Nevanimibe HCl for the Treatment of Classic CAH

NCT03669549 | Terminated Phase 2 Results

• 1 other identifier: ATR-101-202
• Study Type: interventional
• Target: 15
• Locations: 5 countries
• Sites: 11

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.

Conditions

Congenital Adrenal Hyperplasia

Keywords

CAH; nevanimibe

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Nevanimibe hydrochloride

EXPERIMENTAL

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

Drug: Nevanimibe hydrochloride

Interventions

Nevanimibe hydrochloride DRUG

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.

Nevanimibe hydrochloride

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Millendo Therapeutics US, Inc.: lead

Study Sites (11)

University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto

Ribeirão Preto, 14051-140, Brazil

Location

Universidade Federal de São Paulo, Escola Paulista de Medicina

São Paulo, 04037-002, Brazil

Location

Hospital das Clínicas da FMUSP - Prédio do Instituto Central

São Paulo, 05403-900, Brazil

Location

Institute of Endocrinology

Prague, Czechia

Location

Hospital Pitié-Salpetrière

Paris, France

Location

Bnai Zion Medical Center

Haifa, Israel

Location

Beilinson Hospital

Petah Tikva, Israel

Location

Tel-Aviv-Sourasky Medical Center

Tel Aviv, Israel

Location

Hospital General Universitario Gregorio Marañón

Madrid, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Spain

Location

University Hospital La Fe

Valencia, Spain

Location

Related Publications (1)

• 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.

  PMID: 35283460 DERIVED

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Limitations and Caveats

Nevanimibe hydrochloride administered orally at doses of 500-2000 mg BID for up to 16 weeks in the first 10 evaluable patients did not result in sufficient efficacy. The Sponsor has decided to refrain from further development at this time.

Results Point of Contact

• Title: Clinical Trial Information
• Organization: Millendo Therapeutics

millendo@millendo.com

+1 734-845-9000

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2018
• First Posted: September 13, 2018
• Study Start: July 11, 2018
• Primary Completion: June 3, 2020
• Study Completion: July 12, 2020
• Last Updated: March 4, 2021
• Results First Posted: March 4, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); CZ (1); ES (3); BR (3); IL (3)