
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Millendo Therapeutics, Inc.:

Primary Outcome Measures:
  • 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 71 ]

Secondary Outcome Measures:
  • The proportion of subjects with a normal 24-hr UFC [ Time Frame: Through Day 71 ]
  • The proportion of subjects with a reduction in 24-hr UFC of ≥ 50% relative to their baseline value [ Time Frame: Through Day 71 ]
  • The proportion of subjects with a normal 24-hr UFC [ Time Frame: Through Day 43 ]
  • The proportion of subjects with a reduction in 24-hr UFC of ≥ 50% relative to their baseline value [ Time Frame: Through Day 43 ]
  • The change in the 24-hr UFC [ Time Frame: From Day 43 to Day 71 ]
  • The change in the 24-hr UFC [ Time Frame: Through Day 43 ]
  • The proportion of subjects with a normal late night salivary cortisol [ Time Frame: Through Day 71 ]
  • The proportion of subjects with a normal late night salivary cortisol [ Time Frame: Through Day 43 ]
  • The change in the late night salivary cortisol [ Time Frame: From Day 43 to Day 71 ]
  • The change in the late night salivary cortisol [ Time Frame: Through Day 43 ]
  • The change from baseline in blood hormone levels [ Time Frame: Through study completion, up to 20 weeks ]

Estimated Enrollment: 16
Actual Study Start Date: February 16, 2017
Estimated Study Completion Date: November 2018
Estimated Primary Completion Date: November 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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: Placebos
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: Placebos
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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
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 6 months since surgery at the time of screening
  • BMI between 18 and 45 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 1 year
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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: Kentress Davison    205-975-9308    kdavison@uabmc.edu   
Principal Investigator: T. Brooks Vaughan III, MD         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Kerry Crawley, CCRP    507-266-1944    crawley.kerry@mayo.edu   
Principal Investigator: Alice Chang, MD         
United States, Ohio
The Cleveland Clinic Foundation Recruiting
Cleveland, Ohio, United States, 44195
Contact: Amy Orasko, CRC    216-444-3267    oraskoa@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 Not yet 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 Not yet recruiting
Milwaukee, Wisconsin, United States, 53051
Contact: Gerald Coly    414-955-7468    gcoly@mcw.edu   
Principal Investigator: James Findling, MD         
Sponsors and Collaborators
Millendo Therapeutics, Inc.
Investigators
Principal Investigator: James Findling, MD Medical College of Wisconsin
  More Information

Responsible Party: Millendo Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03053271     History of Changes
Other Study ID Numbers: ATR-101-301
Study First Received: February 6, 2017
Last Updated: May 9, 2017
Individual Participant Data  
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

ClinicalTrials.gov processed this record on May 25, 2017