Not provided
Not provided
Not provided
Not provided
Not provided
Following an interim data review, further investment in nevanimibe has been discontinued
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nevanimibe hydrochloride | Experimental | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nevanimibe hydrochloride | Drug | During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Subjects Achieving Serum 17-OHP Targets | The primary efficacy endpoint was the overall response rate within each cohort, defined as the percentage of patients achieving serum 17-OHP targets as follows:
| Through Day 113 |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto | Ribeirão Preto | 14051-140 | Brazil | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35283460 | Derived | White PC. Emerging treatment for congenital adrenal hyperplasia. Curr Opin Endocrinol Diabetes Obes. 2022 Jun 1;29(3):271-276. doi: 10.1097/MED.0000000000000723. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Screening Period of 2-14 weeks After signing informed consent, patients with classic CAH entered the Screening Period to assess preliminary eligibility for the study based on the inclusion and exclusion criteria. In addition, pertinent information was collected such as past medical history, demographic data, and prior and current medications.
Global Amendment 1:
Approximately 20-24 adults with a documented history of classic CAH will be enrolled
Global Amendment 2:
Approximately 20-24 evaluable adults with a documented history of classic CAH will be enrolled 20-24 patients planned, 15 patients analyzed
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Nevanimibe HCl | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Nevanimibe HCl | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Percentage of Subjects Achieving Serum 17-OHP Targets | The primary efficacy endpoint was the overall response rate within each cohort, defined as the percentage of patients achieving serum 17-OHP targets as follows:
| Posted | Number | percent | Through Day 113 |
|
From the time of informed consent through treatment period and until 30 days after the last dose of study drug
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Nevanimibe HCl Dose Level: <500 mg BID | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Uroinfection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Drug hypersensitivity | Immune system disorders | MedDRA (23.0) | Non-systematic Assessment |
Nevanimibe hydrochloride administered orally at doses of 500-2000 mg BID for up to 16 weeks in the first 10 evaluable patients did not result in sufficient efficacy. The Sponsor has decided to refrain from further development at this time.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Information | Millendo Therapeutics | +1 734-845-9000 | millendo@millendo.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 31, 2019 | Jan 26, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 13, 2020 | Jan 26, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D000312 | Adrenal Hyperplasia, Congenital |
| ID | Term |
|---|---|
| D047808 | Adrenogenital Syndrome |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Universidade Federal de São Paulo, Escola Paulista de Medicina |
| São Paulo |
| 04037-002 |
| Brazil |
| Hospital das ClÃnicas da FMUSP - Prédio do Instituto Central | São Paulo | 05403-900 | Brazil |
| Institute of Endocrinology | Prague | Czechia |
| Hospital Pitié-Salpetrière | Paris | France |
| Bnai Zion Medical Center | Haifa | Israel |
| Beilinson Hospital | Petah Tikva | Israel |
| Tel-Aviv-Sourasky Medical Center | Tel Aviv | Israel |
| Hospital General Universitario Gregorio Marañón | Madrid | Spain |
| Hospital Universitario Virgen del RocÃo | Seville | Spain |
| University Hospital La Fe | Valencia | Spain |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Baseline serum 17-hydroxyprogesterone | Mean | Standard Deviation | ng/dL |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| 0 |
| 14 |
| 0 |
| 14 |
| 3 |
| 14 |
| EG001 | Nevanimibe HCl Dose Level:500 to <1000 mg BID | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. | 0 | 5 | 0 | 5 | 5 | 5 |
| EG002 | Nevanimibe HCl Dose Level: 1000 to <1500 mg BID | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. | 0 | 14 | 1 | 14 | 8 | 14 |
| EG003 | Nevanimibe HCl Dose Level: 1500 to <2000 mg BID | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. | 0 | 9 | 0 | 9 | 7 | 9 |
| EG004 | Nevanimibe HCl Dose Level:2000 mg BID | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Nevanimibe hydrochloride: During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. | 0 | 9 | 0 | 9 | 5 | 9 |
| Asthenia, Chest pain, Fatigue, Noncardiac chest pain, Pyrexia | General disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Insomnia, Panic attack | Psychiatric disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Vaginal hemorrhage | Reproductive system and breast disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
|
| Blood glucose increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
|
| Cough, Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Disturbance in attention, Headache, Syncope | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Dry eye | Eye disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Abdominal discomfort, Diarrhoea, Dyspepsia, Flatulence, Gingival bleeding, Nausea | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Dysuria, Hypertonic bladder, Micturition urgency, Pollakiuria | Renal and urinary disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Acne, Eczema, Pruritus, Rash, Urticaria | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
|
| Cystitis, Nasopharyngitis, Urinary tract infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
|
Not provided
Not provided
| 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 |