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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2021-004467-26
    Sponsor's Protocol Code Number:DIUR015
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-12-09
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-004467-26
    A.3Full title of the trial
    A Phase 3 Open-label Extension Study to Evaluate the Long-term Safety and
    Tolerability of Chronocort in the Treatment of Participants Aged 16 Years and Over with Congenital Adrenal Hyperplasia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label Extension Study to Evaluate the Long-term Safety and
    Tolerability of Chronocort in the Treatment of Participants Aged 16 Years and Over with Congenital Adrenal Hyperplasia
    A.4.1Sponsor's protocol code numberDIUR015
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05063994
    A.5.4Other Identifiers
    Name:IND NumberNumber:76485
    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 SponsorNeurocrine UK Limited
    B.1.3.4CountryUnited Kingdom
    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 supportNeurocrine UK Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNeurocrine UK Limited
    B.5.2Functional name of contact pointCardiff office contact information
    B.5.3 Address:
    B.5.3.1Street AddressCardiff Medicentre, Heath Park
    B.5.3.2Town/ cityCARDIFF
    B.5.3.3Post codeCF14 4UJ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number442920682069
    B.5.6E-mailinfoEU@neurocrine.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 Yes
    D.2.1.1.1Trade name Efmody 5 mg modified release hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderNeurocrine Netherlands B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameChronocort 5mg
    D.3.4Pharmaceutical form Modified-release capsule, hard
    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 INNHYDROCORTISONE
    D.3.9.3Other descriptive nameHYDROCORTISONE MICRONIZED
    D.3.9.4EV Substance CodeSUB194422
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.IMP: 2
    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 Yes
    D.2.1.1.1Trade name Efmody 10 mg modified release hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderNeurocrine Netherlands B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameChronocort 10mg
    D.3.4Pharmaceutical form Modified-release capsule, hard
    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 INNhydrocortisone
    D.3.9.3Other descriptive nameHYDROCORTISONE MICRONIZED
    D.3.9.4EV Substance CodeSUB194422
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Congenital Adrenal Hyperplasia due to 21-hydroxylase deficiency
    E.1.1.1Medical condition in easily understood language
    Increase activity of the adrenal glands
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    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 long-term safety and tolerability of Chronocort in the treatment of participants with CAH.
    E.2.2Secondary objectives of the trial
    1. To assess the impact of treatment on dose of steroid required.
    2. To assess the impact of treatment on 17-OHP levels.
    3. To assess the impact of treatment on A4 levels.
    4. To assess the impact of treatment on markers of fertility.
    5. To assess the impact of treatment on testosterone by sex.
    6. To assess the impact of treatment on waist circumference.
    7. To assess the impact of treatment on body weight.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participants with CAH who have successfully completed Chronocort study DIUR-006 (sites in France and US only) or study DIUR-014.
    2. Participants who are capable of giving signed informed consent/assent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
    E.4Principal exclusion criteria
    Medical Conditions
    1. Participants with clinical or biochemical evidence of hepatic or renal disease e.g., creatinine >2 times the upper limit of normal (ULN) or elevated liver function tests (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >2 times the ULN).
    2. Participants with a history of malignancy (other than basal cell carcinoma successfully treated >26 weeks prior to entry into the study).
    3. Participants with a history of bilateral adrenalectomy.
    4. Participants with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the study.


    Prior/Concomitant Therapy
    5. Participants with a co-morbid condition requiring daily administration of a medication or consumption of any material that interferes with the metabolism of glucocorticoids (examples at http://medicine.iupui.edu/clinpharm/ddis/clinical-table/).
    6. Participants on regular daily inhaled, topical, nasal, or oral steroids for any indication other than CAH.
    7. Participants anticipating regular prophylactic use of additional steroids e.g., for strenuous exercise.
    Prior/Concurrent Clinical Study Experience
    8. Participation in another clinical study of an investigational or licensed drug or device within 3 months prior to inclusion in this study, except for another clinical study with the current formulation of Chronocort.
    Other Exclusions
    9. Females who are pregnant or lactating.
    10. Participants, who in the opinion of the Investigator, will be unable to comply with the requirements of the protocol.
    11. Participants who routinely work night shifts and so do not sleep during the usual night-time hours.
    12. Participants with a body weight of 50 kg or less. (Note: this exclusion criterion is only applicable for French sites).
    E.5 End points
    E.5.1Primary end point(s)
    The safety of Chronocort over time, assessed
    using, but not limited to, the following outcome
    variables:
    • Signs and symptoms of adrenal insufficiency or over-treatment throughout the study.
    • Use of IRHC from the emergency packs for stress dosing or use of any additional glucocorticoid treatment during the study.
    • Occurrence of adrenal crises throughout the study.
    • Occurrence of AEs throughout the study.
    • Change from pre-Chronocort baseline in safety laboratory assessments at each visit throughout the study.
    • Change from pre-Chronocort baseline in vital signs at each visit throughout the study.
    E.5.1.1Timepoint(s) of evaluation of this end point
    December 2024 (end of trial)
    E.5.2Secondary end point(s)
    1. Change from pre-Chronocort baseline in total daily steroid dose in hydrocortisone equivalent dose at each visit.
    2. Change in 17-OHP levels from pre-Chronocort baseline at each visit.
    3. Change in A4 levels from pre-Chronocort baseline at each visit.
    4. Change from pre-Chronocort baseline to each visit in menstrual regularity (only in pre-menopausal females without hysterectomy and not using hormonal contraceptives)
    5. Change from pre-Chronocort baseline to each visit in luteinising hormone (LH) levels (only in men).
    6. Change in testosterone from pre-Chronocort baseline at each study visit.
    7. Change from pre-Chronocort baseline to each visit in waist circumference.
    8. Change from pre-Chronocort baseline to each visit in body weight.
    E.5.2.1Timepoint(s) of evaluation of this end point
    December 2024 (end of trial)
    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 No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Japan
    United States
    France
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 4
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 77
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2021-12-09. Yes
    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 state42
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 42
    F.4.2.2In the whole clinical trial 81
    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)
    Participants will continue in the study until a decision has been made to
    either switch the participant onto Chronocort commercial supply,
    extend the study, or return the participant to standard of care therapy.
    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 Decision2022-02-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-08
    P. End of Trial
    P.End of Trial StatusCompleted
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