AchondroplasiaPhase 1/2NCT07297875
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
A Phase 1/2 study testing ABSK061 in people with Achondroplasia, run by Abbisko Therapeutics Co, Ltd at 7 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- ABSK061
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 110 participants
- Where
- 7 sitesChina
- Study dates
- 10 Dec 2025to 15 Mar 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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.
Show all 10Show fewer
- 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).
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
China7 sites
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality
Di Wu, woody0928@sina.com
Di Wu
Chengdu Women's and Children's Central Hospital
Chengde, Sichuan
West China Second University Hospital, Sichuan University
Chengdu, Sichuan
Jin Wu, wangdo620@163.com
Jin Wu
Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang
Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality
Yongguo Yu, yuyongguo@xinhuamed.com.cn
Yongguo Yu, yuyongguo@shsmu.edu.cn
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei
Yanqin Ying, yingtea0909@126.com
Yanqin Ying
Henan Children's Hospital, Zhengzhou Children's Hospital
Zhengzhou, Henan
About this study
Official title: A Phase Ⅰ/Ⅱ, Open-Label Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
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.
Read the 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
- Being tested
- ABSK061
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABSK061Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| ABSK061 | Experimental | 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.
This study will employ the Bayesian optimal interval (BOIN) design for dose escalation part to guide dose escalation. The target toxicity rate for the MTD is φ = 0.25 and the maximum sample size for the dose escalation part will be 80 (50 in Part A and 30 in Part B). The DLT observation period is defined as the first 28 days after administration of ABSK061.
After the RDE is confirmed in the dose Escalation part, the dose Expansion phase will be conducted. Receives: Drug: ABSK061 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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)
Measured: 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)]
Measured: Day 1 of dosing to 78-week End of Treatment
Secondary outcomes (14)
Cmax
maximum observed concentration
Measured: Day 1 of dosing to 78-week End of Treatment
Tmax
time to maximum observed concentration
Measured: Day 1 of dosing to 78-week End of Treatment
AUC
area under the concentration-time curve
Measured: Day 1 of dosing to 78-week End of Treatment
t1/2
half-life
Measured: Day 1 of dosing to 78-week End of Treatment
Vz/F
apparent volume of distribution
Measured: Day 1 of dosing to 78-week End of Treatment
CL/F
apparent oral clearance
Measured: Day 1 of dosing to 78-week End of Treatment
Cmax,ss
maximum observed concentration of steady/state
Measured: Day 1 of dosing to 78-week End of Treatment
Cmin,ss
minimum observed concentration of steady/state
Measured: Day 1 of dosing to 78-week End of Treatment
AUCtau,ss
area under the concentration-time curve after multiple dose
Measured: Day 1 of dosing to 78-week End of Treatment
AR
accumulation ratio
Measured: Day 1 of dosing to 78-week End of Treatment
standing height
calculated to the nearest 0.1 cm
Measured: Day 1 of dosing to 78-week End of Treatment
sitting height
calculated to the nearest 0.1 cm
Measured: 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.
Measured: 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.
Measured: Day 1 of dosing to 78-week End of Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2025
- Study start10 Dec 2025 (estimated)
- Primary completion15 Dec 2028 (estimated)
- Study completion15 Mar 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- More trials run by Abbisko Therapeutics Co, LtdThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 110 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Abbisko Therapeutics Co, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shuqi Zeng, Study director, 12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07297875
- Sponsor's protocol ID
- ABSK061-202
- Status last verified
- Dec 2025
- First submitted
- 24 Nov 2025
- Last updated
- 22 Dec 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ⅰ/Ⅱ, Open-Label Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT07297875
- Acronym / sponsor ID
- ABSK061-202
- Conditions
- Achondroplasia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 110 participants (estimated)
- Lead sponsor
- Abbisko Therapeutics Co, LtdIndustry (pharma, biotech, devices)
- Study start
- 10 Dec 2025 (estimated)
- Primary completion
- 15 Dec 2028 (estimated)
- Study completion
- 15 Mar 2031 (estimated)
- First posted
- 22 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ABSK061 | Experimental | Drug: ABSK061Dose 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. This study will employ the Bayesian optimal interval (BOIN) design for dose escalation part to guide dose escalation. The target toxicity rate for the MTD is φ = 0.25 and the maximum sample size for the dose escalation part will be 80 (50 in Part A and 30 in Part B). The DLT observation period is defined as the first 28 days after administration of ABSK061. After the RDE is confirmed in the dose Escalation part, the dose Expansion phase will be conducted. |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (10 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABSK061ABSK061 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. | Drug | ABSK061 |
Primary outcome
Incidence of dose-limiting toxicities (DLTs)
Time frame: Day 1 to Day 28 of dosing
Incidence of dose-limiting toxicities (DLTs) in the DLT observation period, defined as Day 1 to Day 28 of dosing
Incidence and severity of adverse events (AEs)
Time frame: up to 87 weeks
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)
Changes from baseline in the Annualized Growth Velocity (AGV, cm/year)
Time frame: Day 1 of dosing to 78-week End of Treatment
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)]
14 secondary outcomes
Cmax
Time frame: Day 1 of dosing to 78-week End of Treatment
Tmax
Time frame: Day 1 of dosing to 78-week End of Treatment
AUC
Time frame: Day 1 of dosing to 78-week End of Treatment
t1/2
Time frame: Day 1 of dosing to 78-week End of Treatment
Vz/F
Time frame: Day 1 of dosing to 78-week End of Treatment
CL/F
Time frame: Day 1 of dosing to 78-week End of Treatment
Cmax,ss
Time frame: Day 1 of dosing to 78-week End of Treatment
Cmin,ss
Time frame: Day 1 of dosing to 78-week End of Treatment
AUCtau,ss
Time frame: Day 1 of dosing to 78-week End of Treatment
AR
Time frame: Day 1 of dosing to 78-week End of Treatment
standing height
Time frame: Day 1 of dosing to 78-week End of Treatment
sitting height
Time frame: Day 1 of dosing to 78-week End of Treatment
sitting height to standing height ratio
Time frame: Day 1 of dosing to 78-week End of Treatment
height Z scores
Time frame: Day 1 of dosing to 78-week End of Treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Achondroplasia
RecruitingPhase 2/3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Achondroplasia
Active, not recruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Achondroplasia
Active, not recruitingPhase 3
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Achondroplasia
RecruitingPhase 2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Achondroplasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Shuqi Zeng study director
12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ABSK061 in people with Achondroplasia, run by Abbisko Therapeutics Co, Ltd at 7 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jing Zhang (+86-15002126439, jing.zhang@abbisko.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose-limiting toxicities (DLTs) (time frame: Day 1 to Day 28 of dosing). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2025.