A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

Study Overview
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Brief Summary
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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.

Official Title
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A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

Conditions
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Congenital Adrenal Hyperplasia

Intervention / Treatment
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* Drug: ATR-101


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional 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)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ATR-101<br>Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day. | Drug: ATR-101<br>* 125-1000 mg twice per week<br>* Other names: Nevanimibe hydrochloride;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ATR-101, classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, CAH