AchondroplasiaPhase 2ACHNCT02724228
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
A Phase 2 study testing BMN 111 in people with Achondroplasia, run by BioMarin Pharmaceutical at 9 sites in 4 countries.
Trial summary
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of… Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Achondroplasia
- What is being tested?
- BMN 111
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 26 Jan 2016to Feb 2028
- Phase
- Phase 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 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Have completed 24 months of BMN 111 treatment in Study 111-202.
- Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
- If sexually active, willing to use a highly effective method of contraception while participating in the study.
- Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
- Willing and able to perform all study procedures as physically possible
- Parents/caregivers willing to administer daily injections to the subjects and complete the required training.
Who cannot join
- Requires any investigational agent prior to completion of study period.
- Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
- Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
- Permanently discontinued BMN 111 during the 111-202 study.
- Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study.
- Current chronic therapy with restricted medications.
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
United States6 sites
Johns Hopkins McKusick - Institute of Genetic Medicine
Baltimore, Maryland, 21287
Ann and Robert H. Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611
Baylor College of Medicine
Houston, Texas, 77030
Vanderbilt University
Nashville, Tennessee, 37232-2578
Children's Hospital & Research Center Oakland
Oakland, California, 94609
Harbor - UCLA Medical Center
Torrance, California, 90509
Australia1 site
Murdoch Children's Research Institute
Parkville, Victoria, 3052
United Kingdom1 site
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
London, SE1 9RT
About this study
Official title: A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
- Conditions
- Achondroplasia
- Being tested
- BMN 111
- Keywords
- Achondroplasia, Dwarfism, Bone Diseases, Developmental, Bone Diseases
- Medical subject headings
- Achondroplasia, Dwarfism, Bone Diseases, Developmental, Bone Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN 111Drug
Also called Modified recombinant human C-type natriuretic peptide, Vosoritide
BMN 111 will be administered subcutaneously daily.
| Study group | Role | What they receive |
|---|---|---|
| BMN 111 - Subcutaneous Injection | Experimental | 111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen. Receives: Drug: BMN 111 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
- Number of study participants with treatment-emergent adverse events.
- Number of study participants with treatment-emergent serious adverse events
Measured: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Secondary outcomes (4)
Growth Velocity [Efficacy]
Annualized growth velocity (cm/yr)
Measured: Until near final adult height is reached
Growth Parameters (Efficacy)
Height standard score (Z-score)
Measured: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Body Proportions (Efficacy)
Upper-to-lower body segment ratio
Measured: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Final Adult Height
Height at the age of 16 years for females and 18 years for males
Measured: Up to at least 16 years of age for females and 18 years of age for males
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2016 (estimated)
- Study start26 Jan 2016
- Primary completionDec 2027 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02724228
- Sponsor's protocol ID
- 111-205
- Other IDs
- 2015-004004-30, 2015
- Acronym
- ACH
- Status last verified
- Mar 2026
- First submitted
- 12 Feb 2016
- Last updated
- 13 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23.
PubMed 38649657Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.
PubMed 31269546
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT02724228
- Acronym / sponsor ID
- ACH / 111-205
- Conditions
- Achondroplasia
- Keywords
- Achondroplasia, Dwarfism, Bone Diseases, Developmental, Bone Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 26 Jan 2016 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 31 Mar 2016 (estimated)
- 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 Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BMN 111 - Subcutaneous Injection | Experimental | Drug: BMN 111111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen. |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Have completed 24 months of BMN 111 treatment in Study 111-202.
- Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
- If sexually active, willing to use a highly effective method of contraception while participating in the study.
- Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
- Willing and able to perform all study procedures as physically possible
Key exclusion criteria (6 listed)
- Requires any investigational agent prior to completion of study period.
- Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
- Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
- Permanently discontinued BMN 111 during the 111-202 study.
- Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study.
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 |
|---|---|---|
| BMN 111Also: Modified recombinant human C-type natriuretic peptide, VosoritideBMN 111 will be administered subcutaneously daily. | Drug | BMN 111 - Subcutaneous Injection |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events
4 secondary outcomes
Growth Velocity [Efficacy]
Time frame: Until near final adult height is reached
Growth Parameters (Efficacy)
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Body Proportions (Efficacy)
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Final Adult Height
Time frame: Up to at least 16 years of age for females and 18 years of age for males
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 13 Mar 2026.
- Derived from this study: PubMed 38649657Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23.
- Derived from this study: PubMed 31269546Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.
- Individual participant dataSharing planned: No
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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.
Medical Director, MD study director
BioMarin Pharmaceutical
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BMN 111 in people with Achondroplasia, run by BioMarin Pharmaceutical at 9 sites in 4 countries.
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 6 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 13 Mar 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 United States, Australia, France, United Kingdom. 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 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?
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?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02724228, which also lists 2 linked publications.
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 13 Mar 2026.