Current partner codePEPTIDESDE
NCT04554940·Phase 2·INTERVENTIONAL

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Status

Active, not recruiting

Phase

Phase 2

Enrollment

20

Locations

3

Results

Not posted

Publications

1

Study summary

What the protocol is testing.

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

Interventions

Treatment arms and agents.

BIOLOGICAL

vosoritide

Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Timeline

From registration to results.

  1. First posted

    Sep 18, 2020

  2. Study start

    Oct 10, 2020

  3. Primary completion

    Dec 2027

  4. Study completion

    Dec 2027

  5. Results posted

    Not reported

  6. Registry updated

    Mar 13, 2026

Outcomes

What the study measures.

Primary outcomes

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Time frame · Through Week 260

Secondary outcomes

Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software

Time frame · Through Week 260

Eligibility

Who can take part.

Minimum age
0 Months
Maximum age
12 Months
Sex
ALL
Healthy volunteers
No

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 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 locations

3 registered sites.

Australia · United Kingdom. Showing up to 24 locations stored in the fast local snapshot.

Murdoch Children's Research Institute

Parkville, Victoria, Australia

Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital

London, United Kingdom

Sheffield Children's NHS Foundation Trust

Sheffield, United Kingdom

Related trials

More studies on Vosoritide.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.