AchondroplasiaPhase 2ACHNCT02055157
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
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, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia. Read more about this study
- Who is it for?
- Ages 5 to 14 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- BMN 111
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 13 Jan 2014to 2 Oct 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parent(s) or guardian(s) are willing and able to provide written, signed informed consent
- 5 to 14 years old at end of study
- ACH, documented by clinical grounds, confirmed by genetic testing
- At least 6-month of pretreatment growth assessment in Study 111-901 before study entry, and one standing height at least 6 months prior to screening for 111-202
- Negative pregnancy test at the Screening Visit for females ≥ 10 years old or who have begun menses
- If sexually active, willing to use a highly effective method of contraception while participating in the study
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- Ambulatory, able to stand without assistance
- Willing and able to perform all study procedures as physically possible
- Parents/caregivers willing to administer daily injections to the subjects
- Additional inclusion Criteria Optional, Open-label Extension Phase:
- Appropriate written informed consent
Who cannot join
- Hypochondroplasia or short stature condition other than ACH
- Have any of the following:
- Hypothyroidism or hyperthyroidism
- Insulin-requiring diabetes mellitus
- Autoimmune inflammatory disease
- Inflammatory bowel disease
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- Autonomic neuropathy
- Recent acute illness associated with volume dehydration not completely resolved prior to the first dose of study drug
- Unstable condition requiring surgical intervention during the study
- Growth plates have fused
- Have a history of any of the following:
- Renal insufficiency, defined as creatinine > 2 mg/dl
- Anemia
- Baseline systolic BP < 75 mm Hg or recurrent symptomatic hypotension or recurrent symptomatic hypotension, recurrent symptomatic orthostatic hypotension
- Cardiac or vascular disease, including the following:
- Cardiac dysfunction (abnormal echocardiogram [ECHO] including left ventricle [LV] mass) at Screening Visit
- Hypertrophic cardiomyopathy
- Pulmonary Hypertension
- Congenital heart disease with ongoing cardiac dysfunction
- Cerebrovascular disease
- Aortic insufficiency
- Clinically significant atrial or ventricular arrhythmias
- Have an ECG showing any of the following:
- Right or left atrial enlargement or ventricular hypertrophy
- PR (period of time from the beginning of atrial depolarization until the beginning of ventricular depolarization) interval > 200 msec
- QRS (The Q, R, and S heart waves that are measured on an electrocardiogram) interval > 110 msec
- Corrected QTc-F (Measure of the corrected time between the start of the Q wave and end of the T wave in the heart's electrical cycle) > 450 msec
- Second- or third-degree atrioventricular block
- Documented Vitamin D deficiency
- Require any investigational agent prior to completion of study period
- Have received another investigational product or investigational medical device within 30 days before the Screening visit
- Use of any other investigational product or investigational medical device for the treatment of ACH or short stature
- Current chronic therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
- Treatment with growth hormone, IGF-1 (Insulin-like growth factor), or anabolic steroids in the previous 6 months or long-term treatment (> 3 months) at any time
- Long-term treatment (> 1 month) with oral corticosteroids
- Concomitant medication that prolongs the QT/QTc-F interval within 14 days or 5 half-lives, whichever is longer, before the Screening visit
- Pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study
- Limb-lengthening or bone-related surgery < 18 months prior to study enrollment
- Had a fracture of the long bones or spine within 6 months prior to screening (except for fracture of digits or toes)
- AST (Aspartate Transaminase) or ALT (Alanine Transaminase) at least 3x upper limit of normal (ULN) or total bilirubin at least 2x ULN
- Evidence of severe sleep apnea requiring surgery or new initiation of CPAP (Continuous positive airway pressure).
- History of malignancy and chemotherapy/radiation or currently under work-up for suspected malignancy
- Known hypersensitivity to BMN 111 or its excipients
- 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 would interfere with study participation or safety
- Have abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant as determined by the PI.
- Have a history of hip surgery or severe hip dysplasia
- Have a history of clinically significant hip injury in the 30 days prior to screening.
- History of slipped capital femoral epiphysis or avascular necrosis of the femoral head.
- Are unable to lie flat when in prone position
- Additional Exclusion Criteria for Optional, Open-label Extension Phase:
- Use of restricted therapies during the initial 6 months of the study
- Permanently discontinued BMN 111 during the initial 6 months of the study
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, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia
This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.
