ALS, Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS
Brief summary
The use of Thalidomide in patients with ALS who have disease progression.
Detailed description
Phase II open labeled trial testing the efficacy of thalidomide for ALS in the setting of disease progression.
Interventions
DRUGThalidomide
Thalidomide will start at a dose of 100mg/day, dose escalated every 2 weeks by 100mg/day to a target dose of 400mg/day.
Sponsors
Celgene Corporation
Dartmouth-Hitchcock Medical Center
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Clinically proven ALS * Disease duration less than or equal to 5 years * ALSFRS-R score equal to or greater then 30
Exclusion criteria
* Patients with known deep venous thrombosis or hyper coagulable state will be excluded * Patients with FVC less than 80%
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the effect of Thalidomide in the rate of progression in ALS at 9 months and hence determine preliminary efficacy in the treatment of ALS | Efficacy will be gauged according to the slopeof the expected average decline at nine months after starting Thalidomide |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate 1) toxicity 2) quality of life 3) cytokine profile 4) PFT's 5) sleep questionnaire and 6) survival of thalidomide in the treatment of ALS | survival and progression free suvival will be assessed from the date of initial therapy on study to date od death. Survival and progresion free survival wil be analyzed using the Kaplan Meier method |
Countries
United States
Outcome results
None listed