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Clinical Trials in Italy / NCT05659901
Recruiting Observational

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901 · tracked via the Priya Life Science Italy tracker
Sponsor
Ionis Pharmaceuticals, Inc
Phase
Observational
Started
2022-10-03
Last updated
2026-10-02

Condition(s) studied

Pelizaeus-Merzbacher Disease

Study summary

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.

Eligibility

Sex
MALE
Min age
6 Months
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: 1. Participant has a parent or caregiver capable of providing informed consent (signed and dated) and able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol and be able to comply with all study requirements 2. Participant has a diagnosis of Pelizaeus-Merzbacher Disease with genetic confirmation of PLP1 duplication 3. Male, 6 months-17 years old, inclusive, at the time of informed consent and phenotype consistent with classic PMD 4. No contraindications for lumbar punctures (LPs), blood draws, neuroimaging, sedation (if necessary) or other study procedures Exclusion Criteria: 1. Clinically significant abnormalities in medical history or physical examination 2. \> 2 copies of the PLP1 gene 3. Have any other conditions, which, in the opinion of the investigator would make the participant unsuitable for inclusion, or could interfere with the participant taking part in or completing the study

Primary outcome measure(s)

  • Assess Longitudinal Changes in Fluid Biomarkers — Up to 26 months
    Changes in Proteolipid Protein 1 (PLP1) in CSF and disease related biomarkers
  • Assess Longitudinal Changes in Neuroimaging Parameters — Up to 26 months
    Changes in regional brain volumes (MRI) and in brain metabolites (MRS)
  • Assess longitudinal changes in performance on clinical, and patient and caregiver-reported outcome assessments — Up to 26 months
    Includes collection of gross and fine motor outcomes, spasticity, dysphagia, cognition and behavior, and sleep.

Trial sites (10)

FacilityCityRegionStatus
Ionis Investigative Site Atlanta Georgia Recruiting
Ionis Investigative Site Baltimore Maryland Recruiting
Ionis Investigative Site Philadelphia Pennsylvania Recruiting
Ionis Investigative Site Clermont-Ferrand France Completed
Ionis Investigative Site Le Kremlin-Bicêtre France Active Not Recruiting
Ionis Investigative Site Göttingen Germany Completed
Ionis Investigative Site Tel Aviv Israel Recruiting
Ionis Investigative Site Milan Italy Recruiting
Ionis Investigative Site Amsterdam Netherlands Recruiting
Ionis Investigative Site Leeds United Kingdom Recruiting

More Ionis Pharmaceuticals, Inc. trials in Italy

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) Active, not recruiting
Donidalorsen Treatment in Children With Hereditary Angioedema Recruiting
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome Active, not recruiting
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) Active, not recruiting
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG) Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05659901 on ClinicalTrials.gov ↗ ← All trials in Italy