AchondroplasiaNCT07301463
A Study in Children With Achondroplasia
An observational study of Achondroplasia, run by Abbisko Therapeutics Co, Ltd at 8 sites in China.
Trial summary
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural history observation of ACH for at least 6 months and up to 2 years. Read more about this study
- Who is it for?
- Ages 30 months to 11 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- complete a natural history observation of ACH for at least 6 months and up to 2 years
- How many people
- 260 participants
- Where
- 8 sitesChina
- Study dates
- 20 Jun 2025to 30 Apr 2039
- Phase
- Observational
- 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 30 months to 11 years
- All sexes
- No healthy volunteers
- Child
Study population: Children with Achondroplasia
Who may be able to join
- Prior to screening, the guardians and children with ACH (if applicable) must be willing and able to provide signed informed consent.
- Clinical diagnosis of ACH confirmed FGFR3 mutation by genetic testing.
- Male or female aged ≥2.5 to <11 years old at screening.
- Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening.
- Ambulatory and able to stand without assistance.
Who cannot join
- Bone age ≥14 years as assessed by the investigator based on hand and wrist X-ray taken within 6 months prior to Day 1.
- Current evidence of growth plate closure (proximal tibia, distal femur), or AGV ≤ 1.5 cm/year over a period ≥6 months prior to screening.
- 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 d.
- Celiac disease responsive to a gluten-free diet is allowed
- Vitamin D deficiency or insufficiency with a 25-hydroxyvitamin D [25- (OH) D] level ≥ 30 nmol/L after supplementation is allowed. Vitamin D deficiency is defined as 25-(OH) D level <30 nmol/L. Vitamin D insufficiency is defined as 25-(OH) D level 30~50 nmol/L. Patients with Vitamin D deficiency or insufficiency must be on Vitamin D regimen prior to screening
- Patients with hypothyroidism meeting the following criteria are allowed to enroll: must be clinically euthyroid for one month prior to screening and, in the opinion of the investigator, have achieved any catch-up growth expected from thyroxine replacement
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- Patients with diabetes must have been on stable medication regimen for 3 months prior to screening
- History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
- Impaired cardiac function or clinically significant cardiovascular disease, including any one of the following: New York Heart Association class II or higher heart disease, congenital heart disease (patients with repaired uncomplicated patent ductus arteriosus or atrial/ventricular septal defect with repair are allowed), clinically significant arrhythmias requiring therapy, aortic regurgitation, congestive heart failure, or any other uncontrolled heart disease.
- For ACH-related complications: Current severe sleep apnea, symptomatic and/or requiring intervention for hydrocephalus, or spinal cord compression at the cranio-cervical junction, and has previously undergone ventriculoperitoneal shunt surgery.
- Bone fracture within 6 months prior to screening (within 2 months for finger and toe fractures).
- 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 3 months prior to screening, or long-term treatment (>3 months) with the above drugs at any time.
- Prior treatment with any CNP analogues or FGFR inhibitors. Prior use of any investigational drugs or investigational medical devices that affect stature or body proportionality.
- Any comorbidities, disease or condition that, in the opinion of the investigator, may make the patient unlikely to fully complete the study-related procedures, may affect protocol compliance.
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
China8 sites, 7 recruiting
- Recruiting
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality
Di Wu, woody0928@sina.com
Di Wu
- Not yet recruiting
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan
Xinran Cheng
Xinran Cheng
- Active, not recruiting
West China Second University Hospital, Sichuan University
Chengdu, Sichuan
- Not yet recruiting
Guangzhou Women and Childrens Medical Center
Guangzhou, Guangzhou
Li Liu, liliuchina@qq.com
Li Liu
- Not yet recruiting
Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang
Wei Wu, wuwei120@zju.edu.cn
Wei Wu
- Recruiting
Xin Hua Hospital Affiliatod to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality
Yongguo Yu, yuyongguo@xinhuamed.com.cn
Yongguo Yu
- Recruiting
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei
Yanqin Ying
Yanqin Ying
- Not yet recruiting
Henan Children's Hospital, Zhengzhou Children's Hospital
Zhengzhou, Henan
Haiyan Wei
Haiyan Wei
About this study
Official title: A Multicenter, Longitudinal, Observational Study in Children With Achondroplasia
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural history observation of ACH for at least 6 months and up to 2 years.
