Nevanimibe HCl for the Treatment of Classic CAH

Congenital Adrenal Hyperplasia Clinical Trial

Official title:

A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03669549
• Other study ID #: ATR-101-202
• Status: Terminated
• Phase: Phase 2
• Start date: July 11, 2018
• Est. completion date: July 12, 2020

Study information

• Verified date: February 2021
• Source: Millendo Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: July 12, 2020
• Est. primary completion date: June 3, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• Nevanimibe hydrochloride
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.

Locations

Country	Name	City	State
Brazil	University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto	Ribeirão Preto
Brazil	Hospital das Clínicas da FMUSP - Prédio do Instituto Central	São Paulo
Brazil	Universidade Federal de São Paulo, Escola Paulista de Medicina	São Paulo
Czechia	Institute of Endocrinology	Praha 1
France	Hospital Pitié-Salpetrière	Paris
Israel	Bnai Zion Medical Center	Haifa
Israel	Beilinson Hospital	Petach Tikva
Israel	Tel-Aviv-Sourasky Medical Center	Tel Aviv
Spain	Hospital General Universitario Gregorio Marañón	Madrid
Spain	Hospital Universitario Virgen del Rocío	Sevilla
Spain	University Hospital La Fe	Valencia

Sponsors (1)

Lead Sponsor	Collaborator
Millendo Therapeutics US, Inc.

Countries where clinical trial is conducted

Brazil,  Czechia,  France,  Israel,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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))	Through Day 113