NCT07297875

A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia

A Phase Ⅰ/Ⅱ, Open-Label Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 110 (estimated)
• Sponsor: Abbisko Therapeutics Co, Ltd · Industry
• Sex: All
• Age: 3 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, non-randomized, open-label, phase I/II study in children with ACH. This study will start with a dose escalation of ABSK061 in children with ACH to evaluate the safety, tolerability, PK, and efficacy. The RDE confirmation part will evaluate the safety and efficacy of ABSK061 at the recommended doses for expansion (RDEs) in children with ACH. All patients enrolled in the dose escalation part and RDE confirmation part can enter the extended treatment period to further evaluate the long-term safety, tolerability, and long-term efficacy of ABSK061 in children with ACH.

Detailed description

Up to 50 children aged 6 to \< 12 years (inclusive of 6 years) with ACH and up to 30 children aged 3 to \< 6 years (inclusive of 3 years) with ACH are expected to be enrolled in the dose escalation part of the study; up to 30 children aged 3 to \< 12 years (inclusive of 3 years) with ACH are expected to be enrolled in the RDE confirmation part. Children enrolled in this study will be required to complete at least 6 months and up to 2 years of growth assessment and observation of natural history of ACH in the sponsor's observational study (ABSK061-001). Dose Escalation Part All patients enrolled in the dose escalation part will continue to receive ABSK061 once daily (QD) dosing frequency and will enter the extended treatment period for 52 weeks after completing 26 weeks of treatment. The dose escalation includes a total of 5 potential dose levels starting at an initial dose of 0.064 mg/kg QD. In the absence of DLTs or ≥ grade 2 drug-related adverse events, the initial maximum permitted dose increment will not exceed 200%. In the presence of a DLT or ≥ grade 2 drug-related adverse event, dose escalation for subsequent cohorts will follow a modified Fibonacci scheme, in which the first two consecutive dose cohorts will each receive a maximum dose increase of up to 67% and 50%, respectively, and the remaining dose cohorts will receive a maximum of 33% increase. The potential dose levels to be explored are listed in the table below. Based on cumulative safety, PK/PD, efficacy data, other potential dose levels not listed may also be explored in this study following the dose escalation rules. Dose escalation part will be divided into Part A and Part B according to patient age, with Part A enrolling children with ACH aged 6 to \< 12 years (inclusive of 6 years) and Part B enrolling children with ACH aged 3 to \< 6 years (inclusive of 3 years). In this study, two sentinel patients will be set for both parts of each dose level, thereby the interval for the first dose between the first 2 patients and the third patient is not less than 28 days. RDE Confirmation Part In this part, up to 30 children aged 3 to \< 12 years (inclusive of age 3) with ACH will be enrolled to receive one or two RDEs. The RDEs will be selected from the safe doses determined in the dose escalation to further evaluate the safety and efficacy of ABSK061 in children with ACH. Patients in this part will not be included in the DLT evaluable population. Dose decisions for RDE Confirmation Part will be based on discussion and alignment between the sponsor and investigators. Safety, PK/PD, and efficacy data will be monitored and assessed in real time during the study. The study may be discontinued early if any safety risk to the patients are identified, upon agreement between the investigators and sponsor. Extended Treatment Period After completing 26 weeks of treatment with ABSK061 in the dose escalation part and RDE confirmation part, children with ACH will enter the extended treatment period for 52 weeks to further assess the long-term safety, tolerability and efficacy. In extended treatment period, patients will be treated at the dose assigned for dose escalation (intra-patient dose escalations are allowed for patients at low dose levels in Part A). Dose will be calculated based on body weight and may be adjusted due to weight change at the Week 27 (W27D1, i.e., just entering the extended treatment period), Week 41 (W41D1), Week 53 (W53D1), and Week 67 (W67D1) visits. After completing 78 weeks of study treatment set in the protocol, all patients in this study can be enrolled into a separate long-term extension study conducted by the sponsor to continue ABSK061 treatment. This is to further assess the long-term safety and efficacy of ABSK061 in children with ACH. If the investigator determines that a patient is no longer eligible to continue in the long-term extension study due to safety concerns or lack of clinical benefit, treatment with ABSK061 will be discontinued.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	ABSK061	ABSK061 is supplied as minitablets filled in capsules. Four strengths of capsules will be provided: 0.2 mg, 2 mg, 3 mg, and 5 mg. Each patient can only be administered with a single strength. All patients will be administered orally once daily under the fasted state, i.e., fast from 2 hours before dosing to 1 hour after dosing. Dose will be calculated based on the patient's weight, and detailed rules for dose calculation are provided. Take out the ABSK061 capsule containing minitablets, take off the capsule cap and decant the content (minitablets), swallow the minitablets completely with water without chewing (the recommended water volume is 50-180 mL). If there is difficulty swallowing all the minitablets at once, they may be taken in several portions as needed, but the entire dose should be taken within 10 minutes. If needed, yogurt or apple sauce can be used as vehicles to facilitate swallowing of the minitablets.

Timeline

• Start date: 2025-12-10
• Primary completion: 2028-12-15
• Completion: 2031-03-15
• First posted: 2025-12-22
• Last updated: 2025-12-22

Locations

7 sites across 1 country: China