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19/03/2024

The Arthroscopic Surgery Unit (UCA) begins the final phase of a clinical trial aimed at treating achondroplasia in children.

• Twittear

One in 25,000 children suffers from achondroplasia. A disease considered rare, generated by a DNA modification that affects bone growth, and the main cause of a short final height of about 131 cm in males and 124 cm in females, with an early age and deformities. The most common and widespread treatment is limb lengthening with complex surgeries, with high complication rates, and which can extend over four to five years.

One of these trials is promoted by the pharmaceutical company QED Therapeutics, which on March 6 and 7 organized a specific congress in London to inform specialists and centers about the protocols for the start of Phase 3, which will begin this April. In this context, the UCA is the center that has recruited the most patients, 10 boys and girls, and is responsible for the collection of the study data.

Dr. Josep María de Bergua, specialist in skeletal dysplasias and one of the members of the team that makes up the pediatric unit of the UCA, together with the head of the unit, Dr. Gorka Knörr, Dr. Lucía Guidali, the researcher and coordinator of the clinical trials, Dr. Irene Gimeno, and the physiotherapist Paula Caridad, specialist in anthropometric measurements in these same trials at UCA, Dr. Irene Gimeno, and the physiotherapist Paula Caridad, specialist in anthropometric measurements in these same trials carried out at UCA, know first hand the QED clinical trial from the beginning and the impact it is having on patients. After a first observational phase in patients at the beginning of 2020, Phase 2 began, which allowed the safety and efficacy of the drug to be demonstrated.

For the UCA team, the objective of this new generation of drugs is to gain growth speed, in addition to improving the quality of life and minimizing the complications that occur throughout the life of people affected as a result of this genetic disease.

Start of Phase 3

In this context, remembering that achondroplasia is not just a question of height, but that it carries with it numerous pathologies is another of the mantras that the UCA team self-imposes every day. “We cannot lose focus of the fact that behind all the tests and procedures are both the families and the children who participate in the trials, so we must provide them with all the information we have so that they can choose among the treatment options they have right now,” argues Dr. Irene Gimeno.

Having successfully completed Phase 2, the QED trial has just begun its third, and final, stage. The outcome, once all the medical expectations have been tested and surpassed, could lead to the launching on the market of a revolutionary drug that will completely change the lives of thousands of children affected by achondroplasia.