- 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 daily for 24 months in an open-label sequential dose adjustment fashion.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Cohort 1: 2.5 ug/kg Receives: Drug: BMN 111 |
| Cohort 2 | Experimental | Cohort 2: 7.5 ug/kg, Receives: Drug: BMN 111 |
| Cohort 3 | Experimental | Cohort 3: 15 ug/Kg Receives: Drug: BMN 111 |
| Cohort 4 | Experimental | Cohort 4: 30 ug/kg Receives: Drug: BMN 111 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Summary of Adverse Events During Initial 6-Month Period
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration.
Serious adverse event (SAE).
Measured: Up to Month 6 ± 7 Days
Overall Summary of Adverse Events During Entire Study Period
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration.
TEAE - Treatment-emergent adverse event. SAE - Serious adverse event.
Measured: Up to Month 25 ± 7 Days
Secondary outcomes (21)
Change From Baseline in Annualized Growth Velocity (AGV) During Initial 6-Month
Annualized Growth Velocity at Day 183 is assessed on standing height as ((Height at Day 183 Visit - Height at Baseline Visit)/(Date at Day 183 Visit - Baseline Visit Date)) x 365.25.
Measured: At 6 month (Day 183)
Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 3 and 4
Annualized Growth Velocity at Day 183 visit is assessed on standing height as ((Height at Day 183 Visit - Height at Baseline Visit)/(Date at Day 183 Visit - Baseline Visit Date)) x 365.25.
Measured: At month 24
Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 1 and 2 Switchers
Annualized Growth Velocity at 1st visit with >= 12 months on 15ug/kg is assessed on standing height as ((Height at 1st Visit with >= 12 Months on 15 μg/kg - Height at 1st Visit on 15 μg/kg)/(Date of the 1st Visit with >= 12 months on 15 μg/kg - Date of at 1st Visit on 15 μg/kg)) x 365.25.
Measured: At month 24
Change From Baseline in Height Z-Scores Using Centers for Disease Control and Prevention (CDC) Reference Standard During Initial 6-Months
Height Z scores indicates how far a particular child is from the average height for children of the same sex and age. A positive height Z score indicates the child's height is above average whilst a negative Z score indicates the child's height is below average. Height Z scores below -2 Standard Deviation Scores (SDs) indicate a child's height is no longer within normal height range for average stature children of the same sex and age.
Z-scores are derived using non-ACH age-sex-specific reference data (means and standard deviations) per Centers for Disease Control and Prevention (CDC).
Measured: At month 6 (Day 183)
Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 3 and 4
Height Z scores indicates how far a particular child is from the average height for children of the same sex and age. A positive height Z score indicates the child's height is above average whilst a negative Z score indicates the child's height is below average. Height Z scores below -2 SDs indicate a child's height is no longer within normal height range for average stature children of the same sex and age.
Z-scores are derived using non-ACH age-sex-specific reference data (means and standard deviations) per Centers for Disease Control and Prevention (CDC).
Measured: At month 24
Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 1 and 2 Switchers
Height Z scores indicates how far a particular child is from the average height for children of the same sex and age. A positive height Z score indicates the child's height is above average whilst a negative Z score indicates the child's height is below average. Height Z scores below -2 SDs indicate a child's height is no longer within normal height range for average stature children of the same sex and age.
Z-scores are derived using non-ACH age-sex-specific reference data (means and standard deviations) per Centers for Disease Control and Prevention (CDC).
Measured: At month 24
Change From Baseline in Upper to Lower Body Ratios During Initial 6-Months
The Upper to Lower Body ratio prior to treatment, at baseline, and through 6 months is assessed on Sitting Height / (Standing Height - Sitting Height)
Measured: At month 6 (Day 183)
Change From Baseline in Upper to Lower Body Ratios During Entire Study Period - Cohort 3 and 4
The Upper to Lower Body ratio prior to treatment, at baseline, and through 24 months is assessed on Sitting Height / (Standing Height - Sitting Height)
Measured: At month 24
Change From Baseline in Upper Arm Length to Lower Arm (Forearm) Length Ratio During Initial 6-Months
The Upper Arm Length to Lower Arm (Forearm) Length ratio prior to treatment, at baseline, and through 6 months is assessed on Upper Arm Length / Lower Arm (Forearm) Length.
