Clinical trial • Rare Disease|Endocrinology
ATUMELNANT for Classic congenital adrenal hyperplasia
Clinical trial of ATUMELNANT for Classic congenital adrenal hyperplasia.
Overview
- Trial Therapeutic Area
- Rare Disease|Endocrinology
- Trial Disease
- Classic congenital adrenal hyperplasia
- Drug Modality
- Small molecule
Key dates
- Initial CTIS Submission Date
- 08-08-2025
- First CTIS Authorization Date
- 02-12-2025
Trial design
Randomised, placebo (tablet) comparator; active arms receive atumelnant (atumelnant 80 mg tablets and atumelnant 120 mg tablets). specific dosing schedule not specified in the record.-controlled trial in Sweden, Germany, France and others.
- Randomised
- Yes
- Comparator
- Placebo (tablet) comparator; active arms receive Atumelnant (Atumelnant 80 mg tablets and Atumelnant 120 mg tablets). Specific dosing schedule not specified in the record.
- Target Sample Size
- 86
- Trial Duration For Participant
- 224
Eligibility
Recruits 86 No vulnerable population selected (isVulnerablePopulationSelected: false). Participants must be adults (≥18 years) and sign the Informed Consent Form; assent is not applicable..
- Vulnerable Population
- No vulnerable population selected (isVulnerablePopulationSelected: false). Participants must be adults (≥18 years) and sign the Informed Consent Form; assent is not applicable.
Inclusion criteria
- {"criterion_text":"- 1. Male or female, between ≥18 to <75 years of age at the time of signing the ICF.\n- 2. Willing and able to understand and adhere to the study procedures as specified in the protocol and comply with the study treatment.\n- 3. Have classic CAH due to 21-OHD confirmed by the Investigator and approved by the Medical Monitor.\n- 4. Participants with levels of morning serum A4 as follows: A4 >ULN and treated with <11 mg/m2/day (physiologic) GC doses in hydrocortisone equivalents OR normal A4 (above mid-range to ≤ULN) and treated with ≥15 mg/m2/day GC doses in hydrocortisone equivalents OR A4 >ULN and treated with ≥11 mg/m2 /day GC doses in hydrocortisone equivalents.\n- 5. On a stable (defined as no dose change of >X mg/day hydrocortisone equivalent within 2 months prior to Screening) regimen of GC replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone, dexamethasone) at the time of informed consent.\n- 6. If treated with mineralocorticoids (fludrocortisone), the dose should be stable for at least 2 months prior to Screening without orthostatic hypotension, and with serum sodium and potassium in the normal range.\n- 7. If on estrogen therapy (any route), the dose must be stable for at least 3 months prior to Screening.\n- 8. Female participants who engage in heterosexual intercourse must: a. Be of nonchildbearing potential, defined as either surgically sterile (ie, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), OR b. Agree to use a highly effective or a clinically acceptable method of contraception from the beginning of Screening until at least 2 weeks after the last dose of study drug. Contraceptive use by men and women also should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Periodic abstinence (ie, calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. c. See Section 12.1.4 for additional contraception guidance.\n- 9. Male participants who engage in heterosexual intercourse must: a. Agree to use a condom when sexually active with a female partner of childbearing potential from Screening until at least 2 weeks after the last dose of study drug (or be surgically sterile [ie, vasectomy with a confirmed absence of sperm in ejaculate]) OR b. Agree to remain abstinent on a long-term and persistent basis during the study and until at least 2 weeks after the last dose of study drug. c. Agree to not donate sperm for the duration of the study and until at least 2 weeks after the last dose of study drug. d. See Section 12.1.4 for additional contraception guidance."}
Exclusion criteria
- {"criterion_text":"- Refer to section 5.3 of the Protocol for the full exclusion criteria."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Proportion of participants with morning post-GC A4 ≤ULN who are on physiologic GC replacement at Week 32","definition_or_measurement_approach":"Proportion of participants whose morning post-glucocorticoid (post-GC) androstenedione (A4) measurement is ≤ upper limit of normal (ULN) while receiving physiologic GC replacement at Week 32."}
Secondary endpoints
- {"endpoint_text":"- Percent change from baseline of morning A4 at Week 2\n- Percent change from baseline of morning 17-OHP at Week 32\n- Proportion of participants with morning pre-GC A4 ≤ULN who are on physiologic GC replacement at Week 32\n- Percent change from baseline in GC daily dose when morning post-GC A4 ≤ULN at week 32","definition_or_measurement_approach":"Percent change endpoints are calculated as percentage change from baseline for morning A4 (at Week 2) and morning 17-OHP (at Week 32). Proportion endpoints are based on laboratory measurements of morning A4 (pre- or post-GC as specified) compared to ULN and GC dosing status at Week 32. Percent change in GC daily dose compares baseline daily GC dose to dose at Week 32 when morning post-GC A4 ≤ULN."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 86
- Recruitment Window Months
- 17
- Consent Approach
- Informed consent obtained from adult participants (must sign Informed Consent Form). Country-specific main ICF documents are provided (examples: Main ICF in Swedish, German, French, Dutch, Polish, Italian, English where applicable). Pre-ICF telephone data consent forms are available to capture consent for telephone pre-screening. Additional ICFs for pregnancy/partner/newborn data are present in some country document sets. Assent is not applicable as participants must be ≥18 years.
