Clinical trial • Phase III • Endocrinology|Rare Disease
CRINECERFONT for Classic congenital adrenal hyperplasia (21-hydroxylase deficiency)
Phase III trial of CRINECERFONT for Classic congenital adrenal hyperplasia (21-hydroxylase deficiency).
Overview
- Trial Therapeutic Area
- Endocrinology|Rare Disease
- Trial Disease
- Classic congenital adrenal hyperplasia (21-hydroxylase deficiency)
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 15-04-2024
- First CTIS Authorization Date
- 24-05-2024
Trial design
Randomised, open-label, placebo (crinecerfont placebo for oral solution and crinecerfont placebo for oral capsules). dose/schedule not specified in available data.-controlled Phase III trial across 14 sites in Belgium, France, Greece and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Placebo (Crinecerfont placebo for oral solution and Crinecerfont placebo for oral Capsules). Dose/schedule not specified in available data.
- Target Sample Size
- 58
Eligibility
Recruits 58 paediatric patients.
- Pregnancy Exclusion
- Females who are pregnant or lactating.
- Vulnerable Population
- Pediatric population selected (isVulnerablePopulationSelected = true). Informed consent and assent materials are provided by age group: parental/legal guardian informed consent forms and subject information/ICF documents for adults; assent and ICF/assent documents are available for multiple pediatric age groups (examples in documents: assent to participate 3-5 yr, 6-10 yr, 11-17 yr; Parental/Legal representative ICFs). Materials are provided in country/language-specific versions as indicated by the submitted documents (examples include French, Dutch (NL), German, Spanish, Italian, Greek, Polish).
Inclusion criteria
- {"criterion_text":"-Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit."}
- {"criterion_text":"-Be a female or male 2 to 17 years of age with a body weight of at least 10 kg."}
- {"criterion_text":"-Have a medically confirmed diagnosis of classic CAH due to 21- hydroxylase deficiency."}
- {"criterion_text":"-Be on a stable regimen of glucocorticoid treatment for CAH."}
- {"criterion_text":"-Have elevated adrenal androgens."}
- {"criterion_text":"-If treated with fludrocortisone, dose should be stable for at least 1 month prior to screening. Regardless of fludrocortisone treatment, upright plasma renin activity (PRA) (in the absence of medications that confound interpretation of PRA) during screening should be <3× ULN and >lower limit of normal (LLN) on the subject's usual sodium intake (if PRA >2 × ULN and <3 × ULN, subjects must have normal age-specific systolic blood pressure and heart rate and serum potassium "}
Exclusion criteria
- {"criterion_text":"-Have a diagnosis of any of the other forms of classic CAH."}
- {"criterion_text":"-Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic daily therapy with oral glucocorticoids."}
- {"criterion_text":"-Have a clinically significant unstable medical condition or chronic disease other than CAH"}
- {"criterion_text":"-Have a history of malignancy, unless successfully treated with curative intent and considered to be cured."}
- {"criterion_text":"-Have a known history of clinically significant arrhythmia or abnormalities on screening ECG."}
- {"criterion_text":"-Have a known hypersensitivity or allergy to any corticotropin-releasing hormone (CRH) receptor antagonist or any component of the study drug."}
- {"criterion_text":"-Females who are pregnant or lactating."}
Endpoints
Primary endpoints
- {"endpoint_text":"-Change from baseline in serum androstenedione at Week 4.","definition_or_measurement_approach":"Change from baseline in serum androstenedione measured at Week 4 (change from baseline comparison)."}
Secondary endpoints
- {"endpoint_text":"-Change from baseline in serum 17-hydroxyprogesterone at Week 4","definition_or_measurement_approach":"Change from baseline in serum 17-hydroxyprogesterone measured at Week 4 (change from baseline comparison)."}
- {"endpoint_text":"-Percent change from baseline in glucocorticoid dose at Week 28","definition_or_measurement_approach":"Percent change from baseline in daily glucocorticoid dose measured at Week 28."}
Recruitment
- Planned Sample Size
- 58
- Recruitment Window Months
- 67
- Consent Approach
- Parental/legal guardian informed consent is required for pediatric participants; age-appropriate assent forms are provided for younger subjects (assent documents listed for 3-5 yr, 6-10 yr, 11-17 yr in submitted materials). Subject information sheets and ICFs are provided in country/language-specific versions (documents submitted in French, Dutch, German, Spanish, Italian, Greek, Polish as indicated).
