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

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ClinicalTrials.gov Identifier: NCT03427086
Recruitment Status : Active, not recruiting
First Posted : February 9, 2018
Last Update Posted : February 9, 2018
Sponsor:
Information provided by (Responsible Party):
Johnny Salameh, 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).

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Biotin Drug: Placebo Oral Tablet Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Prospective, double blind, placebo control, randomized 2:1 study
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: The subjects, care givers, investigator, and coordinator will be blinded. The pharmacist, who will be responsible for the drug supply, will be unblinded. The investigational drug and the placebo will have identical pill shape and color. They will be supplied in identical boxes.
Primary Purpose: Treatment
Official Title: Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
Actual Study Start Date : January 29, 2018
Estimated Primary Completion Date : October 10, 2018
Estimated Study Completion Date : December 10, 2018


Arm Intervention/treatment
Active Comparator: Interventional
Patient will received high dose of biotin (300 mg/day)
Drug: Biotin
High dose biotin

Placebo Comparator: Placebo
Patients will receive placebo
Drug: Placebo Oral Tablet
Placebo tablet similar in shape and size to the biotin tablet




Primary Outcome Measures :
  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 Outcome Measures :
  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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03427086


Locations
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Lebanon
American univeristy of Beirut medical center
Beirut, Lebanon, 1107 2020
Sponsors and Collaborators
American University of Beirut Medical Center
Investigators
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Principal Investigator: Achraf Makki, MD American University of Beirut Medical Center

Additional Information:
Publications of Results:

Other Publications:
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Responsible Party: Johnny Salameh, MD, American University of Beirut Medical Center
ClinicalTrials.gov Identifier: NCT03427086     History of Changes
Other Study ID Numbers: BIO-2017-0270
First Posted: February 9, 2018    Key Record Dates
Last Update Posted: February 9, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Johnny Salameh, American University of Beirut Medical Center:
Amyotrophic lateral sclerosis
Biotin

Additional relevant MeSH terms:
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Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Biotin
Vitamin B Complex
Vitamins
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs