SPI-62 for Cushing's Syndrome

(ACSPIRE Trial)

No longer recruiting at 85 trial locations

Overseen By Sarah Hooper

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sparrow Pharmaceuticals

Disqualifiers: Cushing's syndrome, Adrenalectomy, Thyroid disorders, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: 2

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, SPI-62, to determine its effectiveness for individuals with Cushing's Syndrome related to a benign adrenal tumor. Researchers aim to assess both the efficacy and safety of SPI-62 for this condition. Participants will receive either the actual drug or a placebo, which contains no active drug. Suitable candidates have a non-cancerous adrenal gland tumor and symptoms such as high blood sugar, high cholesterol, or high blood pressure. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to significant medical advancements.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop your current medications. However, it mentions that participants can be on stable standard treatments for conditions like diabetes, high cholesterol, and high blood pressure. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that SPI-62 is likely to be safe for humans?

Research has shown that SPI-62 is a promising treatment for conditions related to high cortisol levels, such as Cushing's syndrome. Studies indicate that SPI-62 can reduce cortisol levels in the liver by up to 90%. This is significant because excessive cortisol can lead to various health problems.

Earlier studies have examined the safety of SPI-62, suggesting that most people tolerate the treatment well. Although specific side effects were not mentioned, the progression to Phase 2 trials indicates that earlier tests did not identify major safety issues. In clinical trials, drugs undergo initial phases to assess safety before reaching Phase 2, which focuses more on efficacy while continuing to monitor safety.

Overall, SPI-62 appears to be a safe option based on current research. However, participants should discuss any concerns with their healthcare provider before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Cushing's Syndrome, which often involve surgery or medications that inhibit cortisol production, SPI-62 is unique because it targets 11β-hydroxysteroid dehydrogenase type 1 (11β-HSD1). This enzyme is involved in the conversion of inactive cortisone to active cortisol, and by inhibiting it, SPI-62 aims to reduce cortisol levels more directly and potentially with fewer side effects. Researchers are excited about SPI-62 because it offers a novel approach that could provide relief for patients who may not respond well to existing options or who experience significant side effects from current medications.

What evidence suggests that SPI-62 might be an effective treatment for Cushing's Syndrome?

Research has shown that SPI-62, which participants in this trial may receive, could be a promising treatment for Cushing's syndrome. It blocks a substance called HSD-1, helping to lower cortisol levels in cells. In studies, SPI-62 reduced cortisol in the liver by up to 90%. This reduction might help lessen the symptoms of Cushing's syndrome, which occurs when cortisol levels are too high. Evidence suggests that SPI-62 could effectively manage this condition. ^{1 2 3 4 5}

Who Is on the Research Team?

Frank Czerwiec, MD

Principal Investigator

Sparrow Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for adults with a benign adrenal tumor causing high cortisol levels, which may lead to conditions like diabetes or high blood pressure. Participants should have stable health conditions and not be pregnant, lactating, or have had recent major surgery. They shouldn't have other forms of Cushing's syndrome or certain cancers within the last 3 years.

Inclusion Criteria

I have high cholesterol, high triglycerides, weak bones, a history of fractures, or high blood pressure related to cortisol.

I have diabetes or pre-diabetes with specific blood sugar levels.

My adrenal nodule is non-cancerous, smaller than 4 cm, and has a uniform texture.

Exclusion Criteria

I tested positive for COVID-19 within the last 4 weeks or was hospitalized for it within the last 6 months.

My thyroid condition is not under control.

Pregnant or lactating

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive 2mg of SPI-62 daily to evaluate its effect on hypercortisolism

12 months

Visits at baseline, months 1, 3, 6, 9, 12, and then quarter-annually

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• SPI-62

Trial Overview The study tests SPI-62 at various doses to see if it can safely treat hypercortisolism due to a benign adrenal tumor. Each participant will take a daily dose of SPI-62 (2mg) and their response will be monitored for effectiveness and safety.

How Is the Trial Designed?

Group I: SPI-62 dose Experimental Treatment 1 Intervention

2mg dose level of SPI-62. Active drug by mouth.

Who Is Running the Clinical Trial?

Sparrow Pharmaceuticals

Lead Sponsor

Trials

5

Recruited

160+

Citations

1. sparrowpharma.com sparrowpharma.com/news/sparrow-presents-data-at-european-congress-of-endocrinology

Sparrow Pharmaceuticals Presents Data on SPI-62 at the ... SPI-62, a potent and selective HSD-1 inhibitor, has demonstrated the ability to reduce intracellular cortisol in the liver by 90%.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05307328?cond=Ectopic%20ACTH%20syndrome&rank=9

SPI-62 as a Treatment for Adrenocorticotropic Hormone ... This is a randomized, placebo-controlled, study of SPI-62 in subjects with ACTH-dependent Cushing's syndrome caused by a non-adrenal tumor.

3. biorxiv.org biorxiv.org/content/10.1101/2024.05.27.596076v1.full-text

The 11β-hydroxysteroid dehydrogenase type 1 inhibitor ... The results of this study support potential that SPI-62, or HSD-1 inhibitors in general, might alleviate certain symptoms of Cushing's disease ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05307328

Study Details | NCT05307328 | SPI-62 as a Treatment for ... 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 ...

5. mayo.edu mayo.edu/research/clinical-trials/cls-20537220

SPI-62 as a Treatment for Adrenocorticotropic Hormone ... SPI-62 has the potential to reduce symptoms associated with Cushing's syndrome by possibly reducing the amount of cortisol inside cells in different parts of ...