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Trial record 19 of 108 for:    "21-hydroxylase deficiency"

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

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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

Tracking Information
First Submitted Date  ICMJE August 15, 2017
First Posted Date  ICMJE August 22, 2017
Last Update Posted Date June 26, 2019
Actual Study Start Date  ICMJE July 26, 2017
Actual Primary Completion Date April 15, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 21, 2017)
  • 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
  • Change in 17-hydroxyprogesterone [ Time Frame: 6 weeks ]
    Change in 17-hydroxyprogesterone from Baseline to End-of-study
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03257462 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 21, 2017)
  • Changes in pharmacodynamic (PD) markers [ Time Frame: 6 weeks ]
    Changes in ACTH and androgens from Baseline to End-of-study
  • 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
  • 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
  • PK/PD relationships [ Time Frame: 6 weeks ]
    To explore the potential relationships between pharmacokinetics and pharmacodynamics
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia
Official Title  ICMJE A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH)
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).
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Congenital Adrenal Hyperplasia
  • CAH - Congenital Adrenal Hyperplasia
Intervention  ICMJE Drug: SPR001
SPR001 Capsules
Study Arms  ICMJE
  • 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.
    Intervention: Drug: SPR001
  • 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.
    Intervention: Drug: SPR001
  • 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.
    Intervention: Drug: SPR001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 24, 2019)
26
Original Estimated Enrollment  ICMJE
 (submitted: August 21, 2017)
18
Actual Study Completion Date  ICMJE May 28, 2019
Actual Primary Completion Date April 15, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03257462
Other Study ID Numbers  ICMJE SPR001-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Spruce Biosciences
Study Sponsor  ICMJE Spruce Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Michael Huang, MD Spruce Biosciences
Principal Investigator: Richard Auchus, MD, PhD University of Michigan
PRS Account Spruce Biosciences
Verification Date June 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP