Actively Recruiting
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-04-17
32
Participants Needed
9
Research Sites
334 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to Pelizaeus-Merzbacher disease (PMD) and longitudinal changes in performance on clinical, participant, and caregiver-reported outcome assessments to inform the development of therapies for PMD.
CONDITIONS
Official Title
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant has a parent or caregiver capable of providing informed consent and attending all study visits
- Diagnosis of Pelizaeus-Merzbacher Disease with genetic confirmation of PLP1 duplication
- Male aged 6 months to 17 years inclusive at time of consent with phenotype consistent with classic PMD
- No contraindications for lumbar punctures, blood draws, neuroimaging, sedation, or other study procedures
You will not qualify if you...
- Clinically significant abnormalities in medical history or physical exam
- More than 2 copies of the PLP1 gene
- Any other conditions that would make the participant unsuitable or interfere with study participation according to the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 9 locations
1
Ionis Investigative Site
Atlanta, Georgia, United States, 30342
Actively Recruiting
2
Ionis Investigative Site
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
Ionis Investigative Site
Clermont-Ferrand, France
Active, Not Recruiting
4
Ionis Investigative Site
Le Kremlin-Bicêtre, France, 94270
Active, Not Recruiting
5
Ionis Investigative Site
Göttingen, Germany
Active, Not Recruiting
6
Ionis Investigative Site
Tel Aviv, Israel, 6423906
Actively Recruiting
7
Ionis Investigative Site
Milan, Italy, 20154
Actively Recruiting
8
Ionis Investigative Site
Amsterdam, Netherlands, 1081 HV
Actively Recruiting
9
Ionis Investigative Site
Leeds, United Kingdom, LS1 3EX
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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