Clinical Trial of Ultra-high Dose Methylcobalamin for ALS (JETALS)

Eisai

Status and phase: Completed; Phase 3

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: saline solution; Drug: methylcobalamin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03548311

763

Details and patient eligibility

About

To examine the clinical efficacy and safety of ultra-high dose (50mg, im, twice a week) methylcobalamin in retarding the progression of symptoms in amyotrophic lateral sclerosis (ALS) patients, we enroll ALS patients diagnosed by Updated Awaji Criteria within 12 months after the clinical onset. 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. They are blindly evaluated for drops of ALSFRS-R in 16 weeks, as the primary outcome. After this, all subjects receive methylcobalamin.

Enrollment: 130 patients

Sex: All

Ages: 20+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Treatment and OLE Phase:

• 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

Tsunagi Phase:

• The subjects for the Tsunagi phase are patients with ALS who are continuing the administration of this drug at the time of approval, and have obtained consent for the transition to the Tsunagi phase.

Exclusion criteria

Treatment and OLE Phase:

• Those who have tracheostomy
• Those who had NIPPV
• %FVC<60%
• Those who have Chronic Obstructive Pulmonary Disease (COPD)
• Those who have symptoms and signs of B12 deficiency
• Those who had edaravone less than 4 weeks prior to entry
• Those who changed the schedule and dosing of riluzole
• Those who have dementia
• Those who have the possibility of pregnancy
• Those who have serious respiratory or cardiac diseases
• Those who have malignancies
• Those who participated other clinical trials within 12 weeks
• Those who have allergies to B12 and related compounds

Tsunagi Phase:

Not applicable.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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130 participants in 4 patient groups, including a placebo group

Treatment Phase: Placebo

Placebo Comparator group

Description:

intramuscular injection of saline solution

Treatment:

Drug: saline solution

Treatment Phase: methylcobalamin

Active Comparator group

Description:

intramuscular injection of methylcobalamin

Treatment:

Drug: methylcobalamin

OLE Phase: methylcobalamin

Experimental group

Description:

intramuscular injection of methylcobalamin

Treatment:

Drug: methylcobalamin

Tsunagi Phase: methylcobalamin

Experimental group

Description:

intramuscular injection of methylcobalamin

Treatment:

Drug: methylcobalamin