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

Recruiting now

Conditions studied: Pelizaeus-Merzbacher Disease

In brief

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.

Key facts

Study ID

NCT05659901

Run by

Ionis Pharmaceuticals, Inc.

People needed

32

Starts

2022-10-03

Expected to finish

2029-03-01

Last updated by the study team

2026-04-17

Who can join

Age: 1 and older, up to 17. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Participant has a diagnosis of Pelizaeus-Merzbacher Disease with genetic confirmation of PLP1 duplication
• Male, 6 months-17 years old, inclusive, at the time of informed consent and phenotype consistent with classic PMD
• No contraindications for lumbar punctures (LPs), blood draws, neuroimaging, sedation (if necessary) or other study procedures

You may not qualify if…

• Clinically significant abnormalities in medical history or physical examination
• > 2 copies of the PLP1 gene
• 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

Where it is running

• Ionis Investigative Site — Atlanta, Georgia, United States (enrolling)
• Ionis Investigative Site — Philadelphia, Pennsylvania, United States (enrolling)
• Ionis Investigative Site — Milan, Italy (enrolling)
• Ionis Investigative Site — Amsterdam, Netherlands (enrolling)
• Ionis Investigative Site — Leeds, United Kingdom (enrolling)
• Ionis Investigative Site — Tel Aviv, Israel (enrolling)
• Ionis Investigative Site — Clermont-Ferrand, France
• Ionis Investigative Site — Le Kremlin-Bicêtre, France
• Ionis Investigative Site — Göttingen, Germany

Full record on ClinicalTrials.gov

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