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

• Register: ClinicalTrials.gov
• Last refreshed on: 7 September 2021
• Main ID: NCT03427086
• Date of registration: 29/01/2018
• Prospective Registration: Yes
• Primary sponsor: American University of Beirut Medical Center
• Public title: Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
• Scientific title: Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
• Date of first enrolment: January 29, 2018
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03427086
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Lebanon

Contacts

• Name: Achraf Makki, MD
• Affiliation: American University of Beirut Medical Center

• Name: Johnny Salameh, MD
• Affiliation: American University of Beirut Medical Center

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Biotin

Drug: Placebo Oral Tablet

Primary Outcome(s)

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [Time Frame: 6 months]

Secondary Outcome(s)

Motor disability measurement [Time Frame: 6 months]

Change in Pulmonary function test parameters ( FEV1- FVC) [Time Frame: 6 months]

Weight changes [Time Frame: 6 months]

Secondary ID(s)

BIO-2017-0270

Source(s) of Monetary Support

Please refer to primary and secondary sponsors