NCT05659901 Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
| NCT ID | NCT05659901 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Ionis Pharmaceuticals, Inc. |
| Condition | Pelizaeus-Merzbacher Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 32 participants |
| Start Date | 2022-10-03 |
| Primary Completion | 2029-03 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 32 participants in total. It began in 2022-10-03 with a primary completion date of 2029-03.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05659901 clinical trial?
This trial is open to male participants only, aged 6 Months or older, up to 17 Years, studying Pelizaeus-Merzbacher Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05659901 currently recruiting?
Yes, NCT05659901 is actively recruiting participants. Contact the research team at IonisPelizaeusMerzbacherStudy@clinicaltrialmedia.com for enrollment information.
Where is the NCT05659901 trial being conducted?
This trial is being conducted at Atlanta, United States, Philadelphia, United States, Clermont-Ferrand, France, Le Kremlin-Bicêtre, France and 5 additional locations.
Who is sponsoring the NCT05659901 clinical trial?
NCT05659901 is sponsored by Ionis Pharmaceuticals, Inc.. The trial plans to enroll 32 participants.