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Trial record 1 of 1 for:    NCT03669549
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Nevanimibe HCl for the Treatment of Classic CAH

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03669549
Recruitment Status : Recruiting
First Posted : September 13, 2018
Last Update Posted : March 11, 2020
Information provided by (Responsible Party):
Millendo Therapeutics, Inc. ( 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.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Drug: Nevanimibe hydrochloride Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
Actual Study Start Date : July 11, 2018
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

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.

Primary Outcome Measures :
  1. Percentage of subjects achieving serum 17-OHP targets [ Time Frame: Through Day 113 ]
    <= 2x ULN

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03669549

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Contact: Marian Ijzerman, PhD 734-845-9300
Contact: Eric Henricks, BS 734-845-9423

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University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto Recruiting
Ribeirão Preto, Brazil, 14051-140
Contact: Henrique Pereira    (16)3963-6476   
Principal Investigator: Sonir Antonini, MD         
Universidade Federal de São Paulo, Escola Paulista de Medicina Recruiting
São Paulo, Brazil, 04037-002
Contact: Fernanda Carrara    (11) 5579-2581   
Principal Investigator: Claudio Kater, MD         
Hospital das Clínicas da FMUSP - Prédio do Instituto Central Recruiting
São Paulo, Brazil, 05403-900
Contact: Fernanda Pereira de Campos    (11) 2661-7845/7846   
Principal Investigator: Tânia Bachega, MD         
Institute of Endocrinology Completed
Praha 1, Czechia
Hospital Pitié-Salpetrière Recruiting
Paris, France
Contact: Jerome Dulon    +33 142 16 02 11   
Principal Investigator: Philippe Touraine, MD         
Bnai Zion Medical Center Recruiting
Haifa, Israel
Contact: Michal Yeiches    +972-4-8359484   
Principal Investigator: Mohammad Sheikh-Ahmad, MD         
Beilinson Hospital Completed
Petach Tikva, Israel
Tel-Aviv-Sourasky Medical Center Recruiting
Tel Aviv, Israel
Contact: Miri Margaliot    +972-52-6110243   
Principal Investigator: Yael Sofer, MD         
Hospital General Universitario Gregorio Marañón Recruiting
Madrid, Spain
Contact: Sara Calvo    +34 915 86 81 12   
Principal Investigator: Rogelio Garcia Centeno, MD         
Hospital Universitario Virgen del Rocío Recruiting
Sevilla, Spain
Contact: Cristina Muñoz Gómez    +34 955 01 37 24   
Principal Investigator: Miguel Ángel Mangas Cruz, MD         
University Hospital La Fe Completed
Valencia, Spain
Sponsors and Collaborators
Millendo Therapeutics US, Inc.
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Responsible Party: Millendo Therapeutics US, Inc. Identifier: NCT03669549    
Other Study ID Numbers: ATR-101-202
First Posted: September 13, 2018    Key Record Dates
Last Update Posted: March 11, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Millendo Therapeutics, Inc. ( Millendo Therapeutics US, Inc. ):
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders