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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-18

Study Completion Date

2017-08-17

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.

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Detailed Description

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Conditions

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Congenital Adrenal Hyperplasia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

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).

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ATR-101

Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.

Group Type EXPERIMENTAL

ATR-101

Intervention Type DRUG

125-1000 mg twice per week

Interventions

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ATR-101

125-1000 mg twice per week

Intervention Type DRUG

Other Intervention Names

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Nevanimibe hydrochloride

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

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No