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    Summary
    EudraCT Number:2019-004765-40
    Sponsor's Protocol Code Number:SPR001-204
    National Competent Authority:Romania - National Agency for Medicines and Medical Devices
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2024-10-10
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedRomania - National Agency for Medicines and Medical Devices
    A.2EudraCT number2019-004765-40
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a randomized, double-blinded, Placebo controlled trial that will evaluate the potential of tildacerfont to reduce GC use in adult subjects with classic CAH who are on supraphysiologic doses of GC therapy.
    A.3.2Name or abbreviated title of the trial where available
    Efficacy and Safety of tildacerfont in Reducing Supraphysiologic GC doses in adult subjects with CAH
    A.4.1Sponsor's protocol code numberSPR001-204
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSpruce Biosciences, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSpruce Biosciences, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSpruce Biosciences, Inc.
    B.5.2Functional name of contact pointVaeling Miller
    B.5.3 Address:
    B.5.3.1Street Address2001 Junipero Serra Blvd, Suite 640
    B.5.3.2Town/ cityDaly City
    B.5.3.3Post codeCA 94014
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 4156554169
    B.5.6E-mailvmiller@sprucebiosciences.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/164/17
    D.3 Description of the IMP
    D.3.1Product nameTildacerfont
    D.3.2Product code SPR001
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTILDACERFONT
    D.3.9.1CAS number 1014983-00-6
    D.3.9.2Current sponsor codeSPN001
    D.3.9.4EV Substance CodeSUB192788
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Classic Congenital Adrenal Hyperplasia
    E.1.1.1Medical condition in easily understood language
    CAH is a serious, chronically debilitating, and life-threatening genetic disorder characterized by impaired adrenal synthesis of cortisol and overproduction of adrenal androgens
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10010323
    E.1.2Term Congenital adrenal hyperplasia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the proportion of subjects who can reduce GC use in subjects with CAH
    E.2.2Secondary objectives of the trial
    -To evaluate the percentage change in GC use in subjects with CAH
    -To evaluate the effect of tildacerfont in reducing the median cumulative HCe dose
    -To evaluate the effect of tildacerfont in reducing cardiovascular risk in subjects with CAH
    -To evaluate the effect of tildacerfont in improving homeostatic model assessment of insulin resistance (HOMA-IR) in subjects with CAH
    -To evaluate the effect of tildacerfont on body weight after 24 weeks in subjects with CAH
    -To evaluate the effect of tildacerfont on body weight after 52 weeks of tildacerfont treatment in subjects with CAH
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male and female subjects ≥ 18 years old at screening
    2.Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combinaton of the aforementionned GCs)
    3.Has LLD ≤ A4 ≤ 2.5x ULN at screening measured before an am GC dose
    4. Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥30 mg/day and ≤60 mg/day in HCe) for ≥1 month before screening
    5.For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening
    6. Follow contraception guidelines . Male subjects must also agree to refrain from donating sperm throughout the Treatment Period and for 90 days after the last dose of study drug.
    7.Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol.
    E.4Principal exclusion criteria
    1.Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21 hydroxylase deficiency)
    2.Has a history that includes bilateral adrenalectomy or hypopituitarism
    3.Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
    4.Shows clinical signs or symptoms of adrenal insufficiency
    5.Has had a clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to:
    a.An ongoing malignancy or <3 years of remission history from any malignancy, other than successfully treated localized skin cancer
    b.Estimated glomerular filtration rate (eGFR) of <45 mL/min/1.73 m2
    c. Current or history of liver disease
    d. History of alcohol or substance abuse within the last year
    e. Active hepatitis B, C or HIV at screening
    f. subjects who plan to undergo bariatric surgery during the study are excluded
    g. any other condition that would impact subject safety or confound interpretation
    6.Psychiatric conditions, including but not limited to bipolar disorder, schizophrenia, or schizoaffective disorders that are not effectively controlled on medication and may have an adverse impact on study compliance. Symptoms including hallucinations, delusions, and psychosis are exclusionary. Additionally:
    a. Increased risk of suicide based on the Investigator’s judgment or the results of the Columbia–Suicide Severity Rating Scale (C SSRS) conducted at screening and baseline (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months)
    b. HADS score >12 for either depression or anxiety at screening or baseline
    7.Has clinically significant abnormal ECG or clinical laboratory results.
    8. Routinely works overnight shifts
    9.Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (>2 hours) will require Medical Monitor approval for enrollment.
    10.Females who are pregnant or nursing
    11.Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study
    12.Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before the start of the Treatment Period to the end of the study
    a. Rosiglitazone, aromatase inhibitors, testosterone, or growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results
    b. The drugs listed in Section 13.1
    13.Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects with at least a 5mg/day HCe reduction from baseline in GC dose and A4 ≤ ULN at Week 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    E.5.2Secondary end point(s)
    -Percent change from baseline in GC dose at Week 24
    -Median total cumulative GC dose in HCe at Week 24
    -Proportion of subjects with improvement in at least one cardiovascular risk factor at Week 24
    -Change from baseline in the HOMA-IR at Week 24
    -Percent change from baseline in body weight at Week 24
    -Percent change from baseline in body weight after 52 weeks of tildacerfont treatment
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24 and Week 52
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Biomarkers
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Optional open label extension study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA21
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Switzerland
    Australia
    Brazil
    Canada
    Korea, Republic of
    United Kingdom
    United States
    Denmark
    Estonia
    Germany
    Ireland
    Italy
    Latvia
    Lithuania
    Netherlands
    Poland
    Romania
    Spain
    Sweden
    Türkiye
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    A subject is considered to have completed study treatment if the subject has completed all visits through Week 76 of the Treatment Period. A subject is considered to have completed the study if the subject has completed all phases of the study, including the final follow-up visit. The end of the study is defined as the date of the last follow-up visit of the last subject in the study
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 85
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 5
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 32
    F.4.2.2In the whole clinical trial 90
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After the trial, the patient care will revert to their physicians
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-11-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-22
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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