Measured: At month 6 (Day 183)
Change From Baseline in Upper Arm to Lower Arm Length Ratio During Entire Study Period - Cohort 3 and 4
The Upper Arm Length to Lower Arm (Forearm) Length ratio prior to treatment, at baseline, and through 24 months is assessed on Upper Arm Length / Lower Arm (Forearm) Length.
Measured: At month 24
Change From Baseline in Upper to Lower Body Ratios During Entire Study Period - Cohort 1 and 2 Switchers
The Upper to Lower Body ratio prior to treatment, at baseline, and through 24 months is assessed on Sitting Height / (Standing Height - Sitting Height)
Measured: At month 24
Change From Baseline in Upper Arm to Lower Arm Length Ratio During Entire Study Period - Cohort 1 and 2 Switchers
The Upper Arm Length to Lower Arm (Forearm) Length ratio prior to treatment, at baseline, and through 24 months is assessed on Upper Arm Length / Lower Arm (Forearm) Length.
Measured: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Knee to Heel Length Ratio During Initial 6-months
The Upper Leg Length (Thigh) to Knee to Heel Length Ratio prior to treatment, at baseline, and through 6 months is assessed on Upper Leg Length (Thigh) / Knee to Heel Length.
Measured: At month 6 (Day 183)
Change From Baseline in Upper Leg Length (Thigh) to Knee to Heel Length Ratio During Entire Study Period - Cohort 3 and 4
The Upper Leg Length (Thigh) to Knee to Heel Length Ratio prior to treatment, at baseline, and through 24 months is assessed by Upper Leg Length (Thigh) / Knee to Heel Length.
Measured: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Knee to Heel Length Ratio During Entire Study Period - Cohort 1 and 2 Switchers
The Upper Leg Length (Thigh) to Knee to Heel Length Ratio prior to treatment, at baseline, and through 24 months is assessed by Upper Leg Length (Thigh) / Knee to Heel Length.
Measured: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Tibial Length Ratio During Initial 6-months
The Upper Leg Length (Thigh) to Tibial Length Ratio prior to treatment, at baseline, and through 6 months is assessed by Upper Leg Length (Thigh)/ Tibial Leg Length.
Measured: At month 6 (Day 183)
Change From Baseline in Upper Leg Length (Thigh) to Tibial Length Ratio During Entire Study Period - Cohort 3 and 4
The Upper Leg Length (Thigh) to Tibial Length Ratio prior to treatment, at baseline, and through 24 months is assessed by Upper Leg Length (Thigh)/ Tibial Leg Length.
Measured: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Tibial Length Ratio During Entire Study Period - Cohort 1 and 2 Switchers
The Upper Leg Length (Thigh) to Tibial Length Ratio prior to treatment, at baseline, and through 24 months is assessed by Upper Leg Length (Thigh)/ Tibial Leg Length.
Measured: At month 24
Change From Baseline in Arm Span to Height Ratio During Initial 6-months
The Arm Span to Height Ratio prior to treatment, at baseline, and through 6 months is assessed by Arm Span / Standing Height.
Measured: At month 6 (Day 183)
Change From Baseline in Arm Span to Height Ratio During Entire Study Period - Cohort 3 and 4
The Arm Span to Height Ratio prior to treatment, at baseline, and through 24 months is assessed by Arm Span / Standing Height.
Measured: At month 24
Change From Baseline in Arm Span to Height Ratio During Entire Study Period - Cohort 1 and 2 Switchers
The Arm Span to Height Ratio prior to treatment, at baseline, and through 24 months is assessed by Arm Span / Standing Height.
Values are not available for participants in cohort 1 switchers for Change from Baseline to >=12 Months on 15ug/kg.
Measured: At month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2014 (estimated)
- Study start13 Jan 2014
- Primary completion2 Oct 2017
- Study completion2 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 35 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
- NCT02055157
- Sponsor's protocol ID
- 111-202
- Other IDs
- 2013-004137-32
- Acronym
- ACH
- Status last verified
- Dec 2020
- First submitted
- 18 Apr 2013
- Last updated
- 15 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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 38649657Chan ML, Qi Y, Larimore K, Cherukuri A, Seid L, Jayaram K, Jeha G, Fisheleva E, Day J, Huntsman-Labed A, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Horton WA, Henshaw J. Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Clin Pharmacokinet. 2022 Feb;61(2):263-280. doi: 10.1007/s40262-021-01059-1. Epub 2021 Aug 25.
