Achondroplasia with Cervicomedullary compression protocol amendment 1
Research type
Research Study
Full 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
IRAS ID
282694
Contact name
Ana Zaccaro
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2020-001055-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
7 years, 1 months, 28 days
Research summary
Achondroplasia is the most common type of short stature. It is a genetic condition that causes shorter than average bone growth. 20 children aged 0 – 1 year old with achondroplasia (ACH), cervicomedullary compression (CMC) and at risk of decompression surgery will be asked to participate in this study in specialist children’s hospitals. CMC is a condition where the opening at the base of the skull called the foramen magnum, through which the brain connects to the spinal cord, is narrow. CMC is a life-threatening condition requiring surgery to enlarge the foramen magnum and relieve the pressure on spinal cord. The purpose of this study is to learn about the effects, good and/or bad, of a drug called vosoritide has on children with ACH at risk of requiring cervicomedullary decompression surgery.
For first 2 years, children will be equally assigned to receive either standard of care treatment (as decided by the doctor) or study drug (vosoritide) combined with standard of care treatment. At 2 year mark, eligible children will start receiving study drug in addition to standard of care. Total study duration will be about 5 years.
Some tests may be part of regular care for child’s ACH. However, this study includes tests and procedures that are not part of regular medical care for children with ACH. They are being done in this study to keep track of health and to see how the study drug affects the body. Blood samples will be collected to evaluate safety and the effect of the drug on organs. Body measurements and periodic Magnetic Resonance Imaging (MRI) will monitor changes in the bones. Children will also be assessed for developmental milestones.
Participants will visit the clinic at least 22 times during the initial 5-year period.REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
20/YH/0182
Date of REC Opinion
29 Jul 2020
REC opinion
Further Information Favourable Opinion