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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-004873-17 | EudraCT Number | ||
| 2023-509171-16-00 | EU Trial (CTIS) Number |
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This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency. The study consists of a 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 20 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year. |
|
| Crinecerfont | Experimental | Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Crinecerfont | Drug | CRF type 1 receptor antagonist |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24 | Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model. | Baseline, Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Serum Androstenedione at Week 4 | Baseline, Week 4 | |
| Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24 | Week 24 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Development Lead | Neurocrine Biosciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurocrine Clinical Site | Los Angeles | California | 90027 | United States | ||
| Neurocrine Clinical Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38828955 | Derived | Auchus RJ, Hamidi O, Pivonello R, Bancos I, Russo G, Witchel SF, Isidori AM, Rodien P, Srirangalingam U, Kiefer FW, Falhammar H, Merke DP, Reisch N, Sarafoglou K, Cutler GB Jr, Sturgeon J, Roberts E, Lin VH, Chan JL, Farber RH; CAHtalyst Adult Trial Investigators. Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):504-514. doi: 10.1056/NEJMoa2404656. Epub 2024 Jun 1. |
| Label | URL |
|---|---|
| Study website - CAHtalyst Study | View source |
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The study includes a double-blind (DB) placebo-controlled treatment period, an open-label (OL) treatment period, an OL or DB active-controlled treatment period, and an open-label extension (OLE) treatment period. The study is ongoing. Only the primary analysis results (as of 19 July 2023 data cutoff date) have been reported. The final results will be reported after completion of the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received crinecerfont-matched placebo orally twice daily for 24 weeks during the DB placebo-controlled treatment period. After the 24-week DB placebo-controlled treatment period, there was a 6-month, OL treatment period, during which all participants received crinecerfont. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| DB Treatment Period (24 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 30, 2021 | Dec 27, 2024 |
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| Placebo | Drug | Non-active dosage form |
|
| Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24 | Baseline, Week 24 |
| Percent Change From Baseline in Body Weight at Week 24 | Baseline, Week 24 |
| Change From Baseline in Percent Total Fat Mass at Week 24 | Baseline, Week 24 |
| Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4 | Baseline, Week 4 |
| Change From Baseline in Blood Pressure at Week 24 | Baseline, Week 24 |
| Change From Baseline in Glucose Tolerance at Week 24 | Baseline, Week 24 |
| Change From Baseline in Waist Circumference at Week 24 | Baseline, Week 24 |
| Change From Baseline in Menstrual Regularity at Week 24 | Baseline, Week 24 |
| Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24 | Baseline, Week 24 |
| San Diego |
| California |
| 92123 |
| United States |
| Neurocrine Clinical Site | San Francisco | California | 94143 | United States |
| Neurocrine Clinical Site | Aurora | Colorado | 80045 | United States |
| Neurocrine Clinical Site | Atlanta | Georgia | 30329 | United States |
| Neurocrine Clinical Site | Indianapolis | Indiana | 46202 | United States |
| Neurocrine Clinical Site | Bethesda | Maryland | 20982 | United States |
| Neurocrine Clinical Site | Boston | Massachusetts | 02115 | United States |
| Neurocrine Clinical Site | Ann Arbor | Michigan | 48109 | United States |
| Neurocrine Clinical Site | Minneapolis | Minnesota | 55454 | United States |
| Neurocrine Clinical Site | Rochester | Minnesota | 55905 | United States |
| Neurocrine Clinical Site | St Louis | Missouri | 63110 | United States |
| Neurocrine Clinical Site | Great Neck | New York | 11021 | United States |
| Neurocrine Clinical Site | New York | New York | 10029 | United States |
| Neurocrine Clinical Site | Winston-Salem | North Carolina | 27157 | United States |
| Neurocrine Clinical Site | Tulsa | Oklahoma | 74135 | United States |
| Neurocrine Clinical Site | Philadelphia | Pennsylvania | 19104 | United States |
| Neurocrine Clinical Site | Pittsburgh | Pennsylvania | 15224 | United States |
| Neurocrine Clinical Site | Dallas | Texas | 75390 | United States |
| Neurocrine Clinical Site | Seattle | Washington | 98105 | United States |
| Neurocrine Clinical Site | Graz | 8036 | Austria |
| Neurocrine Clinical Site | Vienna | 1090 | Austria |
| Neurocrine Clinical Site | Brussels | 1070 | Belgium |
| Neurocrine Clinical Site | Leuven | 3000 | Belgium |
| Neurocrine Clinical Site | Sofia | 1431 | Bulgaria |
| Neurocrine Clinical Site | Sofia | 1606 | Bulgaria |
| Neurocrine Clinical Site | Halifax | Nova Scotia | B3H 2Y9 | Canada |
| Neurocrine Clinical Site | Hradec Králové | 50005 | Czechia |
| Neurocrine Clinical Site | Angers | 49933 | France |
| Neurocrine Clinical Site | Grenoble | 38700 | France |
| Neurocrine Clinical Site | Le Kremlin-Bicêtre | 94270 | France |
| Neurocrine Clinical Site | Nantes | 44093 | France |
| Neurocrine Clinical Site | Paris | 75651 | France |
| Neurocrine Clinical Site | Paris | 75679 | France |
| Neurocrine Clinical Site | Dresden | 01307 | Germany |
| Neurocrine Clinical Site | Essen | 45147 | Germany |
