Prof Servais on the GTX-102 Phase 1/2 clinical trial in the UK
Professor Laurent Servais from the University of Oxford discusses the upcoming Phase 1/2 clinical trial of GTX-102-001, also known as KIK-AS, in the UK. He emphasizes that this trial is not a treatment or cure, but rather an opportunity to answer the question of whether the drug is safe and effective. The drug belongs to the oligonucleotide antisense family and works by interfering with the splicing process of RNA. The trial has already started in the US and has shown some positive results, but also some safety concerns at higher doses. The UK trial will start with lower doses and will involve up to 12 children. If successful, the drug could potentially be available in about four years. Participants in the trial will not have to pay for the drug and may continue to receive it after the trial if it proves to be effective. The trial will require anesthesia or sedation for administration. Professor Servais encourages people to sign a petition for newborn screening for spinal muscular atrophy and hopes that one day there will be effective drugs for Angelman syndrome as well.
In this talk, Professor Laurent Servais, a Pediatric Neuromuscular Disease expert at the University of Oxford, provides an overview of the upcoming Phase 1/2 clinical trial of GTX-102-001 in the UK. He emphasizes the importance of understanding that a trial is not a treatment or cure, but rather an opportunity to answer questions about the drug’s safety and efficacy.
Understanding the Drug
GTX-102 belongs to a family of drugs called oligonucleotide antisense, which have been in use for over 22 years. These drugs interfere with the splicing process of RNA and have shown promise in treating various conditions. GTX-102 is similar to Spinraza, another drug in the same family, and is administered intrathecally.
Previous Study in the US
A Phase 1/2 study of GTX-102 was conducted in the US, focusing primarily on safety. The study involved four monthly doses of the drug, with different dose escalations. While adverse events such as transient ataxia and fatigue were observed, they were reversible and did not lead to serious complications. Some patients experienced lower extremity weakness, but it was also reversible. The study showed potential efficacy in improving fine motor skills, communication, and behavior.
The UK Trial
The UK trial, called KIK-AS, has received approval from the MHRA and is awaiting final ethics approval. It will start with lower doses of 3.3 and 5 milligrams, with four monthly doses over three months, followed by maintenance doses every three months. The final dose will depend on safety and efficacy signals. The trial will involve a rigorous schedule of events and require anesthesia or sedation for administration.
Trial Inclusion and Exclusion Criteria
The trial in the US focused on a specific age group (4-17 years) and specific criteria such as stable seizure control and normal kidney and liver function. This does not mean that the drug cannot work for other age groups or genotypes, but trials require well-defined populations for initial testing.
Future Trials and Availability
There are many other trials in progress, and efforts are being made to improve clinical trial readiness in the UK. If successful, the drug may become available in approximately four years, but this is speculative. Participants in clinical trials typically continue to receive the drug if it proves effective. The potential for treating adults will require bridging data from children to adults and further study.
Professor Servais encourages individuals to connect with him or the clinical sites for more information about the trial. He emphasizes the importance of natural history studies, the quality of data, and understanding the epidemiology in the UK for the drug to be recommended by NICE. He also highlights the need for support in advocating for rare diseases, such as Angelman syndrome, and invites readers to sign a parliamentary petition for newborn screening for spinal muscular atrophy.
- Title: Prof Servais on the GTX-102 Phase 1/2 clinical trial in the UK
- Author(s): Laurent Servais
- Author(s)’ affiliation: University of Oxford
- Publication date: 2021-07-11
- Collection: FAST UK Webinars