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Trial · Interventional

Active, not recruitingPhase 2Registry data

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

Overview

Status
Active, not recruiting
Phase
Phase 2
Enrollment
20 participants (actual)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Randomized · Open label (no masking)
Countries
2 countries

Australia, United Kingdom

Start
10 Oct 2020
Primary completion
Dec 2027 (estimated)
Completion
Dec 2027 (estimated)
First posted
18 Sept 2020
Last update posted
13 Mar 2026
Conditions
Achondroplasia

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20202022202420262028
  1. Ph 2

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

    NCT04554940

    10 Oct 2020 to est. Dec 2027

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

  • vosoritideBiological

Arms2

  • Experimental

    Vosoritide + Standard of Care

    Standard of Care treatment for cervicomedullary compression and once daily subcutaneous injection of recommended dose of vosoritide based on weight-band dosing.

  • No intervention

    Standard of Care Alone

    Institutional standard of care monitoring and treatment for cervicomedullary compression

Primary outcomes1

Outcomes the sponsor listed as primary.

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

    Time frame · Through Week 260

Secondary outcomes(1)
  1. Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software

    Time frame · Through Week 260

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

More studies

1 study on this page

Indexing hint: Clinical trial · 2021

Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery.

Savarirayan et al. 2021Science progresscited by 20

PMID 33761804PMC10395166DOI 10.1177/00368504211003782PUB-33761804

Free to read · publisher termsCC BY-NC
Read at source

Eligibility

Who could join, as posted.

Sex
All
Age
from 0 Months up to 12 Months
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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