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Amyotrophic Lateral SclerosisPhase 2NCT03427086

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

A Phase 2 study testing Biotin in people with Amyotrophic Lateral Sclerosis, run by American University of Beirut Medical Center at 1 site in Lebanon.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Biotin
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
12 participants
Where
1 siteLebanon
Study dates
29 Jan 2018to 10 May 2021
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)

Who cannot join

  • 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 locations and contacts

1 site in 1 country.

Lebanon1 site

  • American univeristy of Beirut medical center

    Beirut, 1107 2020

Other trials in Lebanon

About this study

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).

Being tested
Biotin, Placebo Oral Tablet
Keywords
Amyotrophic lateral sclerosis, Biotin
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BiotinDrug

    Also called vitamin B7

    High dose biotin

  • Placebo Oral TabletDrug

    Also called Placebo

    Placebo tablet similar in shape and size to the biotin tablet

Study groups in NCT03427086
Study groupRoleWhat they receive
InterventionalActive comparatorPatient will received high dose of biotin (300 mg/day)
Receives: Drug: Biotin
PlaceboPlacebo comparatorPatients will receive placebo
Receives: Drug: Placebo Oral Tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

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

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

    Measured: 6 months

Secondary outcomes (3)

  1. Motor disability measurement

    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.

    Measured: 6 months

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

    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.

    Measured: 6 months

  3. Weight changes

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

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Feb 2018
  2. Study start29 Jan 2018
  3. Primary completion10 May 2021
  4. Study completion10 May 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Prospective, double blind, placebo control, randomized 2:1 study
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03427086
Sponsor's protocol ID
BIO-2017-0270
Status last verified
Aug 2021
First submitted
29 Jan 2018
Last updated
1 Sep 2021
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Kawamata H, Manfredi G. Mitochondrial dysfunction and intracellular calcium dysregulation in ALS. Mech Ageing Dev. 2010 Jul-Aug;131(7-8):517-26. doi: 10.1016/j.mad.2010.05.003. Epub 2010 May 20.

    PubMed 20493207

  • Wijesekera LC, Leigh PN. Amyotrophic lateral sclerosis. Orphanet J Rare Dis. 2009 Feb 3;4:3. doi: 10.1186/1750-1172-4-3.

    PubMed 19192301

  • Musaro A. Understanding ALS: new therapeutic approaches. FEBS J. 2013 Sep;280(17):4315-22. doi: 10.1111/febs.12087. Epub 2013 Jan 3.

    PubMed 23217177

  • Zimmermann KC, Bonzon C, Green DR. The machinery of programmed cell death. Pharmacol Ther. 2001 Oct;92(1):57-70. doi: 10.1016/s0163-7258(01)00159-0.

    PubMed 11750036

  • Celsi F, Pizzo P, Brini M, Leo S, Fotino C, Pinton P, Rizzuto R. Mitochondria, calcium and cell death: a deadly triad in neurodegeneration. Biochim Biophys Acta. 2009 May;1787(5):335-44. doi: 10.1016/j.bbabio.2009.02.021. Epub 2009 Mar 4.

    PubMed 19268425

  • Atamna H, Newberry J, Erlitzki R, Schultz CS, Ames BN. Biotin deficiency inhibits heme synthesis and impairs mitochondria in human lung fibroblasts. J Nutr. 2007 Jan;137(1):25-30. doi: 10.1093/jn/137.1.25.

    PubMed 17182796

  • Stys PK, Zamponi GW, van Minnen J, Geurts JJ. Will the real multiple sclerosis please stand up? Nat Rev Neurosci. 2012 Jun 20;13(7):507-14. doi: 10.1038/nrn3275.

    PubMed 22714021

  • Luessi F, Siffrin V, Zipp F. Neurodegeneration in multiple sclerosis: novel treatment strategies. Expert Rev Neurother. 2012 Sep;12(9):1061-76; quiz 1077. doi: 10.1586/ern.12.59.

    PubMed 23039386

  • Sedel F, Papeix C, Bellanger A, Touitou V, Lebrun-Frenay C, Galanaud D, Gout O, Lyon-Caen O, Tourbah A. High doses of biotin in chronic progressive multiple sclerosis: a pilot study. Mult Scler Relat Disord. 2015 Mar;4(2):159-69. doi: 10.1016/j.msard.2015.01.005. Epub 2015 Jan 24.

    PubMed 25787192

  • Tourbah A, Lebrun-Frenay C, Edan G, Clanet M, Papeix C, Vukusic S, De Seze J, Debouverie M, Gout O, Clavelou P, Defer G, Laplaud DA, Moreau T, Labauge P, Brochet B, Sedel F, Pelletier J; MS-SPI study group. MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study. Mult Scler. 2016 Nov;22(13):1719-1731. doi: 10.1177/1352458516667568. Epub 2016 Sep 1.

    PubMed 27589059

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biotin in people with Amyotrophic Lateral Sclerosis, run by American University of Beirut Medical Center at 1 site in Lebanon.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 1 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Lebanon. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03427086, which also lists 10 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2021.

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