RecruitingNot Applicable

Longitudinal Study of Neurodegenerative Disorders

United States1,500 participantsStarted 2012-01-11

Plain-language summary

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Who can participate

Sex

ALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Any patient with a genetic neurodegenerative disorder Exclusion Criteria: * none

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Cognitive development

Timeframe: 15 years

Language development

Timeframe: 15 years

Gross Motor development .

Timeframe: 15 years

Fine Motor development

Timeframe: 15 years

Adaptive living skills

Timeframe: 15 years

Trial details

NCT IDNCT03333200

SponsorUniversity of Pittsburgh

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2030-01

Contact for this trial

Deepa Rajan, MD

412-692-8388 rajands@upmc.edu

View on ClinicalTrials.gov More MLD trials