A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children Wit… (NCT07297875) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
China110 participantsStarted 2025-12-10
Plain-language 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.
Who can participate
Age range3 Years – 12 Years
SexALL
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Inclusion criteria
✓. Prior to screening, the guardians and children with ACH (if applicable) must voluntarily provide signed informed consent.
✓. Patients with a clear clinical diagnosis of ACH confirmed by genetic testing for an FGFR3 mutation.
✓. Male or female, age at screening:
✓. Have completed at least 6 months (i.e., the "Day 181" visit) of growth assessment and observation of natural history of ACH in the observational study (ABSK061-001) before study entry.
✓. Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening
Exclusion criteria
✕. Known allergy or hypersensitivity to any component of the study drug.
✕. Bone age ≥ 14 years as assessed by the investigator based on hand and wrist X-ray.
✕. Have a form of skeletal dysplasia other than ACH or known medical conditions that result in short stature or abnormal growth, including but not limited to severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, chronic renal insufficiency, active celiac disease a, Vitamin D deficiency b, untreated hypothyroidism c, poorly controlled diabetes (HbA1c ≥8.0%) or diabetic complications
What they're measuring
1
Incidence of dose-limiting toxicities (DLTs)
Timeframe: Day 1 to Day 28 of dosing
2
Incidence and severity of adverse events (AEs)
Timeframe: up to 87 weeks
3
Changes from baseline in the Annualized Growth Velocity (AGV, cm/year)
Timeframe: Day 1 of dosing to 78-week End of Treatment
✕. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
✕. AGV ≤ 1.5 cm/year over at least 6 months (i.e., must have completed the 'Day 181' visit) in the observational study (ABSK061-001), or current evidence of growth plate closure (proximal tibia, distal femur).
✕. Current epiphyseal injury (Salter-Harris fracture) or severe hip pain.
✕. For ACH-related complications: current severe sleep apnea, symptomatic and/or requiring intervention for hydrocephalus, or spinal cord compression at the cranio-cervical junction, or prior ventriculoperitoneal shunt surgery.
✕. Have received any dose of medications affecting stature or body proportionality, such as human growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids within 12 months prior to screening.