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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02804178
• Date of registration: 01/06/2016
• Prospective Registration: No
• Primary sponsor: Millendo Therapeutics, Inc.
• Public title: A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
• Scientific title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
• Date of first enrolment: May 18, 2016
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02804178
• Study type: Interventional
• Study design: Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Single (Participant).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Documented historical diagnosis of classic CAH due to 21-OHD based on: Documented
genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or
historical documentation of elevated 17-OHP

- Biochemical marker of disease status of 17-OHP = 4 X ULN

- 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Congenital Adrenal Hyperplasia

Intervention(s)

Drug: ATR-101

Primary Outcome(s)

Reduction of 17-OHP to [Time Frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.]

Secondary Outcome(s)

Pharmacokinetics: Time to maximum concentration [Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4, hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID.]

Number of participants with abnormal laboratory values and/or adverse events that are related to treatment [Time Frame: Baseline, Day 1 and Day 15 of each dose level. Each subject will have up to 5 dose levels.]

Pharmacokinetics: Maximum plasma concentration [Time Frame: Evaluated at Day 1 (predose, 1, 2, 4, hours post-dose and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID.]

Pharmacokinetics: Area under the curve [Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4 hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID.]

Secondary ID(s)

ATR-101-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors