According to a story from BioSpace, the biopharmaceutical company Soleno Therapeutics recently announced that it will be updating the minimum age for participating patients in its Phase III clinical trial from eight years old to four years old. This trial is testing Diazoxide Choline Controlled-Release (DCCR) tablets as a treatment for Prader-Willi syndrome. Soleno Therapeutics focuses on the development of innovative therapies for the treatment of rare diseases.
About Prader-Willi Syndrome
Prader-Willi syndrome is a genetic disorder which is most characterized by an appetite that cannot be satisfied. This appears as the result of certain genes not functioning. It is usually caused by the deletion of a certain portion of the father’s chromosome 15. Risk factors are unknown. In newborn babies with the syndrome, symptoms include slow development, poor feeding, and muscle weakness. In childhood symptoms include constant hunger, obesity, type 2 diabetes, mild intellectual disability, behavioral abnormalities, pale skin, short height, small hands and feet, and a narrow forehead. Most patients are also infertile. Treatment can involve growth hormone therapy, restricted access to food, and an exercise program from an early age. There is no cure for Prader-Willi syndrome, and males and females are equally affected. To learn more about Prader-Willi syndrome, click here.
Treating Young Patients
The decision to lower the minimum age was primarily in an effort to more accurately represent the patient population with Prader-Willi syndrome. While the defining symptom of constant hunger is present in essentially all patients by age eight, this symptom can also appear years earlier, including by age four in some cases. The researchers that are managing this study are also confident that lowering the minimum age does not present a safety risk to younger patients. Molecules similar to DCCR have already been approved for treating other conditions in very young children, and in these indications the dosage is substantially higher.
This Phase III clinical trial involves about 100 patients with Prader-Willi syndrome. The trial was initiated in May of 2018 and should be finished by the first half of next year. DCCR has already received Fast Track designation and Orphan Drug designation for the treatment of Prader-Willi syndrome.