Have you or your loved ones been diagnosed with ald?

You may be eligible to participate in a ald clinical trial.

Have you or your loved ones been diagnosed with ald? You may be eligible to participate in a ald clinical trial.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Recruiting for this study? Get access to patient recruitment tools.

ALD Clinical Trial in Pittsburgh PA

NCT03333200 | Observational

Have you or your loved ones been diagnosed with ald?

You may be eligible to participate in a ald clinical trial.

Have you or your loved ones been diagnosed with ald? You may be eligible to participate in a ald clinical trial.

Recruiting

Male & Female

All ages

This study is looking to recruit 1500 Participants

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.

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Where you'll go

Selected location

The Program for the Study of Neurodevelopment in Rare Disorders Children's Hospital of Pittsburgh of UPMC Change

4401 Penn Avenue, Pittsburgh, PA, USA 15224

Jodi Martin 412-692-6351
sausjl@upmc.edu

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Details for the study

Population

Persons with genetic neurodegenerative diseases.

Brief Title

Longitudinal Study of Neurodegenerative Disorders

Official Title

Longitudinal Study of Neurodegenerative Disorders

Brief Summary

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

Detailed Description

Patients would be evaluated by a multidisciplinary team at specific time points every 3
months the first year, every 6 months the second year and once a year thereafter.

Treatments and/or Procedures

Hematopoetic stem cell transplantation

Following patients who have received HSCT as part of their clinical care.

Palliative care

Collecting information about the natural progression of these diseases

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Primary

Adaptive living skills