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

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Placebo Oral Tablet; Drug: Biotin

• Registration Number: NCT03427086
• Lead Sponsor: American University of Beirut Medical Center

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2018-01-29
• Study First Submitted: 2018-01-29
• Study First Submitted QC: 2018-02-02
• Study First Posted: 2018-02-09
• Primary Completion: 2021-05-10
• Study Completion: 2021-05-10
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-27
• Last Update Posted: 2021-09-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo Oral Tablet	Patients will receive placebo
Interventional	Biotin	Patient will received high dose of biotin (300 mg/day)

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.
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.
Weight changes	6 months	Changes in body weight (in kilograms) will be measured in both study arms

Trial Locations

Locations (1)

American univeristy of Beirut medical center; 🇱🇧 Beirut, Lebanon