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A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult 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.
 
ClinicalTrials.gov Identifier: NCT04544410
Recruitment Status : Recruiting
First Posted : September 10, 2020
Last Update Posted : October 29, 2021
Sponsor:
Information provided by (Responsible Party):
Spruce Biosciences

Brief Summary:
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic CAH subjects up to 76 weeks of treatment.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Drug: Tildacerfont/Placebo Phase 2

Detailed Description:
This is a study that will evaluate the ability of Tildacerfont to reduce the glucocorticoid steroid dose used by adult CAH subjects. The first 24-weeks will be a double-blind, placebo controlled, comparison of Tildacerfont vs Placebo. The following 52-weeks will allow all subjects to move to open label Tildacerfont to continue to reduce steroid dose where appropriate, and observe long term safety.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Subjects will be randomized in a 1:1 manner to either Tildacerfont or Placebo.
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Double-Blind
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects With Classic Congenital Adrenal Hyperplasia
Actual Study Start Date : September 29, 2020
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : November 2022


Arm Intervention/treatment
Experimental: Tildacerfont Group
Tildacerfont administered daily via oral tablet for 76 weeks at dose level 1.
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001

Placebo Comparator: Placebo
Placebo administered daily via oral tablet for 24 weeks.
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001




Primary Outcome Measures :
  1. Change in GC dose at Week 24 [ Time Frame: 24 Weeks ]
    Absolute change from baseline in GC dose in HCe at Week 24


Secondary Outcome Measures :
  1. Change in GC use adjusted for body surface area in subjects with CAH [ Time Frame: 24 weeks ]
    Absolute change from baseline in GC dose in HCe in mg/m2

  2. Change in the median cumulative HCe dose in subjects with CAH [ Time Frame: 76 Weeks ]
    Median total cumulative GC dose in HCe

  3. Change in GC use while maintaining control of A4 in subjects with CAH [ Time Frame: 24 Weeks ]

    Composite endpoint:

    Absolute change from baseline in GC dose in HCe in subjects who maintain A4 ≤ ULN


  4. Change in supraphysiologic GC use to physiologic GC use while maintaining control of A4 in subjects with CAH [ Time Frame: 24 Weeks ]

    Composite endpoint:

    Proportion of subjects with GC dose ≤20 mg/day in HCe in subjects who maintain A4 ≤ ULN


  5. Change in GC toxicity in subjects with CAH [ Time Frame: 24 weeks ]

    Glucocorticoid Toxicity Index Aggregate Improvement Score (GTI-AIS) (Miloslavsky 2017)

    Score range: -346 to 439 A positive score means worsening and a negative score means improvement


  6. Change in metabolic parameters (fat mass) in subjects with CAH [ Time Frame: 24 weeks ]
    Change from baseline in fat mass as measured by dual-energy X-ray absorptiometry (DXA)

  7. Change in metabolic parameters (body weight) in subjects with CAH [ Time Frame: 24 weeks ]
    Change from baseline in body weight

  8. Change in metabolic parameters (homeostatic model assessment of insulin resistance) in subjects with CAH [ Time Frame: 24 weeks ]
    Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female subjects over 18 years old, inclusive
  • Has a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or elevated 17-OHP
  • Has been on a stable, supraphysiologic dose of GC replacement for ≥1 month before screening without any evidence of non-adherence to the GC regimen during this period.
  • For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening

Exclusion Criteria:

  • Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency)
  • Has a history that includes bilateral adrenalectomy or hypopituitarism
  • Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
  • Shows clinical signs or symptoms of adrenal insufficiency

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): NCT04544410


Contacts
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Contact: Clinical Trials 415-655-4169 CAH_ClinicalTrials@sprucebiosciences.com

Locations
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United States, Alabama
Spruce Study Site Recruiting
Birmingham, Alabama, United States, 35294
United States, California
Spruce Clinical Site Recruiting
Orange, California, United States, 92868
Spruce Study Site Recruiting
Sacramento, California, United States, 95821
Spruce Study Site Recruiting
San Diego, California, United States, 92123
United States, Florida
Spruce Study Site Recruiting
Tampa, Florida, United States, 33612
United States, Indiana
Spruce Study Site Recruiting
Indianapolis, Indiana, United States, 46202
United States, Maryland
Spruce Study Site Recruiting
Baltimore, Maryland, United States, 21287
Spruce Study Site Recruiting
Camp Springs, Maryland, United States, 20746
United States, Massachusetts
Spruce Study Site Recruiting
Boston, Massachusetts, United States, 02111
United States, Michigan
Spruce Biosciences Clinical Site Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Spruce Study Site Recruiting
Minneapolis, Minnesota, United States, 55454
Spruce Study Site Recruiting
Rochester, Minnesota, United States, 55905
United States, Nevada
Spruce Study Site Recruiting
Las Vegas, Nevada, United States, 89148
United States, New York
Spruce Study Site Recruiting
Syracuse, New York, United States, 13057
United States, North Carolina
Spruce Study Site Recruiting
Hickory, North Carolina, United States, 28601
United States, Ohio
Spruce Study Site Recruiting
Canton, Ohio, United States, 44718
Spruce Study Site Recruiting
Cleveland, Ohio, United States, 44195
Spruce Study Site Recruiting
Columbus, Ohio, United States, 43210
United States, Oklahoma
Spruce Study Site Recruiting
Edmond, Oklahoma, United States, 73034
United States, Oregon
Spruce Study Site Recruiting
Bend, Oregon, United States, 99702
United States, Pennsylvania
Spruce Study Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Spruce Study Site Recruiting
Philadelphia, Pennsylvania, United States, 19107
Spruce Study Site Recruiting
Philadelphia, Pennsylvania, United States, 19140
United States, South Carolina
Spruce Study Site Recruiting
Columbia, South Carolina, United States, 29203
United States, Tennessee
Spruce Study Site Recruiting
Memphis, Tennessee, United States, 38163
United States, Texas
Spruce Study Site Recruiting
Dallas, Texas, United States, 75231
Spruce Study Site Recruiting
Edinburg, Texas, United States, 78539
Spruce Study Site Recruiting
Fort Worth, Texas, United States, 76104
United States, Virginia
Spruce Study Site Recruiting
Richmond, Virginia, United States, 23298
United States, Washington
Spruce Study Site Recruiting
Seattle, Washington, United States, 98105
Australia
Spruce Study Site Recruiting
Melbourne, Australia
Denmark
Spruce Study Site Recruiting
Copenhagen, Denmark
Netherlands
Spruce Study Site Recruiting
Nijmegen, Netherlands
Spain
Spruce Study Site Recruiting
Barcelona, Spain
Spruce Study Site Recruiting
Madrid, Spain
Spruce Study Site Recruiting
Sevilla, Spain
Sweden
Spruce Study Site Recruiting
Falun, Sweden
Sponsors and Collaborators
Spruce Biosciences
Investigators
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Principal Investigator: Ron Newfield, M.D Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
Additional Information:
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Responsible Party: Spruce Biosciences
ClinicalTrials.gov Identifier: NCT04544410    
Other Study ID Numbers: SPR001-204
CAHmelia 204 ( Other Identifier: Spruce Biosciences )
First Posted: September 10, 2020    Key Record Dates
Last Update Posted: October 29, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Spruce Biosciences:
CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Hyperplasia
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