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

20 Participants Needed

Trial Summary

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).

What data supports the effectiveness of the drug Fampridine SR for treating optic neuritis?

Research suggests that 4-Aminopyridine, a component similar to Fampridine, may help with symptoms in multiple sclerosis, which sometimes includes optic neuritis. However, there is no direct evidence for Fampridine SR's effectiveness specifically for optic neuritis. ^{1 2 3 4 5}

Is fampridine safe for humans?

Fampridine has been generally well tolerated in clinical studies for multiple sclerosis, with mild to moderate side effects like insomnia, headache, and dizziness. It is important to avoid exceeding the recommended dose and to monitor kidney function, as the main risks include seizures and issues with kidney function. ^{6 7 8 9 10}

How does the drug Fampridine SR differ from other treatments for optic neuritis?

Fampridine SR is unique because it is primarily used to improve walking in patients with multiple sclerosis by blocking potassium channels, which may help nerve signals travel more effectively. This mechanism is different from typical treatments for optic neuritis, which often focus on reducing inflammation. ^{11 12 13 14 15}

What is the purpose of this trial?

This trial is testing a medication called Fampridine-SR on MS patients who haven't fully recovered their vision after optic neuritis. The goal is to see if it can improve their vision by helping nerves send signals better. Fampridine-SR is primarily used to improve walking ability in MS patients.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Clinical trials in multiple sclerosis. [2007]

2. Thailand pubmed.ncbi.nlm.nih.gov

Optic neuritis: characteristics and visual outcome. [2013]

3. United States pubmed.ncbi.nlm.nih.gov

Does time equal vision in the acute treatment of a cohort of AQP4 and MOG optic neuritis? [2023]

4. United States pubmed.ncbi.nlm.nih.gov

A double-blind, randomized trial of IV immunoglobulin treatment in acute optic neuritis. [2014]

5. France pubmed.ncbi.nlm.nih.gov

[Rapid improvement in visual function after high-dose oral corticosteroids in patients with inflammatory optic neuropathy]. [2017]

6. United States pubmed.ncbi.nlm.nih.gov

Sustained-release fampridine for symptomatic treatment of multiple sclerosis. [2013]

7. England pubmed.ncbi.nlm.nih.gov

Prolonged-release fampridine improves walking in a proportion of patients with multiple sclerosis. [2013]

8. United States pubmed.ncbi.nlm.nih.gov

Steady-state pharmacokinetics and tolerability of orally administered fampridine sustained-release 10-mg tablets in patients with multiple sclerosis: a 2-week, open-label, follow-up study. [2013]

9. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of fampridine for walking disability in multiple sclerosis. [2021]

10. England pubmed.ncbi.nlm.nih.gov

Sustained-release oral fampridine in multiple sclerosis: a randomised, double-blind, controlled trial. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Phase I clinical and pharmacologic trial of intravenous estramustine phosphate. [2017]

12. Greece pubmed.ncbi.nlm.nih.gov

Combination chemotherapy with docetaxel, vinorelbine and estramustine phosphate in metastatic androgen-resistant prostate cancer: a single institution experience. [2018]

13. Japan pubmed.ncbi.nlm.nih.gov

[Low-Dose Estramustine Phosphate Monotherapy in Castration-Resistant Prostate Cancer Patients]. [2018]

14. England pubmed.ncbi.nlm.nih.gov

Randomised phase III study of intravenous vinorelbine plus hormone therapy versus hormone therapy alone in hormone-refractory prostate cancer. [2020]

15. United States pubmed.ncbi.nlm.nih.gov

The Contemporary Use of Radium-223 in Metastatic Castration-resistant Prostate Cancer. [2019]