Fampridine for Optic Neuritis

(FAMP-ON Trial)

Overseen By Riya Dhillon, BA

Age: 18 - 65

Sex: Any

Trial Phase: Phase < 1

Sponsor: Courtney Casserly

Must not be taking: OCT-2 inhibitors

Disqualifiers: Diabetes, Glaucoma, Seizures, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 20

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to investigate whether Fampridine-SR can improve vision in people with multiple sclerosis (MS) who have experienced an episode of optic neuritis (ON) without full recovery. Optic neuritis often causes painful eye movements and vision decline. The trial will test if oral Fampridine-SR can aid in visual recovery. Individuals with MS who experienced an ON episode with incomplete recovery at least a year ago might be suitable for this study. As an Early Phase 1 trial, this research focuses on understanding how Fampridine-SR works in people, offering participants a chance to contribute to groundbreaking insights.

Will I have to stop taking my current medications?

The trial does not require you to stop taking your current medications, as long as there has been no change in dose within 30 days before joining the study and during the study. However, you cannot take medications that inhibit Organic Cation Transporter 2 (OCT-2).

Is there any evidence suggesting that Fampridine-SR is likely to be safe for humans?

Research has shown that fampridine is generally safe for people with multiple sclerosis (MS). Studies have found that most individuals tolerate the drug well, with no serious or long-lasting side effects reported. Common side effects include trouble sleeping and headaches, but these are usually mild to moderate.

The drug helps improve walking and other nerve functions in people with MS by blocking certain channels on damaged nerves. This suggests it might also assist with nerve problems in optic neuritis, a condition related to MS. Overall, fampridine appears safe based on current research, but discussing any concerns with a healthcare provider is always advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Fampridine-SR is unique because it targets optic neuritis by enhancing nerve signal strength. Unlike typical treatments that mainly focus on reducing inflammation, Fampridine-SR works by blocking potassium channels, which helps improve nerve conduction. Researchers are excited because this approach may not only alleviate vision problems but also enhance overall nerve function, offering a new pathway for potential improvements in patients' quality of life.

What evidence suggests that Fampridine-SR might be an effective treatment for optic neuritis?

Research has shown that Fampridine-SR, which participants in this trial will receive, might improve vision for some individuals with optic neuropathy, a condition linked to optic neuritis. Past studies found that Fampridine helps people with multiple sclerosis walk better, suggesting it might also enhance nerve function. Although strong evidence is lacking for its effectiveness on eyes affected by optic neuritis, it shows promising nerve-protecting qualities. Early results from a case of optic neuritis demonstrated improved vision after a month of using Fampridine-SR, suggesting it might benefit those who do not fully regain vision after an optic neuritis episode. ^{2 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for individuals who have had optic neuritis (ON) at least a year ago without full recovery. They should have certain visual acuity or visual evoked potential (VEP) differences, no recent corticosteroids use, and stable doses of some medications. Participants must not have seizures history (except infant febrile seizure), take OCT-2 inhibitors, or other conditions affecting vision like diabetes retinopathy.

Inclusion Criteria

You had a serious eye condition called optic neuritis more than a year ago and it didn't fully get better.

Your vision in the affected eye is better than 20/40.

Have not received corticosteroids in the last thirty (30) days

Exclusion Criteria

You have a medical condition that could affect your vision, such as diabetes-related eye problems, glaucoma, cataracts, previous eye injury, lazy eye, or non-demyelinating optic nerve issues.

Your kidneys are not working well enough, with a creatinine clearance of 80 mL/min or lower.

You have had seizures in the past, except for seizures caused by fever when you were a baby.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Fampridine-SR 10 mg orally twice daily for 8 weeks

8 weeks

Measured at baseline, week 8

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Measured at week 12

What Are the Treatments Tested in This Trial?

Interventions

• Fampridine SR

Trial Overview The study tests Fampridine-SR's effect on improving visual function after demyelinating ON based on promising results from a previous case where it helped with vision recovery. It aims to gather initial data to see if this could be an effective treatment for those who don't fully recover from acute ON.

How Is the Trial Designed?

Group I: Fampridine-SR Experimental Treatment 1 Intervention

Fampridine-SR 10 mg Orally Twice Daily for 8 weeks.

Who Is Running the Clinical Trial?

Courtney Casserly

Lead Sponsor

Trials

1

Recruited

20+

Published Research Related to This Trial

In a study of nine patients with optic neuritis treated with high-dose oral corticosteroids (methylprednisolone at 1000 mg/day for 3-5 days), significant improvements in visual acuity, contrast sensitivity, and visual field index were observed as early as day 4 after treatment began.

