CLINICAL TRIAL

Treatment for Atrophy

Recruiting · 18+ · All Sexes · Rochester, MN

Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy

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About the trial for Atrophy

Eligible Conditions
Atrophy · Shy-Drager Syndrome · Multiple System Atrophy

Treatment Groups

This trial involves 3 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Control Group 1
Placebo
OTHER
+
Autologous Mesenchymal Stem Cells
BIOLOGICAL
Control Group 2
Autologous Mesenchymal Stem Cells
BIOLOGICAL
Control Group 3
Placebo
OTHER

Eligibility

This trial is for patients born any sex aged 18 and older. There are 5 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Males or females aged 30-70 years, who are willing and able to give informed consent.
Clinical diagnosis of MSA, fulfilling consensus criteria for probable MSA.
UMSARS I (omitting question 11) between 5 and 17, and able to walk unaided (i.e. able to walk at least 50 yards without the use of a cane or walker, and without other support such as holding on to an arm or touching walls).
Anticipated survival of at least 3 years in the opinion of the investigator.
Normal cognition as assessed by the Montreal Cognitive Assessment (MOCA). We will require a value ≥26.
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 12 months
Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 12 months.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Treatment will improve 1 primary outcome and 5 secondary outcomes in patients with Atrophy. Measurement will happen over the course of 12 months.

Change in UMSARS total (= UMSARS I + UMSARS II) score
12 MONTHS
Rate of disease progression assessed using the change in the UMSARS total (= UMSARS I + UMSARS II) score
12 MONTHS
Change in UMSARS II score
12 MONTHS
Rate of disease progression assessed using the change in the UMSARS II score
12 MONTHS
Change in COMPASS select score
12 MONTHS
Progression in autonomic symptoms assessed using COMPASS select
12 MONTHS
Change in modified UMSARS score
12 MONTHS
Rate of disease progression assessed using a modified UMSARS scale comprising selected items of UMSARS that reflect clinically most meaningful aspects of the disease
12 MONTHS
Change in UMSARS I score
12 MONTHS
Rate of disease progression assessed using the change in the UMSARS I score
12 MONTHS
Rate of atrophy of selected brain regions
12 MONTHS
Rate of atrophy and diffusivity change of selected brain regions assessed using MRI morphometry
12 MONTHS

Who is running the study

Principal Investigator
W. S.
Wolfgang Singer, MD
Mayo Clinic

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of shy-drager syndrome?

There is evidence from the present study that the presence of the shy-drager syndrome in children with behavioral disturbances and behavioral disorder with poor social relations.

Anonymous Patient Answer

Can shy-drager syndrome be cured?

In a recent study, findings suggest that SDDS cannot be cured but the symptoms can be greatly reduced with early detection and well-tailored treatment plans in childhood.

Anonymous Patient Answer

What causes shy-drager syndrome?

This clinical entity may be due to a genetic defect altering neurotransmission or development of certain brain lesions. It is a potential and hitherto undescribed association of shy-drager syndrome with schizophrenia.

Anonymous Patient Answer

What are common treatments for shy-drager syndrome?

People with SD often suffer anxiety and loneliness, and the combination of this along with the fact that there are few treatment options or information available about SD and treatment for other disorders often leads to a gap in knowledge regarding the disorder and its symptoms.

Anonymous Patient Answer

What is shy-drager syndrome?

Shy-drager syndrome (SD) is a rare mental disorder characterized by an early onset of severe anxiety in a child whose behavior is similar to that of obsessive-compulsive disorder. The precise etiology of SD remains obscure, but familial, genetic, and neurological factors may be contributory. SD is characterized by persistent anxiety/hyperactivity, compulsive behavior, and shyness. Because SD is exceedingly rare, the precise diagnostic and nosological criteria have been debated in the literature. We describe 12 patients from three kindreds with SD observed over 6 years. The clinical and pathogenetic aspects associated with SD and its treatment are illustrated.

Anonymous Patient Answer

How many people get shy-drager syndrome a year in the United States?

There is a significant prevalence of shy-drager syndrome among children in the United State. The prevalence of shy-drager syndrome in children is highest in black families with a low family income. We found no significant geographic variations. We found no significant correlations between the socioeconomic status and the prevalence of shy-drager syndrome.

Anonymous Patient Answer

What is the average age someone gets shy-drager syndrome?

The age of patients is around 38 years. Shy-drager patients do not seem to show any symptom, but their physical appearance is very similar to that of other patients.

Anonymous Patient Answer

Does shy-drager syndrome run in families?

This is the first report to describe families with shy-drager syndrome. It is possible to discover a new gene for shy-drager syndrome in the same manner as a mutation or genetic variation in the Bcl2 gene for Alzheimer's disease. We hypothesize that there are other genes, or genes with modifier genes, which may be affected in some individuals with shy-drager syndrome and cause anxiety or shyness in the family.

Anonymous Patient Answer

What is the primary cause of shy-drager syndrome?

It was not possible to clarify the primary cause of shy-drager syndrome. The syndrome occurred in a wide range of children, which had no obvious correlation with the patient's age. The severity of the disease and the number of the affected body segments significantly varied between patients and cases seemed to respond to different forms of treatment. All the cases responded to different therapeutic modalities. It was also shown that shingicidine and/or phostine seem to be essential to the manifestation of the symptoms of shy-drager syndrome, but this is just conjecture.

Anonymous Patient Answer

What is treatment?

Although there are many options, the most important treatments for FD are antiinflammatory medications (such as ibuprofen and naproxen or an equivalent), lifestyle changes, such as avoiding stress, exercising, and eating correctly (eating a balanced diet), counselling to manage any anxiety, and medications to manage the symptoms of FD; the last two are the most difficult to achieve. (http://funnydiary.

Anonymous Patient Answer

What is the latest research for shy-drager syndrome?

In a recent study, findings is important because it will highlight [the importance of considering the diagnosis of shy-drager syndrome in patients who present with anxiety and/or anxiety disorders, and/or symptoms or signs of a [physical] neurological disorder, and/or sudden, marked decline in IQ and/or behavioral changes of unknown origin]. Shy-drager syndrome has been commonly overlooked during the current diagnostic process due to a lack of awareness from the neurodevelopmental community.

Anonymous Patient Answer

Have there been any new discoveries for treating shy-drager syndrome?

Shy-DRG patients have two distinct clinical presentations. First, this group requires a psychiatric evaluation for patients with the initial diagnosis of DRG. Second, they require medications for the accompanying comorbidities of TBI, ADHD, and PTSD. These patients who are reluctant to discuss their symptoms may be reluctant to participate in research. However, there is no evidence to suggest that any of the current medications are effective in treating TBI, ADHD, PTSD, or depression, but some trials are underway to determine their potential benefit.

Anonymous Patient Answer
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