AchondroplasiaPhase 3NCT06926491
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
A Phase 3 study testing KK8398 in people with Achondroplasia, run by Kyowa Kirin Co., Ltd. at 9 sites in Japan.
Trial summary
This trial will evaluate the efficacy of KK8398 on annualized height velocity after 52 weeks of repeated administration of KK8398 to patients with achondroplasia Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- KK8398
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 9 sitesJapan
- Study dates
- 1 Dec 2024to 31 May 2031
- Phase
- Phase 3
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients who are between 2.5 and 17.5 years old of age at the time of provisional registration. Patients who meet certain criteria in terms of Tanner stage and bone age.
- Patients who have been diagnosed with achondroplasia through genetic testing.
Who cannot join
- Patients who meet the criteria for a certain height determined by age and gender in the "Growth Chart for Patients with Achondroplasia" at the time of the pre-registration examination
- Patients who have received treatment with r-hGH or a CNP analogue within a certain period prior to the pre-enrollment examination.
- Patients who have had previous osteotomy or who are scheduled to undergo osteotomy or epiphyseal growth inhibition during the study period. Epiphyseal growth inhibition in which the plate was removed more than 26 weeks prior to the pre-registration examination will not be excluded if the patient has completely healed without sequelae, based on the judgment of the investigator or sub-investigator.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Japan9 sites
Tokyo Metropolitan Children's Medical Center
Fuchū, Tokyo
Niigata University Medical and Dental Hospital
Niigata
Aichi Children's Health and Medical Center
Ōbu, Aichi-ken
Okayama University Hospital
Okayama
ISEIKAI International General Hospital
Osaka
Osaka City General Hospital
Osaka
Osaka University Hospital
Suita, Osaka
Osaka Women's and Children's Hospital
Waizumi, Osaka
Tottori University Hospital
Yonago, Tottori
About this study
Official title: A Multicenter, Open-label, Single-arm, Phase 3 Study of KK8398 (Infigratinib) in Patients With Achondroplasia (AOBA Study)
This trial will evaluate the efficacy of KK8398 on annualized height velocity after 52 weeks of repeated administration of KK8398 to patients with achondroplasia
- Conditions
- Achondroplasia
- Being tested
- KK8398
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KK8398Drug
KK8398 will be administered.
| Study group | Role | What they receive |
|---|---|---|
| KK8398 | Experimental | Not described Receives: Drug: KK8398 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in annualized height velocity
Measured: Pre-dose, Week 52
Secondary outcomes (1)
Height Z-score using growth tables in patients with achondroplasia and the change from baseline
Measured: Pre-dose, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2025
- Study start1 Dec 2024
- Primary completion31 May 2029 (estimated)
- Study completion31 May 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 Kyowa Kirin Co., Ltd.The 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yu Sato, Study director, Kyowa Kirin Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06926491
- Sponsor's protocol ID
- 8398-001
- Other IDs
- jRCT2031240562
- Status last verified
- Oct 2026
- First submitted
- 1 Apr 2025
- Last updated
- 8 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There is not a plan to make IPD available.
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Single-arm, Phase 3 Study of KK8398 (Infigratinib) in Patients With Achondroplasia (AOBA Study)
- ClinicalTrials.gov ID
- NCT06926491
- Acronym / sponsor ID
- 8398-001
- Conditions
- Achondroplasia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Dec 2024 (actual)
- Primary completion
- 31 May 2029 (estimated)
- Study completion
- 31 May 2031 (estimated)
- First posted
- 13 Apr 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 trial will evaluate the efficacy of KK8398 on annualized height velocity after 52 weeks of repeated administration of KK8398 to patients with achondroplasia
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KK8398 | Experimental | Drug: KK8398 |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patients who are between 2.5 and 17.5 years old of age at the time of provisional registration. Patients who meet certain criteria in terms of Tanner stage and bone age.
- Patients who have been diagnosed with achondroplasia through genetic testing.
Key exclusion criteria (3 listed)
- Patients who meet the criteria for a certain height determined by age and gender in the "Growth Chart for Patients with Achondroplasia" at the time of the pre-registration examination
- Patients who have received treatment with r-hGH or a CNP analogue within a certain period prior to the pre-enrollment examination.
- Patients who have had previous osteotomy or who are scheduled to undergo osteotomy or epiphyseal growth inhibition during the study period. Epiphyseal growth inhibition in which the plate was removed more than 26 weeks prior to the pre-registration examination will not be excluded if the patient has completely healed without sequelae, based on the judgment of the investigator or sub-investigator.
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 |
|---|---|---|
| KK8398KK8398 will be administered. | Drug | KK8398 |
Primary outcome
Change from baseline in annualized height velocity
Time frame: Pre-dose, Week 52
1 secondary outcome
Height Z-score using growth tables in patients with achondroplasia and the change from baseline
Time frame: Pre-dose, Week 52
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 8 Oct 2026.
- Individual participant dataSharing planned: No. There is not a plan to make IPD available.
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
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Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
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A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
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A Study of TYRA-300 in Children With Achondroplasia: BEACH301
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Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
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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. This study is not recruiting at the moment.
Yu Sato study director
Kyowa Kirin Co., Ltd.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing KK8398 in people with Achondroplasia, run by Kyowa Kirin Co., Ltd. at 9 sites in Japan.
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 2 inclusion and 3 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: under way, not looking for participants, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Japan. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in annualized height velocity (time frame: Pre-dose, Week 52). 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 8 Oct 2026.