Takeaways:
- The Independent Data and Safety Monitoring Board approves Foresee Pharmaceuticals' Casppian phase 3 study (NCT05493709) for Leuprolide (CAMCEVI) in central precocious puberty (CPP), recommending trial continuation with a minor amended protocol.
- The open-label, multicenter trial is recruiting participants aged 2 to 9 years, aiming to evaluate the efficacy, safety, and pharmacokinetics of Leuprolide 42 mg-controlled release in CPP patients.
- Inclusion criteria for the trial include confirmed diagnosis of CPP within 12 months, pubertal-type LH response, clinical evidence of puberty, and willingness to participate.
- Foresee Pharmaceuticals anticipates releasing top-line results from the trial in 2025, emphasizing the importance of ensuring participant safety and ethical conduct throughout the study.
- CPP affects approximately 1 in 5000 to 10,000 children, with the potential for significantly short stature in adulthood and associated social, psychological, and emotional challenges.
The Independent Data and Safety Monitoring Board (DSMB) has given a positive safety review to Foresee Pharmaceuticals for the Casppian phase 3 registration study (NCTNCT05493709) evaluating efficacy, safety, and pharmacokinetics of Leuprolide (CAMCEVI; Foresee Pharmaceuticals) in patients with central precocious puberty (CPP).1,2
The Board recommended Foresee Pharmaceuticals continue the trial with a "minor amended protocol," according to a press release from Foresee.1
The trial is an open-label, multicenter, and multinational trial of the injectable emulsion. The trial and evaluation of Leuprolide 42 mg-controlled release in patients with CPP, as of March 21, 2024, is recruiting, according to ClinicalTrials.gov.1,2 The company announced the first patient was dosed in October, 2023.3
The single-arm study has an estimated enrollment of 93 participants, made up of females aged 2 to 8 years and males aged 2 to 9 years. Participants must have a confirmed diagnosis of CPP within 12 months of baseline visit but have not received prior gonadotropin-releasing hormone (GnRHa) treatment for CPP.2
Other inclusion criteria include2:
- Pubertal-type LH response at 60 minutes post GnRHa stimulation test before treatment initiation > 5 mIU/mL
- Clinical evidence of puberty, defined as Tanner stage ≥ 2 for breast development in females or testicular volume ≥ 4 mL in males.
- Willing and able to participate
- Difference between bone age and chronological age of 1 or more year
- Bone age less than 13 years for girls and less than 14 years for boys, among other informed consent requirements.
"We are pleased to receive the positive recommendation from the DSMB to continue our clinical trial," said Bassem Elmankabadi, MD, senior vice president, clinical development, Foresee Pharmaceuticals.1
"Ensuring the safety of the children participating in our trial is paramount and we continue to be dedicated to maintaining the highest standards of safety and ethical conduct throughout this clinical trial. The top-line results from this trial are anticipated in 2025," Elmankabadi said.1
Patients with CPP are at risk of having significantly short stature as adults, which could lead to social, psychological, and emotional issues such as stress, anxiety, lower self-esteem, and depression.1
CPP occurs in 1 out of 5000 to 10,000 children, and it is estimated that approximately 80% to 90% of CPP cases are idiopathic, "especially in females, with a female-to-male ratio of around 20," stated Foresee.1
References:
1. Foresee Pharmaceuticals receives positive recommendation from DSMB to continue the Casppian study, a phase 3 clinical trial of Leuprolide (FP-001) injectable emulsion for the treatment of central precocious puberty. Foresee Pharmaceuticals. Press release. April 1, 2024. Accessed April 3, 2024. https://www.prnewswire.com/news-releases/foresee-pharmaceuticals-receives-positive-recommendation-from-dsmb-to-continue-the-casppian-study-a-phase-3-clinical-trial-of-leuprolide-fp-001-injectable-emulsion-for-the-treatment-of-central-precocious-puberty-302104381.html
2. Efficacy, safety, and pharmacokinetics of Leuprolide Mesylate in subjects with central precocious puberty. ClinicalTrials.gov. Updated March 21, 2024. Accessed April 3, 2024. https://clinicaltrials.gov/study/NCT05493709
3. Foresee Pharmaceuticals announces first patient dosed in the Casppian, phase 3 clinical trial of Leuprolide (FP-001) injectable emulsion, 42 mg for the treatment of central precocious puberty patients. October 13, 2023. Accessed April 3, 2024. https://www.biospace.com/article/releases/foresee-pharmaceuticals-announces-first-patient-dosed-in-the-casppian-phase-3-clinical-trial-of-leuprolide-fp-001-injectable-emulsion-42-mg-for-the-treatment-of-central-precocious-puberty-patients-/
