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

Millendo Therapeutics

Status and phase: Completed; Phase 2

Conditions: Congenital Adrenal Hyperplasia

Treatments: Drug: ATR-101

Study type: Interventional

Funder types: Industry

Identifiers

NCT02804178

ATR-101-201

Details and patient eligibility

About

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.

Enrollment: 10 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

ATR-101

Experimental group

Description:

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

Treatment:

Drug: ATR-101