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临床试验 NCT07297875 针对软骨发育不全目前尚未招募。请查看临床试验雷达卡片视图和 AI 发现工具了解所有详情，或在此提出任何问题。 | ||
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A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia I期, II期 110 随机化 剂量递增 开放性试验
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临床试验NCT07297875旨在研究治疗，主要针对软骨发育不全。这是一项I期 II期 干预性研究试验，当前状态为尚未招募。试验尚未开始，计划于2025年12月10日开始，预计招募110名患者。该研究由Abbisko Therapeutics Co, Ltd主导，计划于2031年3月15日完成。试验数据来源于ClinicalTrials.gov，最后更新时间为2025年12月22日。
简要概括
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 ...显示更多
详细描述
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 ...
显示更多官方标题
A Phase Ⅰ/Ⅱ, Open-Label Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
疾病
软骨发育不全其他研究标识符
- ABSK061-202
NCT编号
实际开始日期
2025-12-10
最近更新发布
2025-12-22
预计完成日期
2031-03-15
计划入组人数
110
研究类型
干预性研究
试验分期 (阶段)
I期
II期
II期
试验状态
尚未招募
主要目的
治疗方法
分配方式
不适用
干预模型
单组试验
盲法
无（开放性试验）
试验组/干预措施
| 参与者组/试验组 | 干预措施/治疗方法 |
|---|---|
实验性ABSK061 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 thir...显示更多 | 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 calcul...显示更多 |
主要终点
次要终点
| 结果指标 | 度量标准描述 | 时间框架 |
|---|---|---|
Incidence of dose-limiting toxicities (DLTs) | Incidence of dose-limiting toxicities (DLTs) in the DLT observation period, defined as Day 1 to Day 28 of dosing | Day 1 to Day 28 of dosing |
Incidence and severity of adverse events (AEs) | Incidence and severity of adverse events (AEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) using Common Terminology Criteria for Adverse Events, version 5.0 (CTCAE v5.0), and relatedness, rate of dose modifications (including dose interruption and dose reduction) or discontinuation of study drug due to toxicity, and changes from baseline in safety assessments such as laboratory parameters, x-ray, electrocardiograms (ECGs), echocardiograms, vital signs, and physical examinations (including ophthalmic examinations) | up to 87 weeks |
Changes from baseline in the Annualized Growth Velocity (AGV, cm/year) | Changes from baseline in the Annualized Growth Velocity (AGV, cm/year) over 52 weeks \[baseline is defined as the AGV obtained through at least 6 months of observation in the observational study (ABSK061-001)\] | Day 1 of dosing to 78-week End of Treatment |
| 结果指标 | 度量标准描述 | 时间框架 |
|---|---|---|
Cmax | maximum observed concentration | Day 1 of dosing to 78-week End of Treatment |
Tmax | time to maximum observed concentration | Day 1 of dosing to 78-week End of Treatment |
AUC | area under the concentration-time curve | Day 1 of dosing to 78-week End of Treatment |
t1/2 | half-life | Day 1 of dosing to 78-week End of Treatment |
Vz/F | apparent volume of distribution | Day 1 of dosing to 78-week End of Treatment |
CL/F | apparent oral clearance | Day 1 of dosing to 78-week End of Treatment |
Cmax,ss | maximum observed concentration of steady/state | Day 1 of dosing to 78-week End of Treatment |
Cmin,ss | minimum observed concentration of steady/state | Day 1 of dosing to 78-week End of Treatment |
AUCtau,ss | area under the concentration-time curve after multiple dose | Day 1 of dosing to 78-week End of Treatment |
AR | accumulation ratio | Day 1 of dosing to 78-week End of Treatment |
standing height | calculated to the nearest 0.1 cm | Day 1 of dosing to 78-week End of Treatment |
sitting height | calculated to the nearest 0.1 cm | Day 1 of dosing to 78-week End of Treatment |
sitting height to standing height ratio | This parameter is calculated as the ratio of sitting height to total standing height. It is used to assess the abnormality in body proportions, specifically the relative trunk-to-lower limb length. In patients with achondroplasia, this ratio is typically increased. Within the clinical trial, this measure is used to evaluate the treatment drug's effect on body proportions. | Day 1 of dosing to 78-week End of Treatment |
height Z scores | This measure is defined as the number of standard deviation units by which the subject's height differs from the mean height of a healthy, age- and sex-matched reference population. It is a standardized value used to precisely quantify the degree of deviation from the normal growth curve. In the clinical trial, it serves as a key parameter for assessing the treatment's efficacy in improving linear height growth. | Day 1 of dosing to 78-week End of Treatment |
参与助手
资格标准
适龄参与研究
儿童
最低年龄要求
3 Years
适龄性别
全部
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:
Dose Escalation Part A: 6 to < 12 years (inclusive 6 years) Dose Escalation Part B: 3 to < 6 years (inclusive 3 years) RDE Confirmation Part: 3 to < 12 years (inclusive 3 years).
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
- 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
- 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.
- Prior treatment with any CNP analogues or FGFR inhibitors. Prior use of any investigational drugs or investigational medical devices that affect height or body proportion.
- History of any prior bone-related surgery that affects long bone growth, such as orthopaedic reconstructive surgery, limb lengthening, or osteotomy (patients who have previously undergone foramen magnum decompression or intervertebral disc/laminectomy are allowed if they have fully recovered after surgery and bone healing has occurred for at least 6 months. Patients who have previously undergone eight-plate epiphysiodesis are allowed if the plate has been removed and healed for at least 4 weeks).
Abbisko Therapeutics Co, Ltd研究中心联系人
联系人: Jing Zhang, +86-15002126439, [email protected]
7 位于 1 个国家/地区的研究中心
Beijing Municipality
Beijing Children's Hospital, Capital Medical University, Beijing, Beijing Municipality, China
Di Wu, 联系人, [email protected]
Di Wu, 主要研究者
Henan
Henan Children's Hospital, Zhengzhou Children's Hospital, Zhengzhou, Henan, China
Hubei
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China
Yanqin Ying, 联系人, [email protected]
Yanqin Ying, 主要研究者
Xiuhua Ren, 主要研究者
Shanghai Municipality
Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, Shanghai Municipality, China
Yongguo Yu, 联系人, [email protected]
Yongguo Yu, 联系人, [email protected]
Yongguo Yu, 主要研究者
Sichuan
Chengdu Women's and Children's Central Hospital, Chengde, Sichuan, China
West China Second University Hospital, Sichuan University, Chengdu, Sichuan, China
Jin Wu, 联系人, [email protected]
Jin Wu, 主要研究者
Zhejiang
Children's Hospital Zhejiang University School of Medicine, Hangzhou, Zhejiang, China