Amyotrophic Lateral Sclerosis
Conditions
Keywords
Amyotrophic lateral sclerosis, Biotin
Brief summary
This is a randomized double blinded randomized 2:1 study. The duration of the study is 6 month. The safety and tolerability of high doses of biotin (300 mg/ day) will be compared to placebo in patients with amyotrophic lateral sclerosis. Patients will be evaluated at baseline, 3, and 6 month. The primary outcome will be any adverse effects recorded. The secondary outcomes will be motor disability measured by ALS-FRS, change in Pulmonary function test parameters (FEV1- FVC), change in subject weight (in kg).
Interventions
DRUGBiotin
High dose biotin
DRUGPlacebo Oral Tablet
Placebo tablet similar in shape and size to the biotin tablet
Sponsors
American University of Beirut Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
The subjects, care givers, investigator, and coordinator will be blinded. The pharmacist, who will be responsible for the drug supply, will be unblinded. The investigational drug and the placebo will have identical pill shape and color. They will be supplied in identical boxes.
Intervention model description
Prospective, double blind, placebo control, randomized 2:1 study
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Amyotrophic Lateral Sclerosis (ALS) volunteers must be diagnosed within 3 years prior to participation as having possible, probable, or definite ALS, either sporadic or familial according to modified El Escorial criteria * Age 18-80, able to provide informed consent, and comply with study procedures * Participants must not have started Riluzole and/or Nuedexta for at least 30 days, or be on a stable dose of Riluzole and/or Nuedexta for at least 30 days, prior to screening (Riluzole and/or Nuedexta -naïve participants are permitted in the study)
Exclusion criteria
* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to PI judgment. * Exposure to any experimental agent within 30 days of entry or at any time during the trial or enrollment in another research study within 30 days of or during this trial. * Slow Vital Capacity test less than 50% of the predicted value Patients who had already undergone tracheostomy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | 6 months | Any adverse effects resulting from receiving high dose biotin in patients with amyotrophic lateral sclerosis will be recorded |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Motor disability measurement | 6 months | The motor disability will be measured in the both arms using the revised amyotrophic lateral sclerosis functional rating scale (ALS-FRSr). This scale measures the progression and the severity of the disease. It is compose of 12 questions, each questions can have a score from 0 to 4. Questions 1 to 3 are related to bulbar onset, questions 4 to 9 are related to limb onset and questions 10-12 are related to respiratory onset. The minimum score is 0 and the maximum total score is 48. The higher the score the better the functional status. The lower the score the worse the functional status of the patient. |
| Change in Pulmonary function test parameters ( FEV1- FVC) | 6 months | Forced expiratory volume in 1 second (FEV1) measured in percents and forced vital capacity (FVC) measured in liters will be measured in the both study arms. |
| Weight changes | 6 months | Changes in body weight (in kilograms) will be measured in both study arms |
Countries
Lebanon
Outcome results
None listed