A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

NCT02804178 | Completed Phase 2 Results

• 1 other identifier: ATR-101-201
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 6

Brief Summary

This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.

Conditions

Congenital Adrenal Hyperplasia

Keywords

ATR-101; classic congenital adrenal hyperplasia; 21-hydroxylase deficiency; CAH

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101

  17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.

  Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.

Study Arms (1)

ATR-101

EXPERIMENTAL

Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.

Drug: ATR-101

Interventions

ATR-101 DRUG

125-1000 mg twice per week

Also known as: Nevanimibe hydrochloride

ATR-101

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 based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone
• Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal
• Chronic glucocorticoid replacement therapy for at least 6 consecutive months
• Stable glucocorticoid and mineralocorticoid regimen for at least 1 month

You may not qualify if:

• Non-classic CAH
• Other causes of adrenal insufficiency
• Surgery within the previous 3 months prior to screening or planned surgery during study participation
• History of active cancer requiring medical or surgical therapy within the past 6 months

Sponsors & Collaborators

• Millendo Therapeutics, Inc.: lead

Study Sites (6)

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

The University of Oklahoma - Tulsa Schusterman Center

Tulsa, Oklahoma, 74135, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 10021, United States

Location

Related Publications (1)

• El-Maouche D, Merke DP, Vogiatzi MG, Chang AY, Turcu AF, Joyal EG, Lin VH, Weintraub L, Plaunt MR, Mohideen P, Auchus RJ. A Phase 2, Multicenter Study of Nevanimibe for the Treatment of Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2020 Aug 1;105(8):2771-8. doi: 10.1210/clinem/dgaa381.

  PMID: 32589738 DERIVED

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital; Congenital adrenal hyperplasia due to 21 hydroxylase deficiency

Interventions

N-(2,6-bis(1-methylethyl)phenyl)-N'-((1-(4-(dimethylamino)phenyl)cyclopentyl)methyl)urea hydrochloride

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

The small number of subjects analyzed as a result of the rarity of the disease (congenital adrenal hyperplasia).and the stage of the study (phase 2a)

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

Model Details: Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).

Study Record Dates

• First Submitted: June 1, 2016
• First Posted: June 17, 2016
• Study Start: May 18, 2016
• Primary Completion: August 17, 2017
• Study Completion: August 17, 2017
• Last Updated: March 10, 2021
• Results First Posted: February 17, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)