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A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont 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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04457336
Recruitment Status : Active, not recruiting
First Posted : July 7, 2020
Last Update Posted : January 8, 2024
Sponsor:
Information provided by (Responsible Party):
Spruce Biosciences

Study Description
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Brief Summary:
An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.

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

Detailed Description:
This is a study that will test the efficacy and safety of Tildacerfont. The first 12-weeks will be a double-blind, placebo controlled, dose ranging study. The following 58-weeks will assess the long term safety of Tildacerfont. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 96 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Subjects will be randomized in a 1:1:1:1 manner to one of three doses of Tildacerfont or Placebo.
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Double-Blind
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects With Classic Congenital Adrenal Hyperplasia
Actual Study Start Date : August 26, 2020
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : November 2029

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Tildacerfont Group 1
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1
 Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001
Experimental: Tildacerfont Group 2
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2
 Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001
Experimental: Tildacerfont Group 3
Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3
 Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001
Placebo Comparator: Placebo
Placebo administered daily via oral tablet for 12 weeks.
 Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001


Outcome Measures
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Primary Outcome Measures :
  1. Change in androstenedione [ Time Frame: 12 weeks ]
    Percent change of androstenedione


Secondary Outcome Measures :
  1. Proportion of subjects who achieve reduction A4 levels [ Time Frame: 12 weeks ]
    Proportion of subjects who achieve A4 ≤ ULN

  2. Proportion of subjects who achieve reduction in 17-OHP [ Time Frame: 12 weeks ]
    Proportion of subjects who achieve 17-OHP≤ 1200ng/dL

  3. Effectiveness in reducing TART(s) in Male CAH subjects [ Time Frame: 12 weeks ]
    Change in lesion volume of TART(s) from baseline


Eligibility Criteria
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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 known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
  • Has been on a stable supraphysiologic dose of GC replacement ≥15 mg/day and ≤60 mg/day in HC equivalents
  • 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
  • Current treatment with dexamethasone as GC therapy for CAH. Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (eg, HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥1 month before screening.
  • Shows clinical signs or symptoms of adrenal insufficiency
Contacts and Locations
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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): NCT04457336


Locations
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Sponsors and Collaborators
Spruce Biosciences
Investigators
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Principal Investigator: Kyriakie Sarafoglou, M.D Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
More Information
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Responsible Party: Spruce Biosciences
ClinicalTrials.gov Identifier: NCT04457336    
Other Study ID Numbers: SPR001-203
CAHmelia 203 ( Other Identifier: Spruce Biosciences )
First Posted: July 7, 2020    Key Record Dates
Last Update Posted: January 8, 2024
Last Verified: January 2024
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
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
 Male Urogenital Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders