Amyotrophic Lateral Sclerosis
Conditions
Keywords
amyotrophic lateral sclerosis, pramipexole, oxidative stress, neuroprotection
Brief summary
The hypothesis of this study is that treatment with R(+) pramipexole at 30 mg/day will alter the slope of decline in ALS functional rating scale over the course of 6 months. ALS patients at an early stage of disease will be observed for 3 months after enrollment and then treated with drug for 6 months.
Detailed description
This is a futility design Phase II study using ALS-FRSr as the primary variable to monitor progression of disease in patients with early ALS. The drug to be tested is R(+) pramipexole, an antioxidant that concentrates into brain and mitochondria. R(+)PPX will be administered at 30 mg/day over 6 months, following a 3 month lead-in period without drug therapy. For purposes of this study, futility is defined as failure to decrease the slope of ALS-FRSr decline by less than 40%.
Interventions
10 mg tid oral
Sponsors
University of Pittsburgh
Bennett, James P., Jr., M.D., Ph.D.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* established diagnosis of ALS FVC\>60% of predicted not being ventilated no difficulty swallowing ambulatory (can use assistance devices)
Exclusion criteria
* ALS duration \>3 years advanced ALS with survival predicted \<6 months dementia (MMSE\<22) prior exposure to R(+) pramipexole orthostatic hypotension \>30 mmHg history of psychosis or hallucinations abnormal baseline safety lab values
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ALS-FRSr score taken each month for 3 months during lead-in and for 6 months during treatment | -3 -2 -1 0 1 2 3 4 5 6 months |
Secondary
| Measure | Time frame |
|---|---|
| hand dynamometry taken each month | -3 -2 -1 0 1 2 3 4 5 6 |
| FVC taken each month | -3 -2 -1 0 1 2 3 4 5 6 months |
Countries
United States
Outcome results
None listed