Symptoms and General Pathology

All Conditions

Nervous System Diseases

Gland and Hormone Related Diseases

Heart and Blood Diseases

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Cushing Syndrome

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ACTH-Secreting Pituitary Adenoma

Pituitary ACTH Hypersecretion

ACTH Syndrome, Ectopic

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ACTH-secreting Pituitary Adenoma

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Mifepristone

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Contraceptives, Oral

Contraceptive Agents, Female

Contraceptives, Postcoital

Hormones

Hormone Antagonists

Contraceptive Agents, Hormonal

mifepristone at doses from 300mg/day up to 1200mg/day

Coleman Gross, M.D.

This is a compassionate use study. In addition to providing compassionate use access to mifepristone, objectives of the study will be to evaluate the safety and utility of mifepristone in the treatment of the signs and symptoms of endogenous Cushing's syndrome when given on a compassionate use basis. The study will only enroll subjects whose physicians have determined that medical treatment is needed to control the symptoms or signs of hypercortisolemia.

Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome

Compassionate Use Protocol for the Administration of CORLUX® (Mifepristone) in the Treatment of the Signs and Symptoms of Endogenous Cushing's Syndrome

Safety was assessed at all visits and adverse events were recorded.

Corcept Therapeutics

All patients who received at least one dose of study drug are included.

Age, Categorical

Age, Continuous

Sex: Female, Male

United States

Region of Enrollment

Subjects who received one dose of study drug were included in the safety analysis.

Medical Director

Due to the small number of patients enrolled (N=4), no formal assessments were planned. All patients who received study drug were included in the safety review.

Number of Participants With Adverse Events

Overall Study

Paraneoplastic Syndromes

Anti-Inflammatory Agents

Hydrocortisone

Hydrocortisone 17-butyrate 21-propionate

Hydrocortisone acetate

Hydrocortisone hemisuccinate

Adrenocorticotropic Hormone

Melanocyte-Stimulating Hormones

beta-Endorphin

Active drug by mouth each morning for up to 12 weeks

Placebo by mouth each morning for up to 12 weeks

11β hydroxysteroid dehydrogenase type 1 (HSD-1) inhibitor

SPI-62

Inactive tablets identical to SPI-62 tablets

Placebo

Frank Czerwiec, MD

This is a randomized, placebo-controlled, study of SPI-62 in subjects with ACTH-dependent Cushing's syndrome caused by a non-adrenal tumor. Subjects will receive each of the following 2 treatments for 24 weeks: SPI-62 and matching placebo with the option of long-term extension.

This is a multicenter, randomized, placebo-controlled, Phase 2 study to evaluate the pharmacologic effect, efficacy, and safety of SPI-62 in subjects with ACTH-dependent Cushing's syndrome. Each subject who provides consent and meets all inclusion and exclusion criteria will participate in a screening period (Days -35 to -8), a baseline period (Days -7 to -1), and a treatment period (Day 1 of Week 1 to Day 168 ± 3 days of Week 24) and, the option of long-term extension. Subjects have the option to continue with the study on active study drug and return to the site every 3 months for blood tests and study drug dispensing. The visits may be conducted remotely if testing can be arranged.

SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome

The urinary HSD-1 ratio (tetrahydrocortisol + allotetrahydrocortisol ) / tetrahydrocortisone will be used as a biomarker of HSD-1 activity in liver. The primary analysis will include only subjects with Cushing's disease.

Adverse events including clinically significant abnormal values on clinical laboratory evaluations, continuous glucose monitoring (CGM), 12-lead ECGs, vital signs measurements (including orthostatic vital sign measurements), physical examinations, and HPA and HPG axis biomarkers

Sparrow Pharmaceuticals

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome	NCT01371565	Cushing's Disea... Cushing's Syndr...	Mifepristone	18 Years -	Corcept Therapeutics
SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome	NCT05307328	Cushing's Syndr... Cushing Disease... Cortisol Excess Cortisol; Hyper... Cortisol Overpr... Ectopic ACTH Se...	SPI-62 Placebo	18 Years -	Sparrow Pharmaceuticals
Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome	NCT01371565	Cushing's Disea... Cushing's Syndr...	Mifepristone	18 Years -	Corcept Therapeutics

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