Methods
- Digital half-page advertisements (documents titled 'Digital Half Page Ad' / 'Digital-Half-Page-Ad') — available country-specific versions (SE, DE, FR, AT, NL, PL, IT) as recruitment materials.
- Half-page print advertisements / promotional adverts (documents titled 'Half-Page-Ad' / 'Half Page Ad') — country-specific (SE, DE, FR, AT, NL, PL, IT).
- Recruitment brochures and flyers (documents titled 'Recruitment-Brochure', 'Recruitment-Flyer') — country-specific materials present for SE, DE, FR, AT, NL, PL, IT.
- PI-to-Patient letters (documents titled 'PI-to-Patient Letter' / 'PI to Patient Letter') — country-specific patient contact letters.
- Recruitment arrangement forms and site procedures documents (documents titled 'Recruitment-arrangements' / 'Recruitment-and-Informed-Consent-Procedure') submitted per country.
- Pre-ICF telephone data consent (documents titled 'Pre-ICF Telephone Data Consent' / 'Pre-ICF-Telephone-Data-Consent') indicating telephone-based pre-screen/contact methods.
Geography
- Total Number Of Sites
- 28
- Total Number Of Participants
- 86
Sweden
- Earliest CTIS Part Ii Submission Date
- 03-11-2025
- Latest Decision Or Authorization Date
- 04-12-2025
- Processing Time Days
- 31
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Sahlgrenska University Hospital-Vaestra Goetalandsregionen
- Department Name
- Medicinmottagning Sahlgrenska
- Principal Investigator Name
- Gudmundur Johannsson
- Principal Investigator Email
- gudmundur.johannsson@medic.gu.se
- Contact Person Name
- Gudmundur Johannsson
- Contact Person Email
- gudmundur.johannsson@medic.gu.se
- Site Name
- Karolinska University Hospital
- Department Name
- Mottagning Endokrinologi
- Principal Investigator Name
- Henrik Falhammar
- Principal Investigator Email
- henrik.falhammar@ki.se
- Contact Person Name
- Henrik Falhammar
- Contact Person Email
- henrik.falhammar@ki.se
Germany
- Earliest CTIS Part Ii Submission Date
- 24-10-2025
- Latest Decision Or Authorization Date
- 03-12-2025
- Processing Time Days
- 40
- Number Of Sites
- 4
- Number Of Participants
- 5
Sites
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- Klinik für Endokrinologie, Diabetologie und Stoffwechsel
- Principal Investigator Name
- Dagmar Führer-Sakel
- Principal Investigator Email
- Dagmar.Fuehrer-Sakel@uk-essen.de
- Contact Person Name
- Dagmar Führer-Sakel
- Contact Person Email
- Dagmar.Fuehrer-Sakel@uk-essen.de
- Site Name
- Medicover Medizin gGmbH
- Principal Investigator Name
- Günter Stalla
- Principal Investigator Email
- guenter.stalla@medicover.de
- Contact Person Name
- Günter Stalla
- Contact Person Email
- guenter.stalla@medicover.de
- Site Name
- Universitaetsklinikum Wuerzburg AöR
- Department Name
- Medizinische Klinik I, Lehrstuhl für Endokrinologie und Diabetologie
- Principal Investigator Name
- Irina Chifu
- Principal Investigator Email
- chifu_i@ukw.de
- Contact Person Name
- Irina Chifu
- Contact Person Email
- chifu_i@ukw.de
- Site Name
- LMU Klinikum Muenchen AöR
- Department Name
- Medizinische Klinik und Poliklinik IV
- Principal Investigator Name
- Nicole Reisch
- Principal Investigator Email
- Nicole.reisch@med.uni-muenchen.de
- Contact Person Name
- Nicole Reisch
- Contact Person Email
- Nicole.reisch@med.uni-muenchen.de
France
- Earliest CTIS Part Ii Submission Date
- 14-11-2025
- Latest Decision Or Authorization Date
- 02-12-2025
- Processing Time Days
- 18
- Number Of Sites
- 7
- Number Of Participants
- 9
Sites
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Service d’Endocrinologie, Diabétologie et Maladies métaboliques
- Principal Investigator Name
- Virginie GROUTHIER
- Principal Investigator Email
- virginie.grouthier@chu-bordeaux.fr
- Contact Person Name
