NCT02868567

A Multicenter, 18-week Open Label Safety and Efficacy Trial of Dalfampridine in Primary Lateral Sclerosis

Active, enrolled Phase 1 Last updated 4 September 2025

What this trial tests

Phase 1 trial testing dalfampridine in Motor Neuron Disease, Upper in 35 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

1 March 2016

Primary endpoint
1 July 2026

1 July 2026

Quick facts

Lead sponsor	Hospital for Special Surgery, New York
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	35
Start date	1 March 2016
Primary completion	1 July 2026
Estimated completion	1 July 2026
Sites	3 locations across United States

Drugs / interventions tested

• dalfampridine — full drug profile →

Conditions studied

• Motor Neuron Disease, Upper — all drugs for Motor Neuron Disease, Upper →

Sponsor

Hospital for Special Surgery, New York

Who can join

Adults 18 to 99, any sex, with Motor Neuron Disease, Upper. 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.

• consistent improvement in the Timed 25 Foot Walk test
  Time frame: over the duration of the study at week 2, 4, 6, 10, 14, 18
  speed of walking 25 feet

Sponsor's own description

This study will comprise an 18-week open label safety and tolerability trial. In this study, a total of 35 subjects with primary lateral sclerosis PLS or upper motor neuron predominate ALS will be enrolled. At the initial screening evaluation, a baseline T25FW will be obtained. This baseline test will be repeated at weeks 2, 4, 6, 10, 14 18. The validity of this measure was shown in MS studies when compared to the MSWS-12 (12 item walking scale) and CGI (clinical global impression) scales (35-37). A consistent responder will be defined as improvement in 3 of 4 Timed 25Foot Walk while on medication, compared with the baseline results while off medication.

Publications & conference data

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

1. Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine.
   Chaytow H, Faller KME, Huang YT, Gillingwater TH. · · 2021 · cited 103× · PMID 34337562 · DOI 10.1016/j.xcrm.2021.100346
2. Primary Lateral Sclerosis Natural History Study: Primary Lateral Sclerosis Functional Rating Scale and Other Outcomes Assessment.
   Lee I, Jang G, Cheung YKK, Sherman AV, et al · · 2026 · cited 1× · PMID 41020440 · DOI 10.1002/ana.78056