NCT01799915 Natural History Study of Synucleinopathies
| NCT ID | NCT01799915 |
| Status | Recruiting |
| Phase | — |
| Sponsor | NYU Langone Health |
| Condition | Patients With Synucleinopathies |
| Study Type | OBSERVATIONAL |
| Enrollment | 800 participants |
| Start Date | 2011-06 |
| Primary Completion | 2026-12-30 |
Eligibility & Interventions
Eligibility Fast-Check
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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 800 participants in total. It began in 2011-06 with a primary completion date of 2026-12-30.
⚠ 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
Synucleinopathies are a group of rare diseases associated with worsening neurological deficits and the abnormal accumulation of the protein α-synuclein in the nervous system. Onset is usually in late adulthood at age 50 or older. Usually, synucleinopathies present clinically with slowness of movement, coordination difficulties or mild cognitive impairment. Development of these features indicates that abnormal alpha-synuclein deposits have destroyed key areas of the brain involved in the control of movement or cognition. Patients with synucleinopathies and signs of CNS-deficits are frequently diagnosed with Parkinson disease (PD), dementia with Lewy bodies (DLB) or multiple system atrophy (MSA). However, accumulation of alpha-synuclein and death of nerve cells can also begin outside the brain in the autonomic nerves. In such cases, syncucleinopathies present first with symptoms of autonomic impairment (unexplained constipation, urinary difficulties, and sexual dysfunction). In rare cases, hypotension on standing (a disorder known as orthostatic hypotension) may be the only clinical finding. This "pre-motor" autonomic stage suggests that the disease process may not yet have spread to the brain. After a variable period of time, but usually within 5-years, most patients with abnormally low blood pressure on standing develop cognitive or motor abnormalities. This stepwise evolution indicates that the disease spreads from the body to the brain. Another indication of this spread is that acting out dreams (i.e., REM sleep behavior disorder, RBD) a problem that occurs when the lower part of the brain is affected, may also be the first noticeable sign of Parkinson disease. The purpose of this study is to document the clinical features and biological markers of patients with synucleinopathies and better understand how these disorders evolve over time. The study will involve following patients diagnosed with a synucleinopathy (PD/DLB and MSA) and those believed to be in the "pre-motor" stage (with isolated autonomic impairment and/or RBD). Through a careful series of follow-up visits to participating Centers, we will focus on finding biological clues that predict which patients will develop motor/cognitive problems and which ones have the resilience to keep the disease at bay preventing spread to the brain. We will also define the natural history of MSA - the most aggressive of the synucleinopathies.
Eligibility Criteria
Inclusion Criteria: 1. Both male and female patients will be included 2. Aged 18 or over 3. Referred to any of the participating consortium sites with orthostatic intolerance, defined as symptoms of dizziness or lightheadedness in the standing position that disappear when supine. Exclusion Criteria: 1. Diabetes according to the American Diabetes Association criteria 2. Congestive heart failure 3. Lupus or other collagen vascular disease 4. Systemic illness thought to be responsible for the orthostatic intolerance 5. Drug-induced orthostatic hypotension (i.e., the use of alpha-blockers, diuretics, tricyclic antidepressants or others thought by the investigator to play an important role in the patient's orthostatic hypotension) 6. Isolated vasovagal syncope 7. Inability to comply with the protocol, e.g. uncooperative attitude, inability to return for follow-up visits, and unlikelihood of completing the study.
Contact & Investigator
Horacio C Kaufmann, MD
PRINCIPAL INVESTIGATOR
NYU MEDICAL CENTER
Frequently Asked Questions
Who can join the NCT01799915 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Patients With Synucleinopathies. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT01799915 currently recruiting?
Yes, NCT01799915 is actively recruiting participants. Contact the research team at horacio.kaufmann@nyulangone.org for enrollment information.
Where is the NCT01799915 trial being conducted?
This trial is being conducted at Boston, United States, Ann Arbor, United States, Rochester, United States, New York, United States and 4 additional locations.
Who is sponsoring the NCT01799915 clinical trial?
NCT01799915 is sponsored by NYU Langone Health. The principal investigator is Horacio C Kaufmann, MD at NYU MEDICAL CENTER. The trial plans to enroll 800 participants.