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

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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Key information

Conditions

Congenital Adrenal Hyperplasia Adrenal Gland Diseases Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Congenital Abnormalities Congenital adrenal hyperplasia due to 21 hydroxylase deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Steroid Metabolism, Inborn Errors Urogenital Abnormalities Urogenital Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Johns Hopkins University, Baltimore, Maryland, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ATR-101

Drug

125-1000 mg twice per week

Other names: Nevanimibe hydrochloride

Primary outcomes

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

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

17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.

Sponsors and collaborators

Lead sponsor

Millendo Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start: 2016
Primary completion: 2017
Study completion: 2017
First posted: Jun 17, 2016
Registry last updated: Mar 10, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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