CompletedPhase 2

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

United States10 participantsStarted 2016-05-18

Plain-language 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.

Who can participate

Age range18 Years – 80 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

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

Timeframe: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.

Trial details

NCT IDNCT02804178

SponsorMillendo Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-08-17

Results submitted2021-01-27