A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04554940|
Recruitment Status : Active, not recruiting
First Posted : September 18, 2020
Last Update Posted : December 22, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Achondroplasia||Biological: vosoritide||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||October 10, 2020|
|Estimated Primary Completion Date :||December 2027|
|Estimated Study Completion Date :||December 2027|
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.
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
No Intervention: Standard of Care Alone
Institutional standard of care monitoring and treatment for cervicomedullary compression
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through Week 260 ]
- Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software [ Time Frame: Through Week 260 ]
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|Ages Eligible for Study:||0 Months to 12 Months (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04554940
|Murdoch Children's Research Institute|
|Parkville, Victoria, Australia, 3052|
|Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital|
|London, United Kingdom, SE1 7EH|
|Sheffield Children's NHS Foundation Trust|
|Sheffield, United Kingdom, S10 2TH|
|Study Director:||Medical Director, MD||BioMarin Pharmaceutical|
|Responsible Party:||BioMarin Pharmaceutical|
|Other Study ID Numbers:||
|First Posted:||September 18, 2020 Key Record Dates|
|Last Update Posted:||December 22, 2022|
|Last Verified:||December 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||Yes|
Bone Diseases, Developmental ACH
Natriuretic Peptide, C-Type
Bone Diseases, Developmental
Genetic Diseases, Inborn