NCT06153966 PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
| NCT ID | NCT06153966 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Ionis Pharmaceuticals, Inc. |
| Condition | Prion Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 76 participants |
| Start Date | 2024-01-04 |
| Primary Completion | 2027-02 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 76 participants in total. It began in 2024-01-04 with a primary completion date of 2027-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Eligibility Criteria
Key Inclusion Criteria * A confirmed diagnosis of probable or definite prion disease. * Early-stage prion disease at the time of Screening. * Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits. * Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial. * Aged ≥ 18 at the time of informed consent. Key Exclusion Criteria * Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion. * Any contraindication or unwillingness to undergo an MRI. * Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter. * Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment. * Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06153966 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Prion Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06153966 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06153966 currently recruiting?
Yes, NCT06153966 is actively recruiting participants. Contact the research team at PrionDisease@clinicaltrialmedia.com for enrollment information.
Where is the NCT06153966 trial being conducted?
This trial is being conducted at Boston, United States, New York, United States, Cleveland, United States, Parkville, Australia and 9 additional locations.
Who is sponsoring the NCT06153966 clinical trial?
NCT06153966 is sponsored by Ionis Pharmaceuticals, Inc.. The trial plans to enroll 76 participants.