Geography
- Total Number Of Sites
- 14
- Total Number Of Participants
- 35
Belgium
- Latest Decision Or Authorization Date
- 10-10-2025
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- SAFE-PEDRUG
- Contact Person Name
- Martine Cools
- Contact Person Email
- martine.cools@urgent.be
France
- Latest Decision Or Authorization Date
- 23-10-2025
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Service d'Endocrinologie Gynécologie et Diabétologie Pédiatriques
- Contact Person Name
- Dinane SAMARA DIAB BOUSTANI
- Contact Person Email
- dinane.samara-boustani@aphp.fr
- Site Name
- Robert Debre University Hospital
- Department Name
- Endocrinologie et Diabétologie Pédiatrique
- Contact Person Name
- Laetitia MARTINERIE
- Contact Person Email
- laetitia.martinerie@aphp.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Endocrinologie et diabète de l'enfant
- Contact Person Name
- Claire Bouvattier
- Contact Person Email
- claire.bouvattier@aphp.fr
- Site Name
- Centre Hospitalier Regional D'Angers
- Department Name
- Endocrinologie et Diabétologie Pédiatrique
- Contact Person Name
- Régis COUTANT
- Contact Person Email
- ReCoutant@chu-angers.f
Greece
- Latest Decision Or Authorization Date
- 13-11-2025
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Nosokomeio Paidon I Agia Sofia
- Department Name
- Endocrinology, Metabolism and Diabetes Unit, First Pediatric Clinic
- Contact Person Name
- Evangelia Charmandari
- Contact Person Email
- evangelia.charmandari@googlemail.com
Italy
- Latest Decision Or Authorization Date
- 14-10-2025
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- Department of Pediatric Endocrinology
- Contact Person Name
- Salerno Mariacarolina
- Contact Person Email
- salerno@unina.it
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- Department of Pediatric Endocrinology
- Contact Person Name
- Gianni Russo
- Contact Person Email
- russo.gianni26@gmail.com
Spain
- Latest Decision Or Authorization Date
- 20-10-2025
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Servicio de Pediatría
- Contact Person Name
- Ana Lucia Gomez
- Contact Person Email
- analucia.gomez.sspa@juntadeandalucia.es
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Servicio de endocrinología pediátrica
- Contact Person Name
- Maria Clemente Leon
- Contact Person Email
- mclement@vhebron.net
Poland
- Latest Decision Or Authorization Date
- 15-10-2025
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Department of Pediatrics, Diabetology and Endocrinology
- Contact Person Name
- Dorota Biskholz-Walerzak
- Contact Person Email
- debirkhol@wp.pl
- Site Name
- Kliniczny Szpital Wojewodzki Nr 2 Im. Sw. Jadwigi Krolowej W Rzeszowie
- Department Name
- Endocrinology and Child Diabetology
- Contact Person Name
- Artur Mazur
- Contact Person Email
- drmazur@poczta.onet.pl
Germany
- Latest Decision Or Authorization Date
- 14-01-2026
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- Universitaetsklinikum Heidelberg AöR
- Department Name
- Division of Paediatric Endocrinology & Diabetes
- Contact Person Name
- Daniela Choukair
- Contact Person Email
- daniela.choukair@med.uni-heidelberg.de
- Site Name
- Otto Von Guericke Universitaet Magdeburg
- Department Name
- Endokrinologie und Diabetologie
- Contact Person Name
- Katja Palm
- Contact Person Email
- katja.palm@med.ovgu.de
Sponsor
Primary sponsor
- Full Name
- Neurocrine Biosciences Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Worldwide Clinical Trials Holdings Inc.
- Responsibilities
- Negotiation of site clinical trial agreements, site activation, Trial Master File, Clinical Assessment Technologies, Risk Based Quality Management (RBQM), Safety Management Maintenance, Set up and process grant payments
- Name
- Syneos Health Inc.
- Responsibilities
- Specialty laboratory - PK Analysis
Third parties
- {"country":"United States","full_name":"Quest Diagnostics Nichols Institute Inc.","duties_or_roles":"Analysis of 17-OHP, PRA, ASD, CAH Common Mutations, LH (Pediatric), Total testosterone","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"Biotec Services International Limited","duties_or_roles":"Vendor for IP distribution and labeling - United Kingdom and Canada","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Worldwide Clinical Trials Holdings Inc.","duties_or_roles":"Negotiation of site clinical trial agreements, site activation, Trial Master File, Clinical Assessment Technologies, Risk Based Quality Management (RBQM), Safety Management Maintenance, Set up and process grant payments","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"Specialty laboratory - PK Analysis","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"Clinical hematology, chemistry Biomarker analyses","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Zrt Laboratory LLC","duties_or_roles":"Salivary sample analysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Central Institutional Review Board (IRB)","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Millmount Healthcare Limited","duties_or_roles":"Vendor for IP distribution and labeling - EU countries Belgium, Germany, Spain, France, Italy, Poland, Greece","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"MARKEN Germany GmbH","duties_or_roles":"Home health services, Direct-to-Patient (DTP) IP shipments","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Vendor for medical image review and analysis: X-ray, ultrasound, Blood pressure equipment provider","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Hungary","full_name":"Accelsiors Kft.","duties_or_roles":"Negotiation of site clinical trial agreements","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"Electronic patient-reported outcome (e-PRO)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Processing and analysis of blood and urine samples, Clinical hematology, chemistry Biomarker analyses","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"Independent Statistical Center supporting Data Monitoring Committee (DMC)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Andersonbrecon Inc.","duties_or_roles":"Vendor for IP distribution and labeling - USA","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"electronic patient-reported outcome (e-PRO)","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- NBI-74788 (CAPSULE)
- Active Substance
- CRINECERFONT
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Authorisation Status
- Authorized (PRD7876236)
- Orphan Designation
- Yes
- Investigational Product Name
- NBI-74788 (ORAL SOLUTION)
- Active Substance
- CRINECERFONT
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Authorisation Status
- Authorized (PRD7537534)
- Orphan Designation
- Yes
- Investigational Product Name
- Crinecerfont placebo for oral solution
- Modality
- Other
- Investigational Product Name
- Crinecerfont placebo for oral Capsules
- Modality
- Other
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