Nevanimibe HCl for the Treatment of Classic CAH

February 11, 2021 updated by: Millendo Therapeutics US, Inc.

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

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 Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
      • Ribeirão Preto, Brazil, 14051-140
        • University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto
      • São Paulo, Brazil, 04037-002
        • Universidade Federal de São Paulo, Escola Paulista de Medicina
      • São Paulo, Brazil, 05403-900
        • Hospital das Clínicas da FMUSP - Prédio do Instituto Central
  • Czechia
      • Praha 1, Czechia
        • Institute of Endocrinology
  • France
      • Paris, France
        • Hospital Pitie-Salpetriere
  • Israel
      • Haifa, Israel
        • Bnai Zion Medical Center
      • Petach Tikva, Israel
        • Beilinson Hospital
      • Tel Aviv, Israel
        • Tel-Aviv-Sourasky Medical Center
  • Spain
      • Madrid, Spain
        • Hospital General Universitario Gregorio Maranon
      • Sevilla, Spain
        • Hospital Universitario Virgen del Rocío
      • Valencia, Spain
        • University Hospital La Fe

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Nevanimibe hydrochloride
Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Subjects Achieving Serum 17-OHP Targets
Time Frame: 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))
Through Day 113

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Millendo Therapeutics US, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 11, 2018

Primary Completion (Actual)

June 3, 2020

Study Completion (Actual)

July 12, 2020

Study Registration Dates

First Submitted

September 10, 2018

First Submitted That Met QC Criteria

September 11, 2018

First Posted (Actual)

September 13, 2018

Study Record Updates

Last Update Posted (Actual)

March 4, 2021

Last Update Submitted That Met QC Criteria

February 11, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATR-101-202

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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