NCT04292080

Long-Term Analysis of DImethyl Fumarate, to Slow the Growth of Areas of Geographic Atrophy

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 55 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The primary objectives of the study are to assess the safety, tolerability and evidence of activity of 12 months oral treatment with TEFIDERA® in subjects with Geographic Atrophy associated with Age-Related Macular Degeneration (AMD).

Detailed description

Age-related Macular Degeneration (AMD) is the leading cause of irreversible blindness in the industrialized world. Approximately 80% of patients with AMD suffer from the dry form of the disease also called geographic atrophy (GA) which is characterized by the progressive loss of photoreceptors and RPE that are essential to the visual cycle. There is currently no therapy to cure GA. This represent a major health problem with millions patients worldwide. GA is a complex multifactorial disease influenced by: aging, environmental factors, genetic predispositions, oxidative stress and inflammation. Oxidative stress is the major environmental risk factor of developing AMD. Whereas inflammation, is now, recognized as one of the main player in AMD pathophysiology. Therefore, antioxidant and immunosuppressive therapies are susceptible to be beneficial in humans for patients with GA. Dimethyl Fumarate activates the NRF2 pathway, which is the major transcription factor regulating anti-oxidative enzymes production. Dimethyl Fumarate commercialized under the name TECFIDERA® by BIOGEN is an effective antioxidant and immunosuppressive drug, used to treat patients with Multiple Sclerosis (MS), an autoimmune disease of the Central Nervous System leading to progressive disability. This new therapeutic agent may be easily repurposed to treat GA patients and could reduce or slow photoreceptors and RPE degeneration and the associated vision loss. In this randomized, open labelled trial we will evaluate the safety and efficacy of TECFIDERA®, in two groups of patients (treated vs. untreated) with GA. This is the first time this strategy, based on evidences that oxidative stress and inflammation are central in GA physiopathology, will be tested in patients with dry AMD.

Conditions

• Age-related Macular Degeneration (AMD)

Interventions

Type	Name	Description
DRUG	Dimethyl Fumarate (TECFIDERA™)	Dimethyl fumarate is a white to off-white powder that is highly soluble in water with a molecular mass of 144.13, containing 120 mg or 240 mg of dimethyl fumarate The starting dose for TECFIDERA is 120 mg twice a day orally. After 7 days, the dose should be increased to the maintenance dose of 240 mg twice a day orally. Body weight, gender, and age do not require dosage adjustment.
OTHER	No active : no treatment	Patients will receive no specific treatment (standard of care) up to 24 months following randomization.

Timeline

• Start date: 2022-02-07
• Primary completion: 2027-04-07
• Completion: 2029-04-07
• First posted: 2020-03-02
• Last updated: 2026-02-04

Locations

1 site across 1 country: France