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Nevanimibe HCl for the Treatment of Classic CAH

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Nevanimibe hydrochloride
Sponsored by
Millendo Therapeutics US, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring CAH, nevanimibe

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto
  • Universidade Federal de São Paulo, Escola Paulista de Medicina
  • Hospital das Clínicas da FMUSP - Prédio do Instituto Central
  • Institute of Endocrinology
  • Hospital Pitié-Salpetrière
  • Bnai Zion Medical Center
  • Beilinson Hospital
  • Tel-Aviv-Sourasky Medical Center
  • Hospital General Universitario Gregorio Marañón
  • Hospital Universitario Virgen del Rocío
  • University Hospital La Fe

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Nevanimibe hydrochloride

Arm Description

Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID

Outcomes

Primary Outcome Measures

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))

Secondary Outcome Measures

Full Information

First Posted
September 10, 2018
Last Updated
February 11, 2021
Sponsor
Millendo Therapeutics US, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03669549
Brief Title
Nevanimibe HCl for the Treatment of Classic CAH
Official Title
A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
Study Type
Interventional

2. Study Status

Record Verification Date
February 2021
Overall Recruitment Status
Terminated
Why Stopped
Following an interim data review, further investment in nevanimibe has been discontinued
Study Start Date
July 11, 2018 (Actual)
Primary Completion Date
June 3, 2020 (Actual)
Study Completion Date
July 12, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Millendo Therapeutics US, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia
Keywords
CAH, nevanimibe

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Nevanimibe hydrochloride
Arm Type
Experimental
Arm Description
Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID
Intervention Type
Drug
Intervention Name(s)
Nevanimibe hydrochloride
Intervention Description
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.
Primary Outcome Measure Information:
Title
Percentage of Subjects Achieving Serum 17-OHP Targets
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto
City
Ribeirão Preto
ZIP/Postal Code
14051-140
Country
Brazil
Facility Name
Universidade Federal de São Paulo, Escola Paulista de Medicina
City
São Paulo
ZIP/Postal Code
04037-002
Country
Brazil
Facility Name
Hospital das Clínicas da FMUSP - Prédio do Instituto Central
City
São Paulo
ZIP/Postal Code
05403-900
Country
Brazil
Facility Name
Institute of Endocrinology
City
Praha 1
Country
Czechia
Facility Name
Hospital Pitié-Salpetrière
City
Paris
Country
France
Facility Name
Bnai Zion Medical Center
City
Haifa
Country
Israel
Facility Name
Beilinson Hospital
City
Petach Tikva
Country
Israel
Facility Name
Tel-Aviv-Sourasky Medical Center
City
Tel Aviv
Country
Israel
Facility Name
Hospital General Universitario Gregorio Marañón
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocío
City
Sevilla
Country
Spain
Facility Name
University Hospital La Fe
City
Valencia
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
35283460
Citation
White PC. Emerging treatment for congenital adrenal hyperplasia. Curr Opin Endocrinol Diabetes Obes. 2022 Jun 1;29(3):271-276. doi: 10.1097/MED.0000000000000723.
Results Reference
derived

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Nevanimibe HCl for the Treatment of Classic CAH

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