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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04554940

Registration number
Ethics application status
Date submitted
Date registered
Date last updated

Titles & IDs
Public title
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Scientific title
A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery
Secondary ID [1] 0 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Study type
Description of intervention(s) / exposure
Other interventions - vosoritide

Experimental: Vosoritide + Standard of Care - Standard of Care treatment for cervicomedullary compression and once daily subcutaneous injection of vosoritide at 30µg/kg (ages 0 - <2 years old) or 15 µg/kg (ages >2 years old)

No Intervention: Standard of Care Alone - Institutional standard of care monitoring and treatment for cervicomedullary compression

Other interventions: vosoritide
Subcutaneous injection of 30µg/kg (ages 0 - <2 years old) or 15 µg/kg (ages >2 years old) of vosoritide daily

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Timepoint [1] 0 0
Through Week 260
Secondary outcome [1] 0 0
Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software
Timepoint [1] 0 0
Through Week 260

Key inclusion criteria
- Parent(s) or guardian(s) willing and able to provide signed informed consent after the
nature of the study has been explained and prior to performance of any research
related procedure.

- Have ACH, documented by genetic testing.

- Are willing and able to perform all study procedures as physically possible.

- Age 0 to = 12 months, at study entry (Day 1). Given that any potential impact of
vosoritide therapy on the foramen magnum is dependent on treating as early as possible
and as long as possible while the synchondroses at the base of the skull are still
open. For subjects > 6 months of age at enrollment, a discussion between the
investigator and the Medical Monitor should occur with the goal of limiting the number
of subjects in the range of > 6 months to = 12 months of age.

- Parent(s) or caregiver(s) are willing to administer daily injections to the subject
and complete the required training.

- Have evidence of CMC that "may" require surgical intervention
Minimum age
No limit
Maximum age
12 Months
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
- Have hypochondroplasia or short-stature condition other than achondroplasia (eg,
trisomy 21, pseudoachondroplasia, etc).

- Have CMC that either does not require surgical intervention (for example foramen
magnum narrowing with preservation of the cerebrospinal fluid space) or does require
immediate surgical intervention .

- Have any of the following: Untreated congenital hypothyroidism or maternal history of
hyperthyroidism, Insulin-requiring neonatal diabetes mellitus, Autoimmune inflammatory
disease, Inflammatory bowel disease, Autonomic neuropathy.

- Have a history of any of the following:Renal insufficiency, Chronic anemia,Baseline
systolic blood pressure below age and gender specified normal range or recurrent
symptomatic hypotension (defined as episodes of low blood pressure generally
accompanied by symptoms eg, pallor, cyanosis, irritability, poor feeding) and Cardiac
or vascular disease.

- Have a clinically significant finding or arrhythmia that indicates abnormal cardiac
function or conduction or QTc-F = 450 msec on screening ECG.

- Have been treated with growth hormone, insulin-like growth factor 1, or anabolic
steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at
any time.

- Have ever had prior cervicomedullary decompression surgery.

- Have had a fracture of the long bones or spine within 6 months prior to Screening.

Study design
Purpose of the study
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?

Intervention assignment
Other design features
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
BioMarin Pharmaceutical

Ethics approval
Ethics application status

Brief summary
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and
young children with a diagnosis of Achondroplasia at a heightened risk of requiring
cervicomedullary decompression surgery
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
BioMarin Pharmaceutical
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Specialist
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04554940