Interventional {{label}}

SOD1 Inhibition by Pyrimethamine in Familial ALS

Affiliated

Please note: All trial information reflects the latest data available from the sponsor on ClinicalTrials.gov and other public databases. However, these sources may occasionally be outdated or inaccurate. For the most current information, we recommend contacting the trial sponsor or sites directly.

Overview

Intervention or Drug Name

Sponsor

Trial Status

Phase

Randomization

Trial Duration

Route of Administration

Genetic Target

Repurposed Drugs

Approved by FDA

Approved outside USA

Is a supplement

Details

Enrollment Criteria

Time since symptom onset

Vital Capacity (FVC or SVC)

Approved Medications

Registry Listing

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Study Contact

Name

Phone

Email

Purpose

The objective of this study is to evaluate the safety, tolerability and effect on SOD1 levels by pyrimethamine in patients with familial amyotrophic lateral sclerosis.

What participation looks like

Our study's primary objective is to determine if familial ALS patients taking pyrimethamine will show a decline in SOD1 levels in the CSF by 15% or more. We will also determine if SOD1 and pyrimethamine are present in the blood and if the SOD-1 levels decline over the course of the study. We will also evaluate the safety and tolerability of pyrimethamine in patients with FALS. Secondary objectives will be to determine dose optimization for maximal SOD1 level reduction. We will also assess the feasibility of proceeding to phase II/III studies using pyrimethamine. Using futility methodology in which patients treated with pyrimethamine will be compared to historical controls, 40 patients with mild to moderate FALS and SOD1 mutations will receive up to 75 mg of pyrimethamine for 36 weeks. A change of 15% in the slope of decline will be deemed significant with a power of 80.7 percent. Change in ALS-FRS and quality of life will also be measured.

Eligibility criteria

18 years and older. All genders. No healthy volunteers.

Locations

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