By day 90, visual acuity improved from an average of 40.1 letters at baseline to 80.3 letters, indicating that high-dose corticosteroids can lead to rapid and substantial recovery of visual function in optic neuritis patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Rapid improvement in visual function after high-dose oral corticosteroids in patients with inflammatory optic neuropathy]. Poujade, A., Le Page, E., Baudet, D., et al.[2017]

In a phase III trial involving 301 patients with multiple sclerosis, fampridine significantly improved walking ability, with 35% of patients showing a positive response compared to only 8% in the placebo group.

The improvement in walking speed for fampridine-treated patients was substantial at 25.2%, and this was linked to a meaningful reduction in reported ambulatory disability, indicating its efficacy as a treatment for motor function deficits in multiple sclerosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sustained-release oral fampridine in multiple sclerosis: a randomised, double-blind, controlled trial. Goodman, AD., Brown, TR., Krupp, LB., et al.[2022]

In a study of 52 patients with androgen-resistant prostate cancer, the combination of docetaxel, vinorelbine, and oral estramustine resulted in a significant prostate-specific antigen (PSA) response of 56%, indicating effective treatment.

The regimen was generally well-tolerated, with mild toxicity observed; however, 33% of patients experienced severe neutropenia, highlighting the need for monitoring blood cell counts during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Combination chemotherapy with docetaxel, vinorelbine and estramustine phosphate in metastatic androgen-resistant prostate cancer: a single institution experience. Pectasides, D., Pectasides, E., Papaxoinis, G., et al.[2018]

There has been only modest progress in finding effective treatments for multiple sclerosis, with no current treatment reliably slowing the disease's progression, although ongoing trials may yield new insights.

Azathioprine has shown strengthened benefits for MS treatment through a meta-analysis, while methylprednisolone may help with severe optic neuritis, but prednisone could increase the risk of recurrent episodes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical trials in multiple sclerosis. Noseworthy, JH.[2007]

The combination of vinorelbine (VRL) and hydrocortisone significantly improved progression-free survival (PFS) and prostate-specific antigen (PSA) response rates in patients with hormone-refractory prostate cancer compared to hydrocortisone alone, with 6-month PFS rates of 33.2% versus 22.8%.

VRL plus hydrocortisone was well tolerated among elderly patients, showing a low incidence of severe side effects, making it a safe treatment option despite not improving overall survival.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomised phase III study of intravenous vinorelbine plus hormone therapy versus hormone therapy alone in hormone-refractory prostate cancer. Abratt, RP., Brune, D., Dimopoulos, MA., et al.[2020]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3908347/

Effect of 4-aminopyridine on vision in multiple sclerosis ... This study provides Class IV evidence supporting the use of 4-AP in certain patients with optic neuropathy to improve visual function.

2. healio.com healio.com/news/neurology/20250307/fampridine-may-have-positive-impact-for-optic-neuritis-in-multiple-sclerosis

Fampridine may have positive impact for optic neuritis in ... Fampridine resulted in no significant observed difference in eyes affected by optic neuritis. Larger randomized controlled clinical trials are ...

3. withpower.com withpower.com/trial/early-phase-1-optic-neuritis-10-2019-60286

Fampridine for Optic Neuritis (FAMP-ON Trial) In a phase III trial involving 301 patients with multiple sclerosis, fampridine significantly improved walking ability, with 35% of patients showing a positive ...

4. neurology.org neurology.org/doi/10.1212/NXI.0000000000000976

Neuroprotective Properties of 4-Aminopyridine To evaluate the long-term efficacy and safety of SR-4-AP, the confirmatory ENHANCE study (10 mg twice daily in 646 MS patients) was performed.

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2211034816301948

The effect of Fampridine-SR on cognitive fatigue in a ... The primary outcome was the PASAT CF score after treatment with Fampridine-SR compared to placebo. T-tests and chi-square were used to compare demographics ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11878642/

Investigating the Safety of Fampridine in Patients with Different ... The findings of this study suggest that fampridine administration has no serious or permanent AEs in patients with different types of MS. · Of ...

7. journals.lww.com journals.lww.com/ijom/fulltext/2025/01310/investigating_the_safety_of_fampridine_in_patients.6.aspx

Investigating the Safety of Fampridine in Patients with... The drug seems to be safe and well tolerated, as the SEs were mild and transient and they were consistent with most of the previous studies focusing on this ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT02166346

Safety and Efficacy of Sustained Release Dalfampridine in ... Dalfampridine, a sustained-release potassium inhibitor has been shown to be effective in improving gait and other neurologic functions in multiple sclerosis.

9. researchgate.net researchgate.net/publication/353888380_Fampridine_improves_acute_optic_neuritis_contributing_to_a_long-lasting_recovery_of_nerve_function

Fampridine improves acute optic neuritis contributing to a ... This drug has demonstrated therapeutic effects on motor dysfunction in patients with MS, by blocking the exposed potassium channels on demyelinated axons.