SPI-62 for Cushing's Syndrome

(RESCUE Trial)

Recruiting at 26 trial locations

Overseen By Frank Czerwiec, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sparrow Pharmaceuticals

Disqualifiers: Recent surgery, Radiation therapy, Adrenalectomy, Pregnancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 24 weeks

26 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

How does the drug SPI-62 differ from other treatments for Cushing's syndrome?

SPI-62 is unique because it targets the enzyme 11β-hydroxylase, which is crucial in the final step of cortisol production, potentially offering a new approach to managing Cushing's syndrome by directly reducing cortisol levels. ^{1 2 3 4 5}

Research Team

Frank Czerwiec, MD

Principal Investigator

Sparrow Pharmaceuticals ([e-mail])

Eligibility Criteria

This trial is for adults over 18 with ACTH-dependent Cushing's syndrome not caused by an adrenal tumor. Participants must have consistent cortisol excess and a confirmed diagnosis. They can't join if they've had recent Cushing's surgery, are at risk of tumor growth complications, have conditions that could affect the study, are pregnant or not using contraception, or have had certain past treatments for Cushing's.

Inclusion Criteria

You have ongoing high levels of cortisol in your body.

I am 18 years old or older.

I have been diagnosed with ACTH-dependent Cushing's syndrome.

Exclusion Criteria

I don't have any health issues that would affect this study's process or results.

I have had or will have surgery for Cushing's disease within 6 months.

I have a history of specific adrenal gland surgery or rare forms of Cushing's syndrome.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Baseline

Participants undergo baseline assessments before starting treatment

1 week

Treatment

Participants receive either SPI-62 or placebo for 24 weeks

24 weeks

Visits every 3 months, with remote options

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

Visits every 3 months

Treatment Details

Interventions

• Placebo
• SPI-62

Trial Overview The study tests SPI-62 against a placebo in patients with ACTH-dependent Cushing's syndrome. Each participant will receive both SPI-62 and a placebo for 24 weeks each, with an option to continue longer. The assignment to either SPI-62 or placebo is random.

Participant Groups

Group I: SPI-62 Experimental Treatment 2 Interventions

Active drug by mouth each morning for up to 12 weeks

Group II: Placebo Placebo Group 2 Interventions

Placebo by mouth each morning for up to 12 weeks

Who Is Running the Clinical Trial?

Sparrow Pharmaceuticals

Lead Sponsor

Trials

5

Recruited

160+

Findings from Research

In a proof-of-concept study involving 12 adult patients with moderate-to-severe Cushing's disease, LCI699 effectively normalized urinary free cortisol (UFC) levels or achieved a significant reduction (≥50%) from baseline after 10 weeks of treatment.

LCI699 was generally well tolerated, with most adverse events being mild or moderate, and no serious drug-related adverse events reported, indicating a favorable safety profile for this potential treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

LCI699, a potent 11β-hydroxylase inhibitor, normalizes urinary cortisol in patients with Cushing's disease: results from a multicenter, proof-of-concept study. Bertagna, X., Pivonello, R., Fleseriu, M., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

LCI699, a potent 11β-hydroxylase inhibitor, normalizes urinary cortisol in patients with Cushing's disease: results from a multicenter, proof-of-concept study. [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

Unrecognized adrenal insufficiency in patients undergoing laparoscopic adrenalectomy. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Use of ketoconazole in the treatment of Cushing's syndrome. [2015]

4. France pubmed.ncbi.nlm.nih.gov

[Short term effects of ketoconazole in Cushing's syndrome]. [2016]

5. United States pubmed.ncbi.nlm.nih.gov

Subclinical Cushing's syndrome. [2022]