Ongoing Clinical Trials for Pituitary-dependent Cushing’s Syndrome
There are currently 2 clinical trials investigating new treatments for pituitary-dependent Cushing’s syndrome. These studies are testing medications that work to reduce excessive cortisol levels in the body, offering hope for patients who haven’t responded to surgery or are awaiting surgical treatment. The trials are taking place across several European countries including Belgium, Bulgaria, France, Italy, and Romania.
Clinical trial locations
- Belgium
- Bulgaria
- France
- Italy
- Romania
Study of SPI-62 for Treating ACTH-Dependent Cushing’s Syndrome in Patients with Cushing’s Disease or Ectopic ACTH/CRH Secretion
This trial is testing SPI-62, a new medication designed to help control excessive cortisol levels in adults with ACTH-dependent forms of the condition. The medication works by blocking an enzyme called 11β-hydroxysteroid dehydrogenase type 1, which plays a role in cortisol production.
Who can join: Adults aged 18 years or older with confirmed diagnosis showing active cortisol excess. Participants must have documented ACTH-dependent syndrome, which includes disease caused by pituitary overproduction of ACTH or ectopic ACTH secretion. Participants should also have related health issues such as high blood sugar, abnormal cholesterol levels, high blood pressure, or reduced bone density. Women must not be pregnant or breastfeeding, and all participants must agree to use contraception during the study.
Who cannot join: The trial excludes patients without confirmed diagnosis, those unable to follow study procedures or take the medication as required, pregnant or breastfeeding women, people currently in other clinical trials, those with a history of substance abuse, patients allergic to the study medication, and those who have had recent surgery or are planning surgery during the study period. Certain abnormal lab test results may also prevent participation.
What the trial involves: Participants will take SPI-62 as an oral tablet over a 12-week treatment period. The study will monitor hormone levels through urine and blood tests, with the primary assessment focusing on urinary measurements at week 6. Researchers will also track how the medication affects blood sugar levels, cholesterol, blood pressure, and bone health. Some participants may continue in a longer monitoring phase to gather additional safety and effectiveness data.
Study of Osilodrostat for Children and Adolescents with Cushing’s Disease
This trial is studying osilodrostat, a medication that reduces cortisol production, specifically in children and teenagers with the condition. Osilodrostat works by blocking an enzyme called 11β-hydroxylase that is involved in making cortisol in the body.
Who can join: Children and adolescents aged 6 to under 18 years with confirmed diagnosis who weigh more than 30 kilograms. The trial is designed for young patients who have tried surgery that didn’t work, are waiting for surgery, or for whom surgery is not currently an option. Participants must be able to swallow tablets whole without crushing or breaking them, and parents or legal guardians must provide permission for their child to participate.
What the trial involves: Participants will take osilodrostat orally in tablet form, available in 1 mg, 5 mg, and 10 mg doses. The study team will determine the appropriate dosage based on individual needs. The trial will last up to 48 weeks, during which researchers will regularly monitor how the body processes the medication and assess its effects on cortisol levels. Safety assessments will be conducted throughout to watch for any side effects or adverse reactions.
Study goal: The trial aims to understand how osilodrostat works in younger patients, determine if it’s well-tolerated, and assess whether it can effectively manage the condition in children and adolescents, particularly those for whom surgery has not been successful or is not yet available.
Summary
These two clinical trials represent important research efforts to develop better treatment options for pituitary-dependent Cushing’s syndrome. Both studies focus on medications that work by targeting different enzymes involved in cortisol production. The SPI-62 trial is aimed at adults and takes place in Bulgaria and Romania, while the osilodrostat trial specifically addresses the needs of pediatric patients across Belgium, France, and Italy.
Notably, the pediatric trial fills an important gap in treatment options for children and teenagers who haven’t responded well to surgery or are awaiting surgical intervention. Both medications offer oral administration, which may be more convenient than some existing treatment approaches. The trials will provide valuable data on safety, effectiveness, and how these medications affect various health aspects related to excessive cortisol, including blood sugar control, blood pressure, cholesterol levels, and bone health.
