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A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Tildacerfont/Placebo
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 CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 supraphsiologic 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

Sites / Locations

  • Spruce Study Site
  • Spruce Study Site
  • Spruce Clinical Site
  • Spruce Study Site
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  • Spruce Clinical Site
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  • Spruce Clinical Site
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  • Spruce Clinical Site
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  • Spruce Clinical Site
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  • Spruce Clinical Site
  • Spruce Clinical Site
  • Spruce Study Site
  • Spruce study site
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Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

Tildacerfont Group 1

Tildacerfont Group 2

Tildacerfont Group 3

Placebo

Arm Description

Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1

Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2

Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3

Placebo administered daily via oral tablet for 12 weeks.

Outcomes

Primary Outcome Measures

Change in androstenedione
Percent change of androstenedione

Secondary Outcome Measures

Proportion of subjects who achieve reduction A4 levels
Proportion of subjects who achieve A4 ≤ ULN
Proportion of subjects who achieve reduction in 17-OHP
Proportion of subjects who achieve 17-OHP≤ 1200ng/dL
Effectiveness in reducing TART(s) in Male CAH subjects
Change in lesion volume of TART(s) from baseline

Full Information

First Posted
June 25, 2020
Last Updated
October 4, 2023
Sponsor
Spruce Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT04457336
Brief Title
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 26, 2020 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
November 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 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.
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.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Subjects will be randomized in a 1:1:1:1 manner to one of three doses of Tildacerfont or Placebo.
Masking
ParticipantCare ProviderInvestigator
Masking Description
Double-Blind
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Tildacerfont Group 1
Arm Type
Experimental
Arm Description
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1
Arm Title
Tildacerfont Group 2
Arm Type
Experimental
Arm Description
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2
Arm Title
Tildacerfont Group 3
Arm Type
Experimental
Arm Description
Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered daily via oral tablet for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
Tildacerfont/Placebo
Other Intervention Name(s)
SPR001
Intervention Description
Tablet, administered daily
Primary Outcome Measure Information:
Title
Change in androstenedione
Description
Percent change of androstenedione
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Proportion of subjects who achieve reduction A4 levels
Description
Proportion of subjects who achieve A4 ≤ ULN
Time Frame
12 weeks
Title
Proportion of subjects who achieve reduction in 17-OHP
Description
Proportion of subjects who achieve 17-OHP≤ 1200ng/dL
Time Frame
12 weeks
Title
Effectiveness in reducing TART(s) in Male CAH subjects
Description
Change in lesion volume of TART(s) from baseline
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kyriakie Sarafoglou, M.D
Organizational Affiliation
Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
Spruce Study Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Spruce Study Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Spruce Clinical Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Spruce Study Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Spruce Study Site
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Facility Name
Spruce Clinical Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Spruce Study Site
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33401
Country
United States
Facility Name
Spruce Study Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Spruce Clinical Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Spruce Study Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Spruce Clinical Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Facility Name
Spruce Study Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Spruce Clinical Site
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89148
Country
United States
Facility Name
Spruce Study Site
City
Hickory
State/Province
North Carolina
ZIP/Postal Code
28601
Country
United States
Facility Name
Spruce Study Site
City
Canton
State/Province
Ohio
ZIP/Postal Code
44718
Country
United States
Facility Name
Spruce Study Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
Spruce Study Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Spruce Study Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Spruce Clinical Site
City
Bend
State/Province
Oregon
ZIP/Postal Code
97702
Country
United States
Facility Name
Spruce Study Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Spruce Study Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
Spruce Study Site
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02903
Country
United States
Facility Name
Spruce Study Site
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
28203
Country
United States
Facility Name
Spruce Clinical Site
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38163
Country
United States
Facility Name
Spruce Study Site
City
Austin
State/Province
Texas
ZIP/Postal Code
78731
Country
United States
Facility Name
Spruce Study Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75093
Country
United States
Facility Name
Spruce Clinical Site
City
Edinburg
State/Province
Texas
ZIP/Postal Code
78539
Country
United States
Facility Name
Spruce Clinical Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Spruce Study Site
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Spruce study site
City
Brisbane
Country
Australia
Facility Name
Spruce Study Site
City
Elizabeth Vale
Country
Australia
Facility Name
Spruce Study Site
City
Melbourne
Country
Australia
Facility Name
Spruce Study Site
City
Brasília
Country
Brazil
Facility Name
Spruce Study Site
City
São Paulo
Country
Brazil
Facility Name
Spruce Study Site
City
St. John's
State/Province
Newfoundland and Labrador
Country
Canada
Facility Name
Spruce Study Site
City
London
State/Province
Ontario
Country
Canada
Facility Name
Spruce Study Site
City
Ottawa
ZIP/Postal Code
K1H7W9
Country
Canada
Facility Name
Spruce Study Site
City
Sherbrooke
ZIP/Postal Code
J1H 5N4
Country
Canada
Facility Name
Spruce Study Site
City
Aarhus
Country
Denmark
Facility Name
Spruce Study Site
City
Copenhagen
Country
Denmark
Facility Name
Spruce Study Site
City
Tallinn
Country
Estonia
Facility Name
Spruce Study Site
City
Tartu
Country
Estonia
Facility Name
Spruce Study Site
City
Munich
Country
Germany
Facility Name
Spruce Study Site
City
Dublin
Country
Ireland
Facility Name
Spruce Study Site
City
Milan
Country
Italy
Facility Name
Spruce Study Site
City
Napoli
Country
Italy
Facility Name
Spruce Study Site
City
Rome
Country
Italy
Facility Name
Spruce Study Site
City
Torino
Country
Italy
Facility Name
Spruce Study Site
City
Seoul
Country
Korea, Republic of
Facility Name
Spruce Study Site
City
Riga
Country
Latvia
Facility Name
Spruce Study Site
City
Kaunas
Country
Lithuania
Facility Name
Spruce Study Site
City
Nijmegen
Country
Netherlands
Facility Name
Spruce Study Site
City
Kraków
Country
Poland
Facility Name
Spruce Study Site
City
Warsaw
Country
Poland
Facility Name
Spruce Study Site
City
Bucharest
Country
Romania
Facility Name
Spruce Study Site
City
Barcelona
Country
Spain
Facility Name
Spruce Study Site
City
Madrid
Country
Spain
Facility Name
Spruce Study Site
City
Sevilla
Country
Spain
Facility Name
Spruce Study Site
City
Tarragona
Country
Spain
Facility Name
Spruce Study Site
City
Falun
Country
Sweden
Facility Name
Spruce Study Site
City
Stockholm
Country
Sweden
Facility Name
Spruce Study Site
City
Saint Gallen
Country
Switzerland
Facility Name
Spruce Study Site
City
Zürich
Country
Switzerland
Facility Name
Spruce Study Site
City
Istanbul
Country
Turkey
Facility Name
Spruce Study Site
City
Birmingham
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

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