Back to resultsA Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Primary Purpose
Congenital Adrenal Hyperplasia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ATR-101
Sponsored by
About this trial
This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring ATR-101, classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, CAH
Eligibility Criteria
Inclusion Criteria:
- Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone
- Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal
- Chronic glucocorticoid replacement therapy for at least 6 consecutive months
- Stable glucocorticoid and mineralocorticoid regimen for at least 1 month
Exclusion Criteria:
- Non-classic CAH
- Other causes of adrenal insufficiency
- Surgery within the previous 3 months prior to screening or planned surgery during study participation
- History of active cancer requiring medical or surgical therapy within the past 6 months
Sites / Locations
- Johns Hopkins University
- National Institutes of Health Clinical Center
- University of Michigan
- Mayo Clinic - Rochester
- The University of Oklahoma - Tulsa Schusterman Center
- The Children's Hospital of Philadelphia
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ATR-101
Arm Description
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
Outcomes
Primary Outcome Measures
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
Secondary Outcome Measures
Full Information
NCT ID
NCT02804178
First Posted
June 1, 2016
Last Updated
February 16, 2021
Sponsor
Millendo Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT02804178
Brief Title
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Official Title
A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Study Type
Interventional
2. Study Status
Record Verification Date
February 2021
Overall Recruitment Status
Completed
Study Start Date
May 18, 2016 (Actual)
Primary Completion Date
August 17, 2017 (Actual)
Study Completion Date
August 17, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Millendo Therapeutics, Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia
Keywords
ATR-101, classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, CAH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ATR-101
Arm Type
Experimental
Arm Description
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
Intervention Type
Drug
Intervention Name(s)
ATR-101
Other Intervention Name(s)
Nevanimibe hydrochloride
Intervention Description
125-1000 mg twice per week
Primary Outcome Measure Information:
Title
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
Description
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
Time Frame
Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone
Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal
Chronic glucocorticoid replacement therapy for at least 6 consecutive months
Stable glucocorticoid and mineralocorticoid regimen for at least 1 month
Exclusion Criteria:
Non-classic CAH
Other causes of adrenal insufficiency
Surgery within the previous 3 months prior to screening or planned surgery during study participation
History of active cancer requiring medical or surgical therapy within the past 6 months
Facility Information:
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
National Institutes of Health Clinical Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Mayo Clinic - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
The University of Oklahoma - Tulsa Schusterman Center
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74135
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
10021
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
32589738
Citation
El-Maouche D, Merke DP, Vogiatzi MG, Chang AY, Turcu AF, Joyal EG, Lin VH, Weintraub L, Plaunt MR, Mohideen P, Auchus RJ. A Phase 2, Multicenter Study of Nevanimibe for the Treatment of Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2020 Aug 1;105(8):2771-8. doi: 10.1210/clinem/dgaa381.
Results Reference
derived
Learn more about this trial
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
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