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A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children With CAH

Primary Purpose

Congenital Adrenal Hyperplasia, 21-OHD

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Tildacerfont
Sponsored by
Spruce Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring Pediatric, CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male and female subjects aged 6 to 17 years
  • Diagnosis of CAH due to 21-OHD and/or elevated 17-OHP requiring ongoing GC replacement since diagnosis
  • Stable dose of GC replacement for at least 1 month prior to screening

Exclusion Criteria:

  • CAH not due to 21-OHD
  • History of bilateral adrenalectomy or hypopituitarism
  • Current treatment with dexamethasone
  • Clinically significant unstable medical conditions, illness, or chronic diseases
  • History of active bleeding disorders
  • Females who are pregnant or nursing

Sites / Locations

  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting
  • Spruce Study SiteRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Cohort 1: Age 11-17 Treatment with Tildacerfont

Cohort 2: Age 11-17 Treatment with Tildacerfont

Cohort 3: Age 2-10 Treatment with Tildacerfont

Arm Description

Oral Tildacerfont administered daily for 12 consecutive weeks.

Oral Tildacerfont administered daily for 12 consecutive weeks.

Oral Tildacerfont administered daily for 12 consecutive weeks.

Outcomes

Primary Outcome Measures

Number of subjects with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
To evaluate safety of tildacerfont in pediatric subjects with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0

Secondary Outcome Measures

Proportion of subjects who achieve reduction in androstenedione (A4) or reduction in glucocorticoid (GC) dosing
To determine the efficacy of tildacerfont on disease control or reduction of GC use in pediatric subjects with classic CAH as measured by number of subjects who achieve a reduction in A4 or reduction in GC dosing at week 12
Proportion of subjects with elevated baseline A4 who achieve a reduction in A4
To determine the efficacy of tildacerfont on disease control in pediatric subjects with classic CAH measured by the number of subjects with elevated baseline A4 who achieve reduction in A4 at week
Tildacerfont pharmacokinetics (PK)
To determine the consistency of preliminary tildacerfont PK in pediatric subjects with those simulated in a PBPK model measured by comparing tildacerfont plasma concentrations with current PBPK simulation for consistency

Full Information

First Posted
October 27, 2021
Last Updated
January 17, 2023
Sponsor
Spruce Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT05128942
Brief Title
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children With CAH
Official Title
A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years With Congenital Adrenal Hyperplasia
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 10, 2021 (Actual)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
March 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Spruce Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
An investigation of the safety and efficacy of tildacerfont in pediatric subjects with CAH.
Detailed Description
This is a study to evaluate the safety and efficacy of tildacerfont in pediatric subjects with Congenital Adrenal Hyperplasia (CAH). Treatment will consist of 12 weeks of continuous dosing followed by safety follow up.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia, 21-OHD
Keywords
Pediatric, CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Subjects will be assigned to treatment cohorts by age.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1: Age 11-17 Treatment with Tildacerfont
Arm Type
Experimental
Arm Description
Oral Tildacerfont administered daily for 12 consecutive weeks.
Arm Title
Cohort 2: Age 11-17 Treatment with Tildacerfont
Arm Type
Experimental
Arm Description
Oral Tildacerfont administered daily for 12 consecutive weeks.
Arm Title
Cohort 3: Age 2-10 Treatment with Tildacerfont
Arm Type
Experimental
Arm Description
Oral Tildacerfont administered daily for 12 consecutive weeks.
Intervention Type
Drug
Intervention Name(s)
Tildacerfont
Other Intervention Name(s)
SPR001
Intervention Description
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Primary Outcome Measure Information:
Title
Number of subjects with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Description
To evaluate safety of tildacerfont in pediatric subjects with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Proportion of subjects who achieve reduction in androstenedione (A4) or reduction in glucocorticoid (GC) dosing
Description
To determine the efficacy of tildacerfont on disease control or reduction of GC use in pediatric subjects with classic CAH as measured by number of subjects who achieve a reduction in A4 or reduction in GC dosing at week 12
Time Frame
12 weeks
Title
Proportion of subjects with elevated baseline A4 who achieve a reduction in A4
Description
To determine the efficacy of tildacerfont on disease control in pediatric subjects with classic CAH measured by the number of subjects with elevated baseline A4 who achieve reduction in A4 at week
Time Frame
4 weeks
Title
Tildacerfont pharmacokinetics (PK)
Description
To determine the consistency of preliminary tildacerfont PK in pediatric subjects with those simulated in a PBPK model measured by comparing tildacerfont plasma concentrations with current PBPK simulation for consistency
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male and female subjects aged 2 to 17 years Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis Stable dose of GC replacement for at least 1 month prior to screening Exclusion Criteria: CAH not due to 21-OHD History of bilateral adrenalectomy or hypopituitarism Clinically significant unstable medical conditions, illness, or chronic diseases History of active bleeding disorders Females who are pregnant or nursing
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Clinical Trials
Phone
415-655-4169
Email
CAHptain@sprucebiosciences.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Will Charlton, MD
Organizational Affiliation
Spruce Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
Spruce Study Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Buffalo
State/Province
New York
ZIP/Postal Code
14203
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23284
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children With CAH

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