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NCT Number: NCT05307328

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

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.

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Key information

Conditions

Cushing's Syndrome I ACTH Syndrome, Ectopic Adrenal Gland Diseases Adrenocortical Hyperfunction Brain Diseases Central Nervous System Diseases Cortisol Excess Cortisol Overproduction Cortisol; Hypersecretion Cushing Disease Due to Increased ACTH Secretion Cushing Syndrome Ectopic ACTH Secretion Endocrine System Diseases Hyperpituitarism Hypothalamic Diseases Neoplasms Nervous System Diseases Paraneoplastic Endocrine Syndromes Paraneoplastic Syndromes Pituitary ACTH Hypersecretion Pituitary Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Medical University of Plovdiv, Plovdiv, Bulgaria

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or non-menstruating female
  • 18 years or older
  • Active and consistent cortisol excess
  • Documented diagnosis of ACTH-dependent Cushing's syndrome including Cushing's disease, ectopic ACTH secretion, and ectopic CRH secretion.

Exclusion criteria

  • Recent (within 6 weeks) surgery for Cushing's or surgery planned within 24 weeks of randomization.
  • History of any fractionated radiation therapy for Cushing's within the past 2 years or conventional radiation therapy within 4 years.
  • History of bilateral adrenalectomy or exogenous, pseudo, cyclic, or non-ACTH-dependent Cushing's syndrome (including certain inherited conditions).
  • High risk of acute morbidity from corticotroph adenoma growth (similar to that which occurs with Nelson's syndrome) defined as current evidence of macroadenoma at risk of impingement of vital structures.
  • Any current or prior medical condition, medical or surgical therapies, or clinical trial participation expected to interfere with the conduct of the study or the evaluation of its results, including but not limited to poor venous access or recent receipt or donation of blood products.
  • Women who are currently pregnant, lactating or planning fertility and unwilling to adhere to approved contraceptives or abstinence.

Treatment and study plan

SPI-62

Drug

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

Placebo

Drug

Inactive tablets identical to SPI-62 tablets

Primary outcomes

  1. Change from baseline in urinary HSD-1 ratio

    Time frame: Baseline to 6 weeks

    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.

Secondary outcomes

  1. Treatment emergent adverse events

    Time frame: Baseline through 24 weeks of treatment

    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

Sponsors and collaborators

Lead sponsor

Sparrow Pharmaceuticals

Industry

Registry information

Acronym: RESCUE

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Apr 1, 2022
Registry last updated
Oct 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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