- Virginie GROUTHIER
- Contact Person Email
- virginie.grouthier@chu-bordeaux.fr
- Site Name
- CHRU De Nancy
- Department Name
- Département d’Endocrinologie
- Principal Investigator Name
- George RILEY
- Principal Investigator Email
- g.riley@chru-nancy
- Contact Person Name
- George RILEY
- Contact Person Email
- g.riley@chru-nancy
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- Département d'Endocrinologie, Diabétologie et Nutrition
- Principal Investigator Name
- Claire BRIET
- Principal Investigator Email
- Claire.briet@chu-angers.fr
- Contact Person Name
- Claire BRIET
- Contact Person Email
- Claire.briet@chu-angers.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Département d’Endocrinologie et Médecine de la Reproduction & CIC Paris Est
- Principal Investigator Name
- Anne BACHELOT
- Principal Investigator Email
- anne.bachelot@aphp.fr
- Contact Person Name
- Anne BACHELOT
- Contact Person Email
- anne.bachelot@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Service d'Endocrinologie
- Principal Investigator Name
- Aude BRAC DE LA PERRIERE
- Principal Investigator Email
- aude.brac@chu-lyon.fr
- Contact Person Name
- Aude BRAC DE LA PERRIERE
- Contact Person Email
- aude.brac@chu-lyon.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- Département d'Endocrinologie Diabétologie – Nutrition & UIC THORAX, ENDROCRINLOGIE
- Principal Investigator Name
- Delphine DRUI
- Principal Investigator Email
- delphine.drui@chu-nantes.fr
- Contact Person Name
- Delphine DRUI
- Contact Person Email
- delphine.drui@chu-nantes.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux (additional site entries may exist)
Austria
- Earliest CTIS Part Ii Submission Date
- 03-11-2025
- Latest Decision Or Authorization Date
- 08-12-2025
- Processing Time Days
- 35
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Medicine III (Division of Endocrinology and Metabolism)
- Principal Investigator Name
- Florian Kiefer
- Principal Investigator Email
- florian.kiefer@meduniwien.ac.at
- Contact Person Name
- Florian Kiefer
- Contact Person Email
- florian.kiefer@meduniwien.ac.at
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-11-2025
- Latest Decision Or Authorization Date
- 03-12-2025
- Processing Time Days
- 30
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- Internal Medicine
- Principal Investigator Name
- N.R. Biermasz
- Principal Investigator Email
- n.r.biermasz@lumc.nl
- Contact Person Name
- N.R. Biermasz
- Contact Person Email
- n.r.biermasz@lumc.nl
Poland
- Earliest CTIS Part Ii Submission Date
- 31-10-2025
- Latest Decision Or Authorization Date
- 08-12-2025
- Processing Time Days
- 38
- Number Of Sites
- 4
- Number Of Participants
- 9
Sites
- Site Name
- Centrum Zdrowia MDM
- Principal Investigator Name
- Urszula Ambroziak
- Principal Investigator Email
- UAMBROZIAK@WUM.EDU.PL
- Contact Person Name
- Urszula Ambroziak
- Contact Person Email
- UAMBROZIAK@WUM.EDU.PL
- Site Name
- Instytut Centrum Zdrowia Matki Polki
- Department Name
- Klinika Endokrynologii i Chorob Metabolicznych
- Principal Investigator Name
- Magdalena Stasiak
- Principal Investigator Email
- magdalena.stasiak@iczmp.edu.pl
- Contact Person Name
- Magdalena Stasiak
- Contact Person Email
- magdalena.stasiak@iczmp.edu.pl
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
- Department Name
- Oddzial Kliniczny Endokrynologii, Endokrynologii Onkologicznej, Meydcyny Nuklearnej i Chorob Wewnetr
- Principal Investigator Name
- Alicja Hubalewska-Dydejczyk
- Principal Investigator Email
- alahub@cm-uj.krakow.pl
- Contact Person Name
- Alicja Hubalewska-Dydejczyk
- Contact Person Email
- alahub@cm-uj.krakow.pl
- Site Name
- Uniwersyteckie Centrum Stomatologii I Medycyny Specjalistycznej Sp. z o.o.
