Clinical trial NCT02804178: A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Official title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Congenital Adrenal Hyperplasia
Keywords: ATR-101
classic congenital adrenal hyperplasia
21-hydroxylase deficiency
CAH
Lead sponsor: Millendo Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2016-06-17
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).
Primary purpose: TREATMENT

Brief 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

Primary outcomes:
measure: 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.
Secondary outcomes:
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Tulsa
country: United States
facility: The University of Oklahoma - Tulsa Schusterman Center
state: Oklahoma
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
References:
citation: El-Maouche D, Merke DP, Vogiatzi MG, Chang AY, Turcu AF, Joyal EG, Lin VH, Weintraub L, Plaunt MR, Mohideen P, Auchus RJ. A Phase 2, Multicenter Study of Nevanimibe for the Treatment of Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2020 Aug 1;105(8):2771-8. doi: 10.1210/clinem/dgaa381.