Clinical Trial of Ultra-high Dose Methylcobalamin for ALS

What Was Tested

This registered phase 3 trial tested Cyanocobalamin (Vitamin B12).

First they are followed for 12 weeks with Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) scores, and only those who exhibit drops of 1-2 points are allowed to enter into the test period.

A total of 128 patients are randomized and the half having placebo.

It had four arms (groups): Treatment Phase: Placebo (placebo comparator), Treatment Phase: methylcobalamin (active comparator), OLE Phase: methylcobalamin (experimental) and Tsunagi Phase: methylcobalamin (experimental).

Intramuscular injection of saline solution.

Intramuscular injection of methylcobalamin.

Intramuscular injection of methylcobalamin.

The registry classes the interventions as drug.

Who Took Part

130 actual participants took part.

It was open to people of any sex aged from 20 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: ALS patients within 12 months after clinical onset at the entry. Diagnosed based on updated Awaji criteria: definite, probably or laboratory supported probable. Decline of ALSFRSR being 1 or 2 during observation period of 12 weeks. Japanese Clinical Severity Scale 1 or 2. Those who can visit the participating medical centers.

The full record lists 5 inclusion and 13 exclusion criteria; among the exclusions: those who have tracheostomy; those who had nippv; those who have chronic obstructive pulmonary disease (copd).

How the Study Was Run

The set-up was randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the Cyanocobalamin (Vitamin B12) group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Blinding on both sides kept the participants and researchers unaware of who received Cyanocobalamin (Vitamin B12).

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2017-11-01 with completion listed for 2020-02-14.

It ran at 16 sites in Japan (16).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Treatment Phase: ALSFRS-R (time frame: during 16 weeks of test period).

It also tracked 7 secondary outcomes, for example Treatment Phase: time to event, Treatment Phase: %Functional Vital Capacity (FVC), Treatment Phase: homocystein, Treatment Phase: Manual Muscle Testing (MMT), Treatment Phase: Norris scale and Treatment Phase: Grip Power.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Cyanocobalamin (Vitamin B12) in 130 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Cyanocobalamin (Vitamin B12) compound page, all human trials.