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NCT05659901 · ClinicalTrials.gov registry record
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
A clinical trial of Pelizaeus-Merzbacher Disease, sponsored by Ionis Pharmaceuticals.
- Recruiting
- Registry status
- 32
- Enrollment target
- 7
- Study locations
NCT05659901: Recruiting study of Pelizaeus-Merzbacher Disease, sponsored by Ionis Pharmaceuticals.
NCT05659901 is a study of Pelizaeus-Merzbacher Disease that is actively recruiting participants, run by Ionis Pharmaceuticals. The registered enrollment target is 32 participants. The trial reports 7 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05659901, a study of Pelizaeus-Merzbacher Disease, is actively recruiting participants, sponsored by Ionis Pharmaceuticals.
- RECRUITING
- Registry status
- 32 participants
- Enrollment target
- 7
- Study locations
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.
Primary Outcome
Changes in Proteolipid Protein 1 (PLP1) in CSF and disease related biomarkers
Conditions Studied
Study Locations (7)
Other
- Ionis Investigative Site - Clermont-Ferrand
- Ionis Investigative Site - Le Kremlin-Bicêtre
- Ionis Investigative Site - Göttingen
- Ionis Investigative Site - Tel Aviv
- Ionis Investigative Site - Milan
- Ionis Investigative Site - Amsterdam
Pennsylvania
- Ionis Investigative Site - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2022-10-03 |
| Est. Completion | 2029-03 |
What NCT05659901 shows while recruiting
NCT05659901 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 32 participants, a relatively small participant target.
The record links to 1 condition, with Pelizaeus-Merzbacher Disease appearing as the primary indexed condition, and to 0 interventions.
NCT05659901 lists 7 locations in 2 states (Other, Pennsylvania).
Frequently Asked Questions
What is clinical trial NCT05659901 about?
NCT05659901 is a clinical study titled "Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease". 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 cli...
What is the current status of trial NCT05659901?
This trial is currently recruiting. The enrollment target is 32 participants. The study started on 2022-10-03. Estimated completion is 2029-03.
What conditions does trial NCT05659901 study?
This clinical trial studies the following conditions: Pelizaeus-Merzbacher Disease.
Who is sponsoring clinical trial NCT05659901?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05659901 being conducted?
This trial has 7 study locations across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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