Nevanimibe HCl for the Treatment of Classic CAH

Phase 2TerminatedNCT03669549

Millendo Therapeutics US, Inc.15 enrolled

Overview

This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Congenital Adrenal Hyperplasia

Interventions

Nevanimibe hydrochlorideDRUG

During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency and/or historical documentation of elevated 17-OHP * Chronic glucocorticoid replacement therapy for at least 6 consecutive months prior to screening * Stable glucocorticoid and mineralocorticoid regimen for at least 4 weeks prior to screening and throughout the treatment period of the study Exclusion Criteria: * Nonclassic CAH * Other causes of adrenal insufficiency * HIV, hepatitis B, or hepatitis C * AST or ALT \>2x ULN, bilirubin or serum creatinine \>1.5x ULN

Locations (11)

University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto

Ribeirão Preto, Brazil

Universidade Federal de São Paulo, Escola Paulista de Medicina

São Paulo, Brazil

Hospital das Clínicas da FMUSP - Prédio do Instituto Central

São Paulo, Brazil

Outcomes

Primary Outcomes

Percentage of Subjects Achieving Serum 17-OHP Targets

The primary efficacy endpoint was the overall response rate within each cohort, defined as the percentage of patients achieving serum 17-OHP targets as follows: * Men and postmenopausal women: 17-OHP ≤ 2x ULN * Premenopausal women: * Follicular phase: 17-OHP ≤ 2x follicular phase ULN * Luteal phase: 17-OHP ≤ (2x follicular phase ULN + (luteal phase ULN - follicular phase ULN))

Time frame: Through Day 113

Data from ClinicalTrials.gov

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