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Tests treatment safety and results for Pelizaeus-Merzbacher Disease
Official title Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
ClinicalTrials.gov ID: NCT06150716
What this study is testing
What is ION356?
ION356 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for pelizaeus-merzbacher disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17, men only
You may be able to join if
- Key 1. Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback...
- Diagnosis of PMD with genetic confirmation of PLP1 gene duplication.
- Clinical phenotype and brain imaging consistent with a diagnosis of PMD.
- Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.
- Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and...
You likely can't join if
- Clinically significant abnormalities in medical history, laboratory tests or physical examination.
- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the...
- Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI).
- Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent...
- Previous treatment with an oligonucleotide (including small interfering ribonucleic acid) within 4 months of Screening if a single dose was received...
- History of gene therapy or cell transplantation, or any experimental brain surgery.
See the full eligibility criteria
- Key 1. Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study...
- Diagnosis of PMD with genetic confirmation of PLP1 gene duplication.
- Clinical phenotype and brain imaging consistent with a diagnosis of PMD.
- Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.
- Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits.
- Clinically significant abnormalities in medical history, laboratory tests or physical examination.
- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator.
- Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI).
- Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent, whichever is longer.
- Previous treatment with an oligonucleotide (including small interfering ribonucleic acid) within 4 months of Screening if a single dose was received, or within 12 months of Screening if multiple doses were received...
- History of gene therapy or cell transplantation, or any experimental brain surgery.
- Current obstructive hydrocephalus.
- Known brain or spinal disease or previous spinal surgery that would interfere with the lumbar puncture (LP) process, CSF circulation, or safety assessment.
- Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study.
- Have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
The study team makes the final eligibility decision.
Where it's taking place
- Atlanta, Georgia, United States
- Philadelphia, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Le Kremlin-Bicêtre, France
- Göttingen, Germany
- Tokyo, Japan
- Amsterdam, Netherlands
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Atlanta, Georgia, United States; Philadelphia, Pennsylvania, United States; Salt Lake City, Utah, United States; Le Kremlin-Bicêtre, France; Göttingen, Germany; Tokyo, Japan and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.