Cushing's Syndrome IPhase 2RESCUENCT05307328
SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome
A Phase 2 study testing SPI-62 in people with Cushing's Syndrome I and Cushing Disease Due to Increased ACTH Secretion, run by Sparrow Pharmaceuticals at 12 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cushing's Syndrome I, Cushing Disease Due to Increased ACTH Secretion, Cortisol Excess
- What is being tested?
- SPI-62
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 12 sitesin 3 countries
- Study dates
- 1 Sep 2022to 1 Aug 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
12 sites in 3 countries.
12 sites in 3 countries
United States8 sites
Southwest General Healthcare Center
Fort Myers, Florida, 33907
Comprehensive and Interventional Pain Management Llp
Henderson, Nevada, 89052
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
St. Joseph's Hospital and Medical Center - Barrow Neurological Institute (BNI) - Pituitary Center
Phoenix, Arizona, 85013
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science University (OHSU) - Northwest Pituitary Center
Portland, Oregon, 97239
Mayo Clinic Cancer Center (MCCC) - Rochester
Rochester, Minnesota, 55905
Washington University School of Medicine - Center for Advanced Medicine (CAM)
St Louis, Missouri, 63110
Bulgaria3 sites
Medical University of Plovdiv
Plovdiv, 4002
Clinical Center of Endocrinology and Gerontology, University Hospital of Endocrinology, Medical University Sofia (USHATE)
Sofia, 1431
Medical University of Sofia
Sofia, 1431
Romania1 site
Carol Davila University of Medicine and Pharmacy
Bucharest, 050474
About this study
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.
Read the detailed description
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.
- Conditions
- Cushing's Syndrome I, Cushing Disease Due to Increased ACTH Secretion, Cortisol Excess, Cortisol; Hypersecretion, Cortisol Overproduction, Ectopic ACTH Secretion
- Being tested
- SPI-62, Placebo
- Keywords
- Cushing's Syndrome, ACTH Secretion, Cushing's Disease, Excessive Cortisol secretion, Cortisol, ectopic CRH secretion
- Medical subject headings
- ACTH Syndrome, Ectopic, Cushing Syndrome, Pituitary ACTH Hypersecretion
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPI-62Drug
11β hydroxysteroid dehydrogenase type 1 (HSD-1) inhibitor
PlaceboDrug
Inactive tablets identical to SPI-62 tablets
| Study group | Role | What they receive |
|---|---|---|
| SPI-62 | Experimental | Active drug by mouth each morning for up to 12 weeks Receives: Drug: SPI-62; Drug: Placebo |
| Placebo | Placebo comparator | Placebo by mouth each morning for up to 12 weeks Receives: Drug: SPI-62; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in urinary HSD-1 ratio
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.
Measured: Baseline to 6 weeks
Secondary outcomes (1)
Treatment emergent adverse events
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
Measured: Baseline through 24 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2022
- Study start1 Sep 2022
- Primary completion1 Aug 2025
- Study completion1 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cushing's Syndrome IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Staggered parallel crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sparrow Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Frank Czerwiec, MD, Study director, Sparrow Pharmaceuticals (info@sparrowpharma.com)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05307328
- Sponsor's protocol ID
- SPI-62-CL-2001
- Acronym
- RESCUE
- Status last verified
- May 2025
- First submitted
- 23 Mar 2022
- Last updated
- 8 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome
- ClinicalTrials.gov ID
- NCT05307328
- Acronym / sponsor ID
- RESCUE / SPI-62-CL-2001
- Conditions
- Cushing's Syndrome I, Cushing Disease Due to Increased ACTH Secretion, Cortisol Excess, Cortisol; Hypersecretion, Cortisol Overproduction, Ectopic ACTH Secretion
- Keywords
- Cushing's Syndrome, ACTH Secretion, Cushing's Disease, Excessive Cortisol secretion, Cortisol, ectopic CRH secretion
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Sparrow PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 1 Aug 2025 (actual)
- Study completion
- 1 Aug 2025 (actual)
- First posted
- 1 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Staggered parallel crossover
| Arm | Type | Receives |
|---|---|---|
| SPI-62 | Experimental | Drug: SPI-62, Drug: PlaceboActive drug by mouth each morning for up to 12 weeks |
| Placebo | Placebo comparator | Drug: SPI-62, Drug: PlaceboPlacebo by mouth each morning for up to 12 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPI-6211β hydroxysteroid dehydrogenase type 1 (HSD-1) inhibitor | Drug | Placebo, SPI-62 |
| PlaceboInactive tablets identical to SPI-62 tablets | Drug | Placebo, SPI-62 |
Primary outcome
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.
1 secondary outcome
Treatment emergent adverse events
Time frame: Baseline through 24 weeks of treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2025.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Frank Czerwiec, MD study director
Sparrow Pharmaceuticals (info@sparrowpharma.com)
12 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SPI-62 in people with Cushing's Syndrome I and Cushing Disease Due to Increased ACTH Secretion, run by Sparrow Pharmaceuticals at 12 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Bulgaria, Romania. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in urinary HSD-1 ratio (time frame: Baseline to 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2025.