About
Arbaclofen (STX209) is currently in Phase III testing for fragile X syndrome and Phase II testing for autism. Both of these clinical trials also had open label extensions which participants could decide to opt in after completing the respective phase II or III trial. During this extension, participants were able control their dosing within the dosing guides of the study and receive the actual medication that they may not have had in the double blind phase II or III portion of their trial.
On May 15, 2013 Seaside Therapeutics, Inc., the sponsor of arbaclofen sent a communication to the clinics involved in the administration of arbaclofen stating the following:
“We regret to inform you that Study 209FX303 [An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of STX209 (arbaclofen) in subjects with Fragile X Syndrome] is being terminated immediately. The closure of the study is due to resource limitations at Seaside Therapeutics, Inc., and is not related to any known safety issues in patients dosed with STX209.”
As this announcement slowly made its’ way through the participants of the open label extension, many of the participants began speaking up and asking questions. The participants had previously been led to believe they would have access to this drug until FDA approval and should any changes be made they would be given 4-6 months advance notice to make any necessary plans. In some cases, this announcement led to just days before having to start weaning off this drug that had been helping the participants so much.
We are asking Roche Pharmaceuticals to reinstate the financing that was withdrawn from Seaside following certain Phase II results which had demonstrated improvements in the secondary endpoints. This funding made the Phase III STX209 extension trial in fragile X and the Phase II STX209 extension trial in autism possible. We are asking for Roche’s assistance in reinstating the open label extensions for both the fragile X and the autism trial under Seaside. We are asking Roche to continue to support Seaside, as it has previously, develop this medication and pursue FDA approval.
About Fragile X Syndrome:
Fragile X syndrome (FXS) is the most common known cause of inherited intellectual disability. Symptoms of FXS include a range in learning disabilities to more severe cognitive or intellectual disabilities that were previously referred to as “mental retardation.” Delays in speech and language development are common, as are a variety of physical and behavioral characteristics. FXS is also the most common known cause of autism or autism spectrum disorders. FXS is caused by a “full mutation” of the FMR1 gene, a human gene that codes for a protein called fragile X mental retardation protein, or FMRP. This protein, most commonly found in the brain, is essential for normal cognitive development and female reproductive function.
About Clinical Trials:
There are eight steps to a clinical trial: animal testing , new drug (IND) application process, phase I testing, phase II testing, phase III testing, review meeting, new drug application (nda), and application review.
Animal testing:
1. Pre-clinical testing is performed on laboratory animals.
2. IND Application Process: Those involved in the process of developing a drug have to show the results of animal testing and what they are doing in human testing. The FDA and a local IRB then determine and approve the trial protocols, make sure the study is acceptable, the participants have given informed consent, and that researches take appropriate steps to protect patients from harm.
Human testing:
Each phase of testing sets a primary endpoint and possibly some secondary endpoints. A primary endpoint is what the study hopes to prove with the data from the clinical trial. Secondary endpoints are hypotheses that are measured and expected to be met in the clinical trial as well. In order for these phases to succeed, the primary endpoint must be met. Should the primary endpoint not be met, the trial will fail even if the secondary endpoints are met.
3. Phase 1 Testing: Phase 1 studies are usually conducted in healthy volunteers. The goal is primarily to determine side effects and how the drug is metabolized and excreted.
4. Phase 2 Testing: While Phase 1 focuses on safety, Phase 2 focuses on effectiveness. Companies are looking for effectiveness of a drug in a certain subset of people with a disease or condition while still focusing on safety and side effects.
5. Phase 3 Testing: After Phase 2 trials have shown effectiveness, phase 3 testing begins to gather more information about safety and effectiveness using different dosing and participants.
6. Review Meeting: A review meeting takes place between the drug sponsor and the FDA prior to the new drug application.
7. New Drug Application: This is the actual application that will either give the drug approval or not. This includes all information about animal and human testing as well as how the drug works in the body.
8. Application Review: Every application is reviewed to see if it is allowed to be filed for approval. If filed, a team will be assigned to go over research, safety, and effectiveness.