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Completed

NCT Number: NCT03427086

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

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).

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Key information

Conditions

Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

American univeristy of Beirut medical center

Beirut, 1107 2020, Lebanon

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Biotin

Drug

High dose biotin

Other names: vitamin B7

Placebo oral tablet

Drug

Placebo tablet similar in shape and size to the biotin tablet

Other names: Placebo

Primary outcomes

  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    Time frame: 6 months

    Any adverse effects resulting from receiving high dose biotin in patients with amyotrophic lateral sclerosis will be recorded

Secondary outcomes

  1. Motor disability measurement

    Time frame: 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.

  2. Change in Pulmonary function test parameters ( FEV1- FVC)

    Time frame: 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.

  3. Weight changes

    Time frame: 6 months

    Changes in body weight (in kilograms) will be measured in both study arms

Sponsors and collaborators

Lead sponsor

American University of Beirut Medical Center

Other

Registry information

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Feb 9, 2018
Registry last updated
Sep 1, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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