Ionis Pharmaceuticals Announces Clinical Study

Ionis Pharmaceuticals has announced the ORBIT Study to evaluate the safety, pharmacokinetics, and pharmacodynamics of intrathecally administered ION356 in participants with Pelizaeus Merzbacher Disease (PMD). 

Years of hard work, industry support, and collaboration have made this moment possible. We at the PMD Foundation are incredibly excited to be witnessing this moment alongside the members of the PMD community. 

Ionis activated the first clinical trial sites for ORBIT as the initial phase of clinical trials for the investigational drug, ION356, to benefit people with Pelizaeus-Merzbacher Disease (PMD). Site investigators at the Amsterdam University Medical Center in the Netherlands and Assistance Publique-Hôpitaux of Paris in France are currently screening potential trial participants.

Up to 24 participants with ‘Classic PMD’ caused by PLP1 duplication between the ages of 2 and 17 will be enrolled into ORBIT. While additional inclusion and exclusion criteria apply, during the trial, everyone enrolled will receive ION356; there is no placebo control arm in ORBIT. The primary purpose of this study is to evaluate the safety and tolerability of ION356. ORBIT is not designed to determine the efficacy of ION356 as a potential treatment option for PMD.

We at the PMD Foundation recognize that this milestone is bittersweet for families of individuals with PMD who do not qualify for this trial. 

A spokesperson for Ionis assured the PMD Foundation that the Ionis ION356 team is focused on working with ORBIT site investigators and the PMD community to enroll ORBIT as quickly as possible. 

Ionis has expressed its gratitude for its partnership with the PMD community and the patients, caregivers, and families who will participate in ORBIT. Ionis will continue to provide the PMD community with updates at key program milestones in the future.

For more information about the purpose of clinical trials, further details are available via the World Health Organization (WHO) and the European Medicines Agency (EMA). Additionally, the US Food & Drug Administration (FDA) website has online resources that help explain the drug development process, including the different phases of clinical trials. The US Health and Human Services website has helpful educational fact sheets and videos regarding participating in research, including a suggested “Questions to Ask” when considering participating in a clinical trial.

Frequently Asked Questions about ORBIT

Where can someone interested in participating in ORBIT go for more information?

Individuals interested in participating in ORBIT should discuss it with their doctor. Individuals may call +1-844-387-9520 or email IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com for information about open trial site locations. 

Please note that Ionis and the PMD Foundation are not involved in individual enrollment decisions at clinical trial sites.

When will additional sites be opened?

Ionis is working with planned trial investigators and their institutions to open enrollment at a limited number of sites across multiple different countries. Each site will be activated only after all regulatory and individual institutional requirements are met. Therefore, additional sites will be ready to screen people for potential enrollment into the trial on a rolling basis. Ionis will update the clinicaltrials.gov page as new trial sites are added.

When will ION356 be tested for connatal PMD?

ORBIT is an early-phase study and the first time that the investigational drug will be given to anyone with PMD. The study’s objectives and design were developed with input from regulators, clinicians, ethicists, and PMD community members. The study’s objectives are to investigate the potential safety and tolerability of ION356. Considering the study’s objectives and stakeholder feedback, enrollment is focused on people with ‘Classic PMD’ (PLP1 duplications) between the ages of 2 and 17. Results from this study will inform potential future studies of ION356, which may include research in people with connatal PMD.

Is there a way to receive ION356 outside the clinical trial, such as for people who are not eligible for this clinical trial and those who don’t live near a clinical trial site?

Evaluation of both the potential safety and efficacy of the investigational drug, ION356, in clinical trials is essential to establishing whether ION356 can help people diagnosed with PMD. For this reason, ION356 is not available outside this clinical trial. Ionis is working diligently to complete the clinical trials necessary to evaluate ION356 in individuals with PMD.  For more information about Ionis’ expanded access policy, click here.

Must a person be a citizen or resident in the same country as the trial site to participate in the trial? (aka cross-border/cross-country participation)?

For this study, Ionis does not require a person to be a citizen or resident of the country where the trial site is located. However, each site may have its own policies for receiving individuals from other countries. These policies are independent of Ionis. Prospective study participants should speak directly with their preferred study site to identify any restrictions or challenges based on the institution’s policies.