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

Achondroplasia Clinical Trial

Official 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04554940
• Other study ID #: 111-209
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: October 10, 2020
• Est. completion date: December 2027

Study information

• Verified date: December 2023
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 20
• Est. completion date: December 2027
• Est. primary completion date: December 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Months to 12 Months

Eligibility

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.

Related Conditions & MeSH terms

• Achondroplasia

Intervention

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

Locations

Country	Name	City	State
Australia	Murdoch Children's Research Institute	Parkville	Victoria
United Kingdom	Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital	London
United Kingdom	Sheffield Children's NHS Foundation Trust	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

Australia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]		Through Week 260
Secondary	Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software		Through Week 260