| Neurocrine Clinical Site | Frankfurt | 60590 | Germany |
| Neurocrine Clinical Site | Leipzig | 04103 | Germany |
| Neurocrine Clinical Site | Munich | 80336 | Germany |
| Neurocrine Clinical Site | Athens | 106 76 | Greece |
| Neurocrine Clinical Site | Athens | 115 27 | Greece |
| Neurocrine Clinical Site | Athens | 11527 | Greece |
| Neurocrine Clinical Site | Thessaloniki | 54642 | Greece |
| Neurocrine Clinical Site | Afula | 1834111 | Israel |
| Neurocrine Clinical Site | Beersheba | 8410101 | Israel |
| Neurocrine Clinical Site | Petah Tikva | 4941480 | Israel |
| Neurocrine Clinical Site | Tel Aviv | 64239 | Israel |
| Neurocrine Clinical Site | Ancona | 60126 | Italy |
| Neurocrine Clinical Site | Bologna | 40138 | Italy |
| Neurocrine Clinical Site | Florence | 50139 | Italy |
| Neurocrine Clinical Site | Messina | 98125 | Italy |
| Neurocrine Clinical Site | Milan | 20132 | Italy |
| Neurocrine Clinical Site | Milan | 20149 | Italy |
| Neurocrine Clinical Site | Naples | 80131 | Italy |
| Neurocrine Clinical Site | Padova | 35128 | Italy |
| Neurocrine Clinical Site | Roma | 00161 | Italy |
| Neurocrine Clinical Site | Leiden | 2333 | Netherlands |
| Neurocrine Clinical Site | Krakow | 31-011 | Poland |
| Neurocrine Clinical Site | Poznan | 60-355 | Poland |
| Neurocrine Clinical Site | Warsaw | 01-809 | Poland |
| Neurocrine Clinical Site | Porto | 4200-319 | Portugal |
| Neurocrine Clinical Site | Belgrade | 11000 | Serbia |
| Neurocrine Clinical Site | Madrid | 28034 | Spain |
| Neurocrine Clinical Site | Seville | 41013 | Spain |
| Neurocrine Clinical Site | Gothenburg | 41345 | Sweden |
| Neurocrine Clinical Site | Stockholm | 17176 | Sweden |
| Neurocrine Clinical Site | Cardiff | CF14 4HH | United Kingdom |
| Neurocrine Clinical Site | London | WC1B 5EH | United Kingdom |
| Neurocrine Clinical Site | Manchester | M20 4BX | United Kingdom |
| Neurocrine Clinical Site | Salford | M6 8HD | United Kingdom |
| Crinecerfont |
Participants received crinecerfont orally twice daily for 24 weeks during the DB placebo-controlled treatment period. After the 24-week DB placebo-controlled treatment period, there was a 6-month, OL treatment period, during which all participants received crinecerfont. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| OL Treatment Period (6 Months) |
|
|
Full Analysis Set included all randomized participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received crinecerfont-matched placebo orally twice daily for 24 weeks during the DB placebo-controlled treatment period. After the 24-week DB placebo-controlled treatment period, there was a 6-month, OL treatment period, during which all participants received crinecerfont. |
| BG001 | Crinecerfont | Participants received crinecerfont orally twice daily for 24 weeks during the DB placebo-controlled treatment period. After the 24-week DB placebo-controlled treatment period, there was a 6-month, OL treatment period, during which all participants received crinecerfont. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Daily Glucocorticoid Dose | Mean | Standard Deviation | milligrams (mg)/day |
| |||||||||||||||
| Daily Glucocorticoid Dose Adjusted for Body Surface Area | Mean | Standard Deviation | mg/square meter (m^2)/day |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24 | Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model. | Full Analysis Set included all randomized participants. | Posted | Least Squares Mean | Standard Error | percent change | Baseline, Week 24 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Serum Androstenedione at Week 4 | Not Posted | Baseline, Week 4 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24 | Not Posted | Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Body Weight at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent Total Fat Mass at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4 | Not Posted | Baseline, Week 4 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Blood Pressure at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Glucose Tolerance at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Waist Circumference at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Menstrual Regularity at Week 24 | Not Posted | Baseline, Week 24 | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24 | Not Posted | Baseline, Week 24 | Participants |
Baseline up to Week 24
Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | DB Period: Placebo | Participants received crinecerfont-matched placebo orally twice daily for 24 weeks during the DB placebo-controlled treatment period. | 0 | 60 | 0 | 59 | 39 | 59 |
| EG001 | DB Period: Crinecerfont | Participants received crinecerfont orally twice daily for 24 weeks during the DB placebo-controlled treatment period. | 0 | 122 | 4 | 122 | 76 | 122 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Groin abscess | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Adrenocortical insufficiency acute | Endocrine disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Blood androstenedione increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Renin increased | Investigations | MedDRA 26.0 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Neurocrine Medical Information Call Center | Neurocrine Biosciences | 877-641-3461 | medinfo@neurocrine.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 10, 2023 | Dec 27, 2024 | SAP_001.pdf |
| 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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| ID | Term |
|---|---|
| C000723083 | crinecerfont |
Not provided
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| Adverse Event |
|
| Ongoing in the Study |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|