The study protocol synopsis for the CS1-004 Expanded Access Study, titled; "Expanded Access, Open-Label, Safety Extension Study for Patients That Have Completed Parent Study CS1-003 and Who Are Judged by the Investigator to Benefit From Continued CS1 Treatment" is now published on ClinicalTrials.gov.
The EAP study named CS-004, is an extension of the CS1-003 Study. The primary objective of the CS1-004 study is to evaluate long-term safety and tolerability of continued treatment with CS1. The exploratory objectives are to evaluate clinical benefit by using
Safety and exploratory efficacy endpoints will be measured, using the same methods as in the parent study, at the start of the study and subsequently at 4, 8 and 12 months every year. As such, the EAP study will allow Cereno to gather further documentation of CS1 use in patients suffering from PAH, which will help in discussions with regulatory authorities and to design our Phase IIb/III pivotal study with CS1.
Up to 30 patients could participate in Study CS1-004, if they completed the parent study, tolerated CS1 treatment, and if, in the investigator's judgement, the benefits of continued treatment with CS1 outweigh the risk. Some patients will directly roll-over into Study CS1-004 with no disruption in CS1 treatment, while other patients that have already completed Study CS1-003, will need to be restarted on CS1. Patients will, initially, receive the same dose they received in the parent study CS1-003. All patients will later have the option to be titrated to the most efficacious and safe dose determined from the ongoing CS1-003 study, when completed.
On a yearly basis, the principal investigator will determine if a patient continues the Expanded Access study for a subsequent year based on safety, tolerability, and clinical benefit of CS1. Continuation of the Expanded Access study will also be evaluated on a yearly basis by
As previously reported, investigators have shown substantial interest in the EAP with two-thirds of the patients, having completed the study or are currently on therapy, deemed by investigators to be eligible for continued access to CS1. Currently site-specific and Ethics Committee (IRB) approvals are underway and once these are in place the first patients can be dosed in the EAP.
"The EAP is a long-term follow-up study of CS1 treatment in patients with PAH. We are happy not only that we can continue to, under an FDA approved protocol, provide patients, judged to benefit from further CS1 treatment, with continued access to CS1 for as long as deemed favorable by their treating physician. In addition, we value that this extended study design allows us to gather additional data on the long-term safety and efficacy of CS1 treatment. Data that can be used to guide discussions with regulators and, in the future, support commercial positioning of CS1," said Sten R. Sörensen, CEO,
The information on ClinicalTrials.gov contains a detailed description of study design, inclusion and exclusion criteria as well as primary and secondary objectives.
Read the protocol synopsis here.
ClinicalTrial.gov identifier: NCT06321705
For further information, please contact:
Email: henrik.westdahl@cerenoscientific.com
Phone: +46 70-817 59 96
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
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