- Department Name
- Poradnia Internistyczna
- Principal Investigator Name
- Marek Ruchala
- Principal Investigator Email
- mruchala@ump.edu.pl
- Contact Person Name
- Marek Ruchala
- Contact Person Email
- mruchala@ump.edu.pl
Italy
- Earliest CTIS Part Ii Submission Date
- 28-08-2025
- Latest Decision Or Authorization Date
- 23-01-2026
- Processing Time Days
- 148
- Number Of Sites
- 9
- Number Of Participants
- 14
Sites
- Site Name
- Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
- Department Name
- UOC Malattie endocrine, del Ricambio e della Nutrizione
- Principal Investigator Name
- Giorgio Arnaldi
- Principal Investigator Email
- giorgio.arnaldi@policlinico.pa.it
- Contact Person Name
- Giorgio Arnaldi
- Contact Person Email
- giorgio.arnaldi@policlinico.pa.it
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- UOC Endocrinologia, Diabetologia e Nutrizione
- Principal Investigator Name
- Rosario Pivonello
- Principal Investigator Email
- rosario.pivonello@unina.it
- Contact Person Name
- Rosario Pivonello
- Contact Person Email
- rosario.pivonello@unina.it
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico Umberto I
- Department Name
- UOC Endocrinologia, Malattie del Metabolismo e Andrologia
- Principal Investigator Name
- Andrea Isidori
- Principal Investigator Email
- andrea.isidori@uniroma1.it
- Contact Person Name
- Andrea Isidori
- Contact Person Email
- andrea.isidori@uniroma1.it
- Site Name
- IRCCS Ospedale Policlinico San Martino
- Department Name
- Dipartimento di Medicina Interna, Unità Operativa Clinica Endocrinologica,
- Principal Investigator Name
- Federico Gatto
- Principal Investigator Email
- federico.gatto@hsanmartino.it
- Contact Person Name
- Federico Gatto
- Contact Person Email
- federico.gatto@hsanmartino.it
- Site Name
- Istituto Auxologico Italiano
- Department Name
- Unità di Endocrinologia e Malattie del Metabolismo
- Principal Investigator Name
- Valentina Morelli
- Principal Investigator Email
- v.morelli@auxologico.it
- Contact Person Name
- Valentina Morelli
- Contact Person Email
- v.morelli@auxologico.it
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- Unità di endocrinologia
- Principal Investigator Name
- Mattia Barbot
- Principal Investigator Email
- mattia.barbot@unipd.it
- Contact Person Name
- Mattia Barbot
- Contact Person Email
- mattia.barbot@unipd.it
- Site Name
- Humanitas Mirasole S.p.A.
- Department Name
- Endocrinology and Diabetology Unit
- Principal Investigator Name
- Andrea Gerardo Antonio Lania
- Principal Investigator Email
- andrea.lania@humanitas.it
- Contact Person Name
- Andrea Gerardo Antonio Lania
- Contact Person Email
- andrea.lania@humanitas.it
- Site Name
- Azienda Ospedaliero Universitaria Di Modena
- Department Name
- SC Endocrinologia
- Principal Investigator Name
- Vincenzo Rochira
- Principal Investigator Email
- rochira.vincenzo@unimore.it
- Contact Person Name
- Vincenzo Rochira
- Contact Person Email
- rochira.vincenzo@unimore.it
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- Department of Pediatrics
- Principal Investigator Name
- Gianni Russo
- Principal Investigator Email
- russo.gianni@hsr.it
- Contact Person Name
- Gianni Russo
- Contact Person Email
- russo.gianni@hsr.it
Sponsor
Primary sponsor
- Full Name
- Crinetics Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- PPD Development LP
Third parties
- {"country":"United States","full_name":"Pharmaron (Germantown) Lab Services Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Qualitymetric Incorporated LLC","duties_or_roles":"In trial optional interview","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"LabConnect Europe B.V.","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Edetek Inc.","duties_or_roles":"ePRO Management","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Canada","full_name":"Altasciences Compagnie Inc.","duties_or_roles":"Plasma PK, Whole blood PK (Mitra)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"13","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Germany","full_name":"Labor Berlin Charite Vivantes GmbH","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Canada","full_name":"Everest Clinical Research Corporation","duties_or_roles":"6","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac safety","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"SGS Analytics Germany GmbH","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Germany","full_name":"LabConnect GmbH","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Advarra Inc.","duties_or_roles":"Optional training platform","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Atumelnant 120 mg tablets
- Active Substance
- ATUMELNANT
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- Oral
- Authorisation Status
- 1
- Starting Dose
- 120 mg
- Dose Levels
- 120 mg (active); 80 mg also listed as an active dose form in trial materials
- Maximum Dose
- 120 mg
- Investigational Product Name
- Atumelnant 80 mg tablets
- Active Substance
- ATUMELNANT
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- Oral
- Authorisation Status
- 1
- Starting Dose
- 80 mg
- Dose Levels
- 80 mg (active); 120 mg also listed as an active dose form in trial materials
- Maximum Dose
- 120 mg
- Investigational Product Name
- Tablet (Placebo)
- Modality
- Other
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