PubMed 34431071Savarirayan 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 15 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT02055157
- Acronym / sponsor ID
- ACH / 111-202
- Conditions
- Achondroplasia
- Keywords
- Achondroplasia, Dwarfism, Bone Diseases, Developmental, Bone Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 13 Jan 2014 (actual)
- Primary completion
- 2 Oct 2017 (actual)
- Study completion
- 2 Oct 2017 (actual)
- First posted
- 5 Feb 2014 (estimated)
- Results
- Posted 15 Jan 2021
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, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: BMN 111Cohort 1: 2.5 ug/kg |
| Cohort 2 | Experimental | Drug: BMN 111Cohort 2: 7.5 ug/kg, |
| Cohort 3 | Experimental | Drug: BMN 111Cohort 3: 15 ug/Kg |
| Cohort 4 | Experimental | Drug: BMN 111Cohort 4: 30 ug/kg |
Who may qualify
- Ages
- 5 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Parent(s) or guardian(s) are willing and able to provide written, signed informed consent
- 5 to 14 years old at end of study
- ACH, documented by clinical grounds, confirmed by genetic testing
- At least 6-month of pretreatment growth assessment in Study 111-901 before study entry, and one standing height at least 6 months prior to screening for 111-202
- Negative pregnancy test at the Screening Visit for females ≥ 10 years old or who have begun menses
Key exclusion criteria (53 listed)
- Hypochondroplasia or short stature condition other than ACH
- Have any of the following:
- Unstable condition requiring surgical intervention during the study
- Growth plates have fused
- Have a history of any of the following:
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 daily for 24 months in an open-label sequential dose adjustment fashion. | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Overall Summary of Adverse Events During Initial 6-Month Period
Time frame: Up to Month 6 ± 7 Days
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. Serious adverse event (SAE).
Overall Summary of Adverse Events During Entire Study Period
Time frame: Up to Month 25 ± 7 Days
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. TEAE - Treatment-emergent adverse event. SAE - Serious adverse event.
21 secondary outcomes
Change From Baseline in Annualized Growth Velocity (AGV) During Initial 6-Month
Time frame: At 6 month (Day 183)
Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Change From Baseline in Height Z-Scores Using Centers for Disease Control and Prevention (CDC) Reference Standard During Initial 6-Months
Time frame: At month 6 (Day 183)
Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Change From Baseline in Upper to Lower Body Ratios During Initial 6-Months
Time frame: At month 6 (Day 183)
Change From Baseline in Upper to Lower Body Ratios During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Upper Arm Length to Lower Arm (Forearm) Length Ratio During Initial 6-Months
Time frame: At month 6 (Day 183)
Change From Baseline in Upper Arm to Lower Arm Length Ratio During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Upper to Lower Body Ratios During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Change From Baseline in Upper Arm to Lower Arm Length Ratio During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Knee to Heel Length Ratio During Initial 6-months
Time frame: At month 6 (Day 183)
Change From Baseline in Upper Leg Length (Thigh) to Knee to Heel Length Ratio During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Knee to Heel Length Ratio During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Tibial Length Ratio During Initial 6-months
Time frame: At month 6 (Day 183)
Change From Baseline in Upper Leg Length (Thigh) to Tibial Length Ratio During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Upper Leg Length (Thigh) to Tibial Length Ratio During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Change From Baseline in Arm Span to Height Ratio During Initial 6-months
Time frame: At month 6 (Day 183)
Change From Baseline in Arm Span to Height Ratio During Entire Study Period - Cohort 3 and 4
Time frame: At month 24
Change From Baseline in Arm Span to Height Ratio During Entire Study Period - Cohort 1 and 2 Switchers
Time frame: At month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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 34431071Chan ML, Qi Y, Larimore K, Cherukuri A, Seid L, Jayaram K, Jeha G, Fisheleva E, Day J, Huntsman-Labed A, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Horton WA, Henshaw J. Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Clin Pharmacokinet. 2022 Feb;61(2):263-280. doi: 10.1007/s40262-021-01059-1. Epub 2021 Aug 25.
- 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.
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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 11 inclusion and 53 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: finished, as of the sponsor's last update on 15 Jan 2021. 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?
Yes. Results are posted in the registry (first posted 15 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Summary of Adverse Events During Initial 6-Month Period (time frame: Up to Month 6 ± 7 Days). The study lists 2 primary outcomes in all. 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 NCT02055157, which also lists 3 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 15 Jan 2021.