Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis

American University of Beirut Medical Center

Status and phase: Completed; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo Oral Tablet; Drug: Biotin

Study type: Interventional

Funder types: Other

Identifiers

NCT03427086

BIO-2017-0270

Details and patient eligibility

About

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).

Enrollment: 12 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

12 participants in 2 patient groups, including a placebo group

Interventional

Active Comparator group

Description:

Patient will received high dose of biotin (300 mg/day)

Treatment:

Drug: Biotin

Placebo

Placebo Comparator group

Description:

Patients will receive placebo

Treatment:

Drug: Placebo Oral Tablet