PIONEER Trials
Overview of the PIONEER-1 and
PIONEER-2 trials
The objective of the PIONEER-1 and PIONEER-2 studies is to further evaluate the efficacy of Privosegtor versus placebo on low contrast visual acuity (LCVA) in participants with optic neuritis at Month 3 and to evaluate its safety and tolerability profile over 12 months.
Patients enrolled in the PIONEER-1 or PIONEER-2 trials will be randomly placed in one of two groups to receive either Privosegtor + steroid or placebo + steroid. Participants will receive treatment regardless of which group to which they are assigned. All participants will receive methylprednisolone, which is the standard-of-care treatment for this condition which works by reducing swelling in the optic nerve.
If participants are assigned to the Privosegtor group, in addition to the standard of care methylprednisolone they will also receive the investigational treatment Privosegtor which is intended to potentially protect the retinal ganglion cells and the axons in the optic nerve after a sudden episode of ON that may lead to improved vision. The study treatment which includes in both groups the standard of care must be started as early as possible to help treat optic neuritis.
standard of care methylprednisolone they will also receive the investigational treatment Privosegtor which is intended to potentially protect the retinal ganglion cells and the axons in the optic nerve after a sudden episode of ON that may lead to improved vision. The study treatment which includes in both groups the standard of care must be started as early as possible to help treat optic neuritis.
During the treatment period, participants will receive a daily dose of treatment, ideally during 5 consecutive days. At each treatment, participants will first receive the standard of care (methylprednisolone IV at 1 g/day) followed by the study medicine infusion. After completing this treatment cycle of 5 days, participants will remain in this study for about 12 months. Throughout the trial, participants will meet with their trial doctor in regular scheduled visits, where they will take part in assessments and procedures.
To participate in the PIONEER-1 or PIONEER-2 trials,
participants must:
- Be between 18 and 50 years old, and experiencing their first episode of unilateral optic neuritis (ON) in the eye with vision loss
- Have had vision symptoms start within the last 12 days before receiving the study treatment
- Be able and willing to sign an informed consent form and agree to follow all study requirements and restrictions for 12 months
If you are considering referring a patient to participate in the PIONEER trials it is important to refer them as early as possible. Please make sure the patients referred are adult men and women (aged 18 to 50 years) with the first episode of ON in the study eye, associated with unilateral visual loss. The onset of visual loss symptoms must be within the previous 12 days before first administration of study treatment, for the patient to be eligible.
To refer a patient to the PIONEER-1 and PIONEER-2 trials, please follow this link
What are the risks and benefits of being
in one of the PIONEER trials?
Any research study has some risks, which may include things that could make study participants feel unwell, uncomfortable, or harm them. Study participants may have adverse effects related to study medicine while taking part in the study that are listed below, however, all the possible adverse effects that the study medicine may cause are not known. In the Phase 2 trial conducted in optic neuritis participants, there were no drug-related adverse events (AEs) leading to drug withdrawal or study discontinuation and no drug-related serious adverse events. The most common drug‑related adverse events (AEs) were headache and acne (each in two participants; 10.5%).
The study treatment may work better than the standard of care for the study participant’s condition, but it cannot be promised that this will happen. The study treatment might not work at all, or it might have bad side effects. Even if the study does not help study participants directly, their participation in this study may help other people in the future by helping us learn more about Privosegtor and ON.
The PIONEER trials are registrational clinical trials. These trials follow the completed Phase 2 trial in optic neuritis that supported further investigation of Privosegtor.
Registrational trials are usually completed within a larger patient population to evaluate the investigational treatment. If results support the use of Privosegtor for treating optic neuritis, applications will be submitted to the US FDA (and counterpart regulatory agencies in other countries) for review.1,2
Risks of participating in one of the PIONEER trials
Any research study has some risks, which may include things that could make study participants feel unwell, uncomfortable, or harm them. Study participants may have adverse effects related to study medicine while taking part in the study that are listed below, however, all the possible adverse effects that the study medicine may cause are not known. In the previous study conducted in optic neuritis participants the most common drug‑related adverse events (AEs) were headache and acne (each in two participants; 10.5%).
All participants in the studies will be closely monitored for any side effects; however, the
study team does not know all the potential side effects that Privosegtor may have on study participants. The study team may give study participants medicine(s) to help reduce side effects.
With any clinical trial involving human subjects, an Institutional Review Board (IRB) is required to review the trial and ensure all volunteers have their rights and welfare protected.3 For more information on the role of an IRB, browse this list of Frequently Asked Questions by the US Food & Drug Administration.
Benefits of participating in one of the PIONEER trials
While there is no guarantee that study participants with optic neuritis will receive medical benefits from participating in one of the PIONEER trials, there are several reasons to consider getting screened for the study.
- Study participants will be helping others by contributing to medical research
- Study participants will receive close medical monitoring of their condition throughout their participation in the study
- Through random assignment, study participants may be selected to receive Privosegtor (instead of placebo), which may provide potential treatment benefits
As with any medical decision, it is important to discuss the risks and benefits with potential study participants when determining whether to enroll in one of the PIONEER trials.
Oculis is not responsible for the content of any third-party sites or resources, and cannot guarantee the quality of information or support provided.
Privosegtor is an investigational drug candidate and has not been approved by the FDA. Safety and efficacy of Privosegtor for optic neuritis have not been established.
References
1. ClinicalTrials.gov. Protocol Registration Data Element Definitions for Interventional and Observational Studies. Accessed August 2, 2024. https://clinicaltrials.gov/policy/protocol-definitions. 2. Food and Drug Administration. What Are the Different Types of Clinical Research? Accessed August 6, 2024. https://www.fda.gov/patients/clinical-trials-what-patients-need-know/what-are-different-types-clinical-research. 3. Food and Drug Administration. Institutional Review Boards (IRBs) and Protection of Human Subjects in Clinical Trials. Accessed July 23, 2024. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/institutional-review-boards-irbs-and-protection-human-subjects-clinical-trials.