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Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

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: NCT03257462
Recruitment Status : Completed
First Posted : August 22, 2017
Last Update Posted : June 26, 2019
Sponsor:
Information provided by (Responsible Party):
Spruce Biosciences

Study Description
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Brief Summary:
This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia CAH - Congenital Adrenal Hyperplasia Drug: SPR001 Phase 2

Detailed Description:

This is a 6-week, multiple-dose, dose escalation study of SPR001 for the treatment of adults with classic CAH. After screening, eligible patients will be enrolled into a 6-week treatment period followed by a 4-week washout/safety follow-up period.

It is initially planned that up to approximately 18 patients in 2 dose cohorts will be enrolled. Additional patients or dose groups may be considered based upon specific safety, PK/PD, and/or efficacy findings, or if an active dose has not yet been reached.

SPR001 will be administered as an oral daily dose. Patients will undergo titration of SPR001 through three escalating dosage strengths at 2-week intervals. Patients will have overnight PK/PD assessments performed at baseline, which include an pre-dose overnight assessment and a post-dose overnight assessment for PK/PD following administration of the first dose. At the end of each 2-week dosing period, patients will return for single overnight visits for steady-state PK/PD assessments.

A follow-up outpatient visit will occur 30 days after their last dose.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH)
Actual Study Start Date : July 26, 2017
Actual Primary Completion Date : April 15, 2019
Actual Study Completion Date : May 28, 2019

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort A
The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks.
 Drug: SPR001
SPR001 Capsules
Experimental: Cohort B
Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A.
 Drug: SPR001
SPR001 Capsules
Experimental: Cohort C
Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B.
 Drug: SPR001
SPR001 Capsules


Outcome Measures
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Primary Outcome Measures :
  1. Safety of SPR001 in patients with CAH [ Time Frame: 6 weeks ]
    Incidence of treatment-emergent adverse events; changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters

  2. Change in 17-hydroxyprogesterone [ Time Frame: 6 weeks ]
    Change in 17-hydroxyprogesterone from Baseline to End-of-study


Secondary Outcome Measures :
  1. Changes in pharmacodynamic (PD) markers [ Time Frame: 6 weeks ]
    Changes in ACTH and androgens from Baseline to End-of-study

  2. Maximum plasma concentration (Cmax) [ Time Frame: 6 weeks ]
    To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH

  3. Area under the concentration-time curve (AUC) [ Time Frame: 6 weeks ]
    To evaluate the PK parameter of area under the concentration-time curve (AUC) of SPR001 in patients with CAH

  4. PK/PD relationships [ Time Frame: 6 weeks ]
    To explore the potential relationships between pharmacokinetics and pharmacodynamics


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 patients age 18 or older.
  • Documented diagnosis of classic CAH due to 21-hydroxylase deficiency
  • Elevated 17-OHP at screening
  • On a stable glucocorticoid replacement regimen for a minimum of 30 days

Exclusion Criteria:

  • Clinically significant unstable medical condition, illness, or chronic disease
  • Clinically significant psychiatric disorder.
  • Clinically significant abnormal laboratory finding or assessment
  • History of bilateral adrenalectomy or hypopituitarism
  • Pregnant or nursing females
  • Use of any other investigational drug within 30 days
  • Unable to understand and comply with the study procedures, understand the risks, and/or unwilling to provide written informed consent.
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): NCT03257462


Locations
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United States, California
Spruce Biosciences Clinical Site
Orange, California, United States, 92123
Spruce Biosciences Clinical Site
San Diego, California, United States, 92123
United States, Florida
Spruce Biosciences Clinical Site
Melbourne, Florida, United States, 32935
United States, Georgia
Spruce Biosciences Clinical Site
Atlanta, Georgia, United States, 30046
United States, Indiana
Spruce Biosciences Clinical Site
Indianapolis, Indiana, United States, 46202
United States, Michigan
Spruce Biosciences Clinical Site
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Spruce Biosciences Clinical Site
Minneapolis, Minnesota, United States, 55414
United States, Nevada
Spruce Biosciences Clinical Site
Las Vegas, Nevada, United States, 89148
United States, Pennsylvania
Spruce Biosciences Clinical Site
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Spruce Biosciences
Investigators
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Study Director: Michael Huang, MD Spruce Biosciences
Principal Investigator: Richard Auchus, MD, PhD University of Michigan
More Information
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Additional Information:

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Responsible Party: Spruce Biosciences
ClinicalTrials.gov Identifier: NCT03257462    
Other Study ID Numbers: SPR001-201
First Posted: August 22, 2017    Key Record Dates
Last Update Posted: June 26, 2019
Last Verified: June 2018
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:
17-hydroxyprogesterone
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