Nevanimibe HCl for the Treatment of Classic CAH

Phase 2

Terminated

• Conditions: Congenital Adrenal Hyperplasia
• Interventions: Drug: Nevanimibe hydrochloride

• Registration Number: NCT03669549
• Lead Sponsor: Millendo Therapeutics US, Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2018-07-11
• Study First Submitted: 2018-09-10
• Study First Submitted QC: 2018-09-11
• Study First Posted: 2018-09-13
• Primary Completion: 2020-06-03
• Study Completion: 2020-07-12
• Results First Submitted: 2021-01-26
• Status Verified: 2021-02
• Results First Submitted QC: 2021-02-11
• Last Update Submitted: 2021-02-11
• Results First Posted: 2021-03-04
• Last Update Posted: 2021-03-04

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Nevanimibe hydrochloride	Nevanimibe hydrochloride	Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID

Primary Outcome Measures

Name	Time	Method
Percentage of Subjects Achieving Serum 17-OHP Targets	Through Day 113	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))

Trial Locations

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

Institute of Endocrinology; 🇨🇿 Praha 1, Czechia

Hospital Pitié-Salpetrière; 🇫🇷 Paris, France

Hospital das Clínicas da FMUSP - Prédio do Instituto Central; 🇧🇷 São Paulo, Brazil

Hospital General Universitario Gregorio Marañón; 🇪🇸 Madrid, Spain

Hospital Universitario Virgen del Rocío; 🇪🇸 Sevilla, Spain

University Hospital La Fe; 🇪🇸 Valencia, Spain

Bnai Zion Medical Center; 🇮🇱 Haifa, Israel

Beilinson Hospital; 🇮🇱 Petach Tikva, Israel

Tel-Aviv-Sourasky Medical Center; 🇮🇱 Tel Aviv, Israel