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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03053271
Recruitment Status : Terminated (Slow enrollment)
First Posted : February 15, 2017
Last Update Posted : September 19, 2019
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc. ( Millendo Therapeutics US, 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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 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 : April 13, 2017
Actual Primary Completion Date : June 17, 2019
Actual Study Completion Date : August 12, 2019

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 ]


Information from the National Library of Medicine

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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


Locations
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United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Ohio
The Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53051
United Kingdom
The James Cook University Hospital
Middlesbrough, United Kingdom, TS4 3 BW
Sponsors and Collaborators
Millendo Therapeutics US, Inc.
Investigators
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Principal Investigator: James Findling, MD Medical College of Wisconsin

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Responsible Party: Millendo Therapeutics US, Inc.
ClinicalTrials.gov Identifier: NCT03053271    
Other Study ID Numbers: ATR-101-301
First Posted: February 15, 2017    Key Record Dates
Last Update Posted: September 19, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Millendo Therapeutics, Inc. ( Millendo Therapeutics US, Inc. ):
Endogenous Cushing's Syndrome
ATR-101
CS
Additional relevant MeSH terms:
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Cushing Syndrome
Syndrome
Disease
Pathologic Processes
Adrenocortical Hyperfunction
Adrenal Gland Diseases
Endocrine System Diseases