Phase 2 N=10 Treatment

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

Congenital Adrenal Hyperplasia

Bottom Line

View on ClinicalTrials.gov: NCT02804178 ↗

Enrolled (actual)

Serious AEs

10.0%

Results posted

Feb 2021

Primary outcome: Primary: 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 — 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ATR-101 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Millendo Therapeutics, Inc.
Primary completion: Aug 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY 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	2	—

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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02804178). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.