NCT00444613

A Phase II/III Study in Patients With Amyotrophic Lateral Sclerosis (ALS)

Completed Phase 2/Phase 3 Last updated 16 June 2023

What this trial tests

Phase 2/Phase 3 trial testing E0302 (mecobalamin) in Amyotrophic Lateral Sclerosis (ALS) in 373 participants. Completed in 1 July 2014.

Timeline

1 April 2007

Primary endpoint
1 March 2014

1 July 2014

Quick facts

Lead sponsor	Eisai Co., Ltd.
Phase	Phase 2/Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	373
Start date	1 April 2007
Primary completion	1 March 2014
Estimated completion	1 July 2014
Sites	44 locations across Japan

Drugs / interventions tested

• E0302 (mecobalamin) — full drug profile →
• E0302 (mecobalamin) — full drug profile →
• Placebo

Conditions studied

• Amyotrophic Lateral Sclerosis (ALS) — all drugs for Amyotrophic Lateral Sclerosis (ALS) →

Sponsor

Eisai Co., Ltd. — full company profile →

Who can join

20 and older, any sex, with Amyotrophic Lateral Sclerosis (ALS). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Survival rate
  Time frame: Every 3 months.
• Functional rating scale.
  Time frame: Every 3 months.

Sponsor's own description

The purpose of this study is to investigate the efficacy and confirm the safety of E0302 in patients with Amyotrophic Lateral Sclerosis (ALS) by assessing changes in scores of survival rate and functional rating scale.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Ultra-high-dose methylcobalamin in amyotrophic lateral sclerosis: a long-term phase II/III randomised controlled study.
   Kaji R, Imai T, Iwasaki Y, Okamoto K, et al · · 2019 · cited 63× · PMID 30636701 · DOI 10.1136/jnnp-2018-319294
2. Amyotrophic lateral sclerosis: update and new developments.
   Pratt AJ, Getzoff ED, Perry JJ. · · 2012 · cited 56× · PMID 23019386 · DOI 10.2147/dnnd.s19803
3. A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed?
   Xu X, Shen D, Gao Y, Zhou Q, et al · · 2021 · cited 49× · PMID 34372914 · DOI 10.1186/s40035-021-00250-5
4. Exploring targets and therapies for amyotrophic lateral sclerosis: current insights into dietary interventions.
   Ngo ST, Mi JD, Henderson RD, McCombe PA, et al · · 2017 · cited 22× · PMID 30050381 · DOI 10.2147/dnnd.s120607
5. ABC transporter-driven pharmacoresistance in Amyotrophic Lateral Sclerosis.
   Jablonski M, Miller DS, Pasinelli P, Trotti D. · · 2015 · cited 20× · PMID 25175835 · DOI 10.1016/j.brainres.2014.08.060
6. The Ying and Yang of Hydrogen Sulfide as a Paracrine/Autocrine Agent in Neurodegeneration: Focus on Amyotrophic Lateral Sclerosis.
   Spalloni A, de Stefano S, Gimenez J, Greco V, et al · · 2023 · cited 3× · PMID 37443723 · DOI 10.3390/cells12131691
7. Drug repurposing candidates for amyotrophic lateral sclerosis using common and rare genetic variants.
   Gerring ZF, Bhalala OG, Fearnley LG, Oikari LE, et al · · 2025 · cited 2× · PMID 40395632 · DOI 10.1093/braincomms/fcaf184