New clinical sites open in US and Germany for Ionis ORBIT Phase 1 clinical trial of an investigational drug, ION356, in people with Pelizaeus-Merzbacher Disease (PMD)
ORBIT is an important step in the clinical development process for the investigational drug, ION356. 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.
Recruitment sites:
- Amsterdam University Medical Center in the Netherlands
- Assistance Publique-Hôpitaux of Paris in France
- Children’s Hospital of Atlanta in United States
- Georg-August-Universität-Göttingen in Germany
Study Information: Study Details | Orbit Study, Pelizaeus Merzbacher Disease (PMD) | ClinicalTrials.gov
Any individuals interested in participating should speak with their doctor or contact our team at 844-387-9520 or IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com to learn more about sites and enrolment.
Can I take part in the clinical trial if I live in the UK?
Ionis does not require that a person be a citizen or resident of the country in which the study site is located. So, families from the UK could be screened in Germany, Netherlands, or France. However, each study site has its own policies for receiving individuals from other countries. These policies are independent of Ionis. Families are encouraged to first speak with their physician about potentially participating in the study, and then interested individuals can reach out to the Ionis clinical trial team at +1 844 387 9520 or by email at IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com to be connected to a potential study site. Eligibility for the study is then determined by the trial site investigator at that site, as Ionis does not make individual enrollment decisions.
Additionally, it is important to note that Ionis requires that individuals in ORBIT and their caregivers provide data throughout the trial to investigators. Some of that data collection must be done using an electronic tablet that Ionis has provided to each research site. Each electronic tablet contains questionnaires in the languages common to the region where the study site is located. Participants are not allowed to complete their questionnaires using their own tablet. Therefore, participants and their caregivers must be able to read and write in the languages available on that study site’s tablet to participate in the study. Again, this is something that families can speak to a study site about once they are connected to learn more about their participation. All prospective study participants will discuss this aspect of the trial with the study site before being accepted.
