Tolerability and Efficacy of L-Serine in Patients With Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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The purpose of this study is to determine the tolerability of L-Serine oral doses for ALS patients and assess preliminary indications of efficacy

Detailed Description
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All patients will receive the same dose of the study treatment over 6 months. For each participant the study will last approximately one year with follow up visits after the treatment period of 6 months is completed. The visits will include blood draws, vital sign checks, neurological and physical exams, pulmonary testing with forced vital capacity (FVC), and questionnaires.

Official Title
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Tolerability and Efficacy of L-Serine in Patients With Amyotrophic Lateral Sclerosis: A Phase IIa Study

Conditions
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Amyotrophic Lateral Sclerosis (ALS)

Intervention / Treatment
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* Drug: L-Serine


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of probable or definite ALS ALSFRS-R score >25 and FVC score ≥ 60% predicted If currently taking Riluzole and/or Edaravone/Radicava must be on stable dose for 3 months prior to Baseline/Screening. If the dosing has not been stable for 3 months prior to Baseline/Screening or if stopped due to an adverse event, the waiting period off the medication will be 7 days. If not on either of these medications may start if desired either or both medications after enrollment into study. Exclusion Criteria: Diagnosis of probable or definite ALS more than 3 years prior to study enrollment Diagnosis or previous history of ischemic stroke, brain tumor, uncontrolled diabetes, renal insufficiency, or severe hypertension. Diagnosis or previous history of comorbid progressive neurodegenerative disease such as Alzheimer's disease, Parkinson's disease, Lewy Body disease, Pick's disease, Huntington's disease, Progressive Supranuclear palsy. ALS patients diagnosed with frontotemporal dementia will not be excluded from this study. Diagnosis or previous history of symptomatic peripheral neuropathy. Patients with findings of peripheral neuropathy on electrodiagnostic tests only but no clinical symptoms at the time of enrollment are eligible. Undergoing any chemotherapy or radiation therapy for any cancer Any medical condition likely to interfere with the conduct of the trial or survival of the patient during this study period Pregnant women or women who are breast feeding Has taken L-Serine supplement within 30 days prior to start of study drug

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: L-Serine<br>L-Serine 15 grams orally twice a day as tolerated for 6 months | Drug: L-Serine<br>* L-Serine is a naturally occurring dietary amino acid. It is abundant in soy products, some edible seaweeds, sweet potatoes, eggs and meat.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dose tolerability based on subject reporting | Dose tolerability is based on subject interviews and diary assessment evaluating the presence or absence of adverse events | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy based on ALS Functional Rating Scale - Revised (ALSFRS-R) | Change in ALSFRS-R questionnaire scale. The ALSFRS-R provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The ALSFRS includes ten questions that ask the physician to rate his/her impression of the patients level of functional impairment in performing one of ten common tasks, e.g. climbing stairs. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 40=best. | Baseline, 1 year | 
| Efficacy based on neurological exam | Change in neurological exams with testing of muscle flexion and extension (scale: 0 to 5 with 0 being the most impaired) reflexes (scale: absent to brisk with absent being the most impaired), and sensation (scale: normal to abnormal), cranial nerves (scale: normal to abnormal on ocular movement, yes to no on Ptosis, normal to atrophy on tongue, and normal to abnormal on tongue movement). | Baseline, 6 months | 
| Efficacy based on pulmonary forced vital capacity | Change in % predicted in forced vital capacity | Baseline, 6 months |