We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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 : March 7, 2023
Sponsor:
Information provided by (Responsible Party):
Spruce Biosciences

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic CAH subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.

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. Subjects will be offered a long term open label extension up to 240 weeks.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
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 for 24 weeks followed by 52 weeks open label Tildacerfont
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Double-Blind for first 24 weeks, then open label
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 : December 2024
Estimated Study Completion Date : September 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Steroids

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: Tildacerfont Group
Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks
 Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001
Placebo Comparator: Placebo
Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 weeks
 Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Proportion of subjects who can reduce GC dose at Week 24 [ Time Frame: 24 Weeks ]
    Proportion of subjects with at least a 5 mg/day HCe reduction from baseline in GC dose and A4 ≤ULN at Week 24


Secondary Outcome Measures :
  1. Percentage change in GC use in subjects with CAH [ Time Frame: 24 weeks ]
    Percent change from baseline in GC dose at week 24

  2. Change in the median cumulative HCe dose in subjects with CAH [ Time Frame: 24 Weeks ]
    Median total cumulative GC dose in HCe at Week 24

  3. Effectiveness in reducing cardiovascular risk in subjects with CAH [ Time Frame: 24 Weeks ]
    Proportion of subjects with improvement in at least one cardiovascular risk factor at week 24

  4. Effectiveness in improving HOMA-IR in subjects with CAH [ Time Frame: 24 Weeks ]
    Change from baseline in the HOMA-IR at Week 24

  5. Effect on body weight in subjects with CAH [ Time Frame: 24 weeks ]
    Percent change from baseline in body weight after 24 weeks of tildacerfont treatment

  6. Effect on body weight in subjects with CAH [ Time Frame: 52 weeks ]
    Percent change from baseline in body weight after 52 weeks of tildacerfont treatment


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.


Layout table for eligibility information
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 documented elevated 17-OHP and currently treatment with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
  • Has been on a stable, supraphysiologic dose of GC replacement for ≥1 month before screening.
  • 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
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Layout table for location contacts
Contact: Clinical Trials 415-655-4169 CAHmelia@sprucebiosciences.com

Locations
Show Show 64 study locations
Sponsors and Collaborators
Spruce Biosciences
Investigators
Layout table for investigator information
Principal Investigator: Ron Newfield, M.D Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
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: March 7, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
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