Clinical trial NCT03427086: Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
Official title: Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic lateral sclerosis
Biotin
Lead sponsor: American University of Beirut Medical Center
Sponsor class: OTHER
First posted: 2018-02-09
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Prospective, double blind, placebo control, randomized 2:1 study
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
timeFrame: 6 months
Secondary outcomes:
measure: Motor disability measurement
timeFrame: 6 months
measure: Change in Pulmonary function test parameters ( FEV1- FVC)
timeFrame: 6 months
measure: Weight changes
timeFrame: 6 months
Other outcomes:
Locations:
city: Beirut
country: Lebanon
facility: American univeristy of Beirut medical center
Study officials:
affiliation: American University of Beirut Medical Center
name: Achraf Makki, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: American University of Beirut Medical Center
name: Johnny Salameh, MD
role: PRINCIPAL_INVESTIGATOR
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