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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02804178
First Posted: June 17, 2016
Last Update Posted: June 22, 2017
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. Read our disclaimer for details.
Information provided by (Responsible Party):
Millendo Therapeutics, Inc.
  Purpose
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.

Condition Intervention Phase
Congenital Adrenal Hyperplasia Drug: ATR-101 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

Resource links provided by NLM:


Further study details as provided by Millendo Therapeutics, Inc.:

Primary Outcome Measures:
  • Reduction of 17-OHP to </= 2X ULN [ Time Frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels. ]

Secondary Outcome Measures:
  • Number of participants with abnormal laboratory values and/or adverse events that are related to treatment [ Time Frame: Baseline, Day 1 and Day 15 of each dose level. Each subject will have up to 5 dose levels. ]
    Safety evaluations will include adverse events, vital signs, physical examinations, laboratory measures and ECGs.

  • Pharmacokinetics: Area under the curve [ Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4 hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
  • Pharmacokinetics: Maximum plasma concentration [ Time Frame: Evaluated at Day 1 (predose, 1, 2, 4, hours post-dose and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
  • Pharmacokinetics: Time to maximum concentration [ Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4, hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]

Enrollment: 15
Study Start Date: May 2016
Estimated Study Completion Date: September 2017
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth BID up to 1000 mg BID.
Drug: ATR-101

  Eligibility

Information from the National Library of Medicine

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

Inclusion Criteria:

  • Documented historical diagnosis of classic CAH due to 21-OHD based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-OHP
  • Biochemical marker of disease status of 17-OHP ≥ 4 X ULN
  • Chronic glucocorticoid replacement therapy for at least 6 consecutive months
  • Stable glucocorticoid and mineralocorticoid regimen for at least 1 month

Exclusion Criteria:

  • 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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT02804178


Locations
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
United States, Oklahoma
The University of Oklahoma - Tulsa Schusterman Center
Tulsa, Oklahoma, United States, 74135
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 10021
Sponsors and Collaborators
Millendo Therapeutics, Inc.
  More Information

Responsible Party: Millendo Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02804178     History of Changes
Other Study ID Numbers: ATR-101-201
First Submitted: June 1, 2016
First Posted: June 17, 2016
Last Update Posted: June 22, 2017
Last Verified: June 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Millendo Therapeutics, Inc.:
ATR-101
classic congenital adrenal hyperplasia
21-hydroxylase deficiency
CAH

Additional relevant MeSH terms:
Hyperplasia
Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
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
Epinephrine
Racepinephrine
Epinephryl borate
Adrenergic alpha-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Adrenergic beta-Agonists
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Anti-Asthmatic Agents
Respiratory System Agents