Read the detailed description
Primary Objectives To evaluate the safety, tolerability, and recommended dose for expansion (RDE) of oral ABSK061 in children with ACH To evaluate the efficacy of oral ABSK061 in children with ACH
Secondary Objectives To characterize the pharmacokinetics (PK) of ABSK061 and potential disproportional metabolites (if applicable) To evaluate changes from baseline in anthropometric parameters after administration of oral ABSK061 To evaluate the acceptability of ABSK061 minitablets for peroral administration in children with ACH
Exploratory Objectives To evaluate changes in ACH complications and disease burden after oral administration of ABSK061 To evaluate the pharmacodynamic (PD) profile in children with ACH after oral administration of ABSK061
- Conditions
- Achondroplasia
- Being tested
- No Interventions, complete a natural history observation of ACH for at least 6 months and up to 2 years
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionsOther
No Interventions
complete a natural history observation of ACH for at least 6 months and up to 2 yearsOther
no interventions
| Study group | Role | What they receive |
|---|---|---|
| Children with Achondroplasia | Male or female aged ≥2.5 to <11 years old at screening Receives: Other: No Interventions; Other: complete a natural history observation of ACH for at least 6 months and up to 2 years |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized growth velocity (AGV)
Annualized growth velocity (AGV)
Measured: Through the study completion, an average of three months, up to 2 years
Secondary outcomes (3)
standing height
calculated to the nearest 0.1 cm
Measured: an average of three months, up to 2 years
sitting height
calculated to the nearest 0.1 cm
Measured: an average of three months, up to 2 years
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: an average of three months, up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2025
- Study start20 Jun 2025
- Primary completion30 Dec 2028 (estimated)
- Study completion30 Apr 2039 (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.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 260 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Abbisko Therapeutics Co, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yuan Lu, Study director, 12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07301463
- Sponsor's protocol ID
- ABSK061-001
- Status last verified
- Nov 2025
- First submitted
- 18 Nov 2025
- Last updated
- 24 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Longitudinal, Observational Study in Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT07301463
- Acronym / sponsor ID
- ABSK061-001
- Conditions
- Achondroplasia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 260 participants (estimated)
- Lead sponsor
- Abbisko Therapeutics Co, LtdIndustry (pharma, biotech, devices)
- Study start
- 20 Jun 2025 (actual)
- Primary completion
- 30 Dec 2028 (estimated)
- Study completion
- 30 Apr 2039 (estimated)
- First posted
- 24 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.
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural history observation of ACH for at least 6 months and up to 2 years.
Detailed description
Primary Objectives To evaluate the safety, tolerability, and recommended dose for expansion (RDE) of oral ABSK061 in children with ACH To evaluate the efficacy of oral ABSK061 in children with ACH
Secondary Objectives To characterize the pharmacokinetics (PK) of ABSK061 and potential disproportional metabolites (if applicable) To evaluate changes from baseline in anthropometric parameters after administration of oral ABSK061 To evaluate the acceptability of ABSK061 minitablets for peroral administration in children with ACH
Exploratory Objectives To evaluate changes in ACH complications and disease burden after oral administration of ABSK061 To evaluate the pharmacodynamic (PD) profile in children with ACH after oral administration of ABSK061
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Children with Achondroplasia | – | Other: No Interventions, Other: complete a natural history observation of ACH for at least 6 months and up to 2 yearsMale or female aged ≥2.5 to <11 years old at screening |
Who may qualify
- Ages
- 30 months to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with Achondroplasia
Key inclusion criteria (5 listed)
- Prior to screening, the guardians and children with ACH (if applicable) must be willing and able to provide signed informed consent.
- Clinical diagnosis of ACH confirmed FGFR3 mutation by genetic testing.
- Male or female aged ≥2.5 to <11 years old at screening.
- Tanner Stage 1 breast development for females or Tanner Stage 1 external genitalia development for males at screening.
- Ambulatory and able to stand without assistance.
Key exclusion criteria (14 listed)
- Bone age ≥14 years as assessed by the investigator based on hand and wrist X-ray taken within 6 months prior to Day 1.
- Current evidence of growth plate closure (proximal tibia, distal femur), or AGV ≤ 1.5 cm/year over a period ≥6 months prior to screening.
- 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 d.
- History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
- Impaired cardiac function or clinically significant cardiovascular disease, including any one of the following: New York Heart Association class II or higher heart disease, congenital heart disease (patients with repaired uncomplicated patent ductus arteriosus or atrial/ventricular septal defect with repair are allowed), clinically significant arrhythmias requiring therapy, aortic regurgitation, congestive heart failure, or any other uncontrolled heart disease.
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 |
|---|---|---|
| No InterventionsNo Interventions | Other | Children with Achondroplasia |
| complete a natural history observation of ACH for at least 6 months and up to 2 yearsno interventions | Other | Children with Achondroplasia |
Primary outcome
Annualized growth velocity (AGV)
Time frame: Through the study completion, an average of three months, up to 2 years
Annualized growth velocity (AGV)
3 secondary outcomes
standing height
Time frame: an average of three months, up to 2 years
sitting height
Time frame: an average of three months, up to 2 years
sitting height to standing height ratio
Time frame: an average of three months, up to 2 years
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 24 Dec 2025.
- Individual participant dataSharing planned: Undecided
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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.
Yuan Lu study director
12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia, run by Abbisko Therapeutics Co, Ltd at 8 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 5 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 24 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 8 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.
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?
Annualized growth velocity (AGV) (time frame: Through the study completion, an average of three months, up to 2 years). 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 24 Dec 2025.