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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-519578-38-00 | EU Trial (CTIS) Number |
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The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
This Phase 2/3 plus open-label extension study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic CAH. Part A is a Phase 2, open-label, semi-sequential cohorts portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is the open-label extension (OLE) portion of the study. Participants in Part A and B are eligible to enroll in Part C (OLE).
A total of approximately 153 participants may be enrolled in the study (planned and optional cohorts) ages 1 to < 18 years old.
The first 3 cohorts in Part A are for ages 12 to <18 years and will be semi-sequential, and Safety Review Committee (SRC) review of data and approval to proceed is required prior to enrolling each subsequent cohort. The fourth cohort in Part A is for ages 1 to 11 years old and will begin after Cohorts 1 and 2 have been completed, additional requirements are fulfilled, and following SRC review of Cohorts 1 and 2 data.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment (Part A) | Experimental | Open-label, semi-sequential cohorts. |
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| Active Treatment (Part B) | Experimental | Randomized, Parallel Arms, Double-Blind |
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| Placebo (Part B) | Placebo Comparator | Randomized, Parallel Arms, Double-Blind |
|
| Open-Label Treatment (Part C) | Experimental | Open-label treatment period for participants entering Part C from Part A and B. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Atumelnant | Drug | Atumelnant, tablets, once daily by mouth, weight-based dosing |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in morning serum androstenedione (A4) (Part A) | Week 8 | |
| Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B) | Week 28 | |
| Change from baseline in serum early morning A4 over time (Part C) | Up to Week 260 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) (Part A) | Week 8 | |
| Plasma and/or blood concentrations of atumelnant (Part A) | Up to Week 8 | |
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Inclusion Criteria:
Part A and B participants are eligible to be included in the study only if all of the following criteria apply:
Part C inclusion criteria require participants to complete treatment in either Part A or Part B and in the Investigator's opinion it would benefit the participant to continue in Part C, regardless of age.
Exclusion Criteria:
Part A and Part B: Individuals in Part A and Part B who meet any of the following criteria will be excluded from participation in this study:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Crinetics Clinical Trials | Contact | 833-827-9741 | clinicaltrials@crinetics.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Ron Conway Gateway Medical Building | Recruiting | San Francisco | California | 94158 | United States | |
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| Placebo | Drug | Placebo, tablets, once daily by mouth, weight-based dosing |
|
| Change from baseline in serum early morning A4 (Part B) |
| Week 4 |
| Change from baseline in serum early morning 17-OHP (Part B) | Week 4 |
| Proportion of participants with physiologic GC dose while serum early morning A4 ≤ULN (Part B) | Week 28 |
| Change from baseline in serum early morning 17-OHP over time (Part C) | Up to Week 260 |
| Percent change from baseline in GC daily dose over time (Part C) | Up to Week 260 |
| Proportion of participants with physiologic GC dose while serum early morning A4 ≤ULN over time (Part C) | Up to Week 260 |
| Boston's Children's Hospital |
| Recruiting |
| Boston |
| Massachusetts |
| 02115 |
| United States |
| University of Michigan | Recruiting | Ann Arbor | Michigan | 48109 | United States |
| University of Minnesota | Recruiting | Minneapolis | Minnesota | 55454 | United States |
| Rutgers Robert Wood Johnson Medical School | Recruiting | New Brunswick | New Jersey | 08901 | United States |
| Children's Hospital of Philadelphia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Cook Children's Health Care System | Recruiting | Fort Worth | Texas | 76104 | United States |
| University of Virginia Health System | Recruiting | Charlottesville | Virginia | 22903 | United States |
| Instituto de Investigaciones Metabólicas | Recruiting | Buenos Aires | Buenos Aires | C1012AAR | Argentina |
| Hospital de Niños de la Santísima Trinidad | Recruiting | Córdoba | Córdoba Province | X5000 | Argentina |
| Hospital Italiano de Buenos Aires | Recruiting | Buenos Aires | C1199ABB | Argentina |
| Instituto Médico Especializado (IME) | Recruiting | Buenos Aires | C1405BCH | Argentina |
| CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez | Recruiting | Buenos Aires | C1425EFD | Argentina |
| Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead | Not yet recruiting | Westmead | New South Wales | 2145 | Australia |
| Queensland Children's Hospital | Not yet recruiting | South Brisbane | Queensland | 4101 | Australia |
| Monash Children's Hospital, Monash Health | Recruiting | Clayton | Victoria | 3168 | Australia |
| Perth Children's Hospital | Not yet recruiting | Nedlands | Western Australia | 6009 | Australia |
| UZA (Antwerp University Hospital) | Recruiting | Edegem | Antwerp | 2650 | Belgium |
| Cliniques Universitaires Saint-Luc | Recruiting | Brussels | Brussels Capital | 1200 | Belgium |
| UZ Gent (University Hospital Ghent) | Recruiting | Ghent | East Flanders | 9000 | Belgium |
| UZ Leuven (Universitair Ziekenhuis Leuven) | Recruiting | Leuven | Flemish Brabant | 3000 | Belgium |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP) | Recruiting | São Paulo | São Paulo | 05403-000 | Brazil |
| Centre Hospitalier Universitaire (CHU) d'Angers | Recruiting | Angers | 49100 | France |
| Hopital Kremlin-Bicétre - APHP Paris Saclay | Recruiting | Le Kremlin-Bicêtre | 94270 | France |
| Hopital Jeanne de Flandre - CHU de Lille | Recruiting | Lille | 59037 | France |
| APHM -Hopital La Timone Enfants | Recruiting | Marseille | 13385 Cedex 5 | France |
| Hopital Necker - Enfants Malades | Recruiting | Paris | 75015 | France |
| Hopital Robert Debre | Recruiting | Paris | 75019 | France |
| Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin | Recruiting | Tübingen | Baden | 72076 | Germany |
| Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie | Recruiting | Berlin | 13353 | Germany |
| AOU Federico II | Recruiting | Naples | Campania | 80131 | Italy |
| IRCCS Istituto Giannina Gaslini | Recruiting | Genoa | Liguria | 16147 | Italy |
| IRCCS Ospedale San Raffaele | Recruiting | Milan | Lombardy | 20132 | Italy |
| Azienda Ospedaliera Universitaria Meyer IRCCS | Recruiting | Florence | Tuscany | 50139 | Italy |
| lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych | Recruiting | Lodz | Poland | 93-338 | Poland |
| Uniwersytecki Szpital Kliniczny Nr 1 im. Prof. Tadeusza Sokotowskiego PUM w Szczecinie, Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie | Recruiting | Szczecin | West Pomeranian Voivodeship | 71-252 | Poland |
| Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank | Recruiting | Sheffield | S10 2TH | United Kingdom |
| ID | Term |
|---|---|
| D000312 | Adrenal Hyperplasia, Congenital |
| ID | Term |
|---|---|
| D047808 | Adrenogenital Syndrome |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D043202 | Steroid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |
| D006058 | Gonadal Disorders |
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