Clinical trial NCT03324399: A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With ALS
Official title: A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Amyotrophic Lateral Sclerosis
ALS
Keywords: ALS
Amyotrophic Lateral Sclerosis
Lead sponsor: Avera McKennan Hospital & University Health Center
Sponsor class: OTHER
First posted: 2017-10-27
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: ALS, also known as "Lou Gehrig's" disease, is a neurodegenerative disease which is fatal. Treatment for ALS is limited and currently consists of primary symptom relief or support. In addition, time from diagnosis to death averages 3-5 years. New Biotic, LLC has submitted an Orphan Drug Designation Application for an investigational probiotic and have indicated the need for more study of this orphaned drug in ALS patients.

Detailed description: This will be an exploratory protocol of 10 patients and expect to screen approximately 25-30 patients.

The purpose of our study is to examine amino acid levels in plasma pre and post prandial as well as longitudinal gut microbiome in ALS patients taking the investigational probiotic.

1. Purpose: Evaluate the clinical outcomes of muscle strength, clinical global assessments and spasticity in patients taking an Investigational probiotic.
2. Measure changes in questionnaires relating to subjective functional assessments, quality of life, and pain levels over 24 weeks.
3. Describe plasma amino acid levels before and after a protein food challenge in ALS patients of spinal and bulbar type.
4. Evaluate and describe the gut microbiome of patients with ALS.

Design:

This is a non-randomized, open-label, two-group pilot study of plasma amino acid levels, gut microbiome, and of investigational probiotic use in patients with ALS. Participants will be placed into two groups based on certain post-prandial amino acid levels. Patients will be stratified for eligibility based on the results of their amino acid profiles.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of sporadic ALS, definite or probable disease,-revised El Escorial criteria
* Patient must be able to understand the purpose and procedures of the study, sign informed consent and comply with requirements of the protocol.
* Age 18 and older.
* Normal serum Magnesium (1.7 - 2.3 mg/dL) and Manganese (4.7 - 18.3 ng/mL) levels or adequate supplement to obtain normal serum Mg (if Manganese levels are low (\<1.7 mg/dL), Hair Manganese will be evaluated before starting probiotic, and inclusion to the protocol will be at the principal investigators discretion).

Exclusion Criteria:

* Need for consumption of frequent antibiotics, gut pH increasing medications, and/or alkaline water.
* Patient unable to maintain regular follow up or submit to informed consent
* Stool pH \>7.5 - The ideal stool pH for growth and function of the investigational probiotic is 6-6.5.
* Patients who are judged to be ineligible for study entry by investigator or sub-investigator.

Primary outcomes:
measure: Evaluate amino acid levels before and after probiotic use.
timeFrame: 6 months
Secondary outcomes:
measure: Measure changes in questionnaires relating to subjective functional assessments.
timeFrame: 6 months
measure: Measure changes in Quality of Life.
timeFrame: 6 months
measure: Evaluate pain levels with the Numbered Pain Scale.
timeFrame: 6 months
Other outcomes:
Locations:
city: Sioux Falls
country: United States
facility: Avera Medical Group Palliative Medicine Sioux Falls
state: South Dakota
Study officials:
affiliation: Avera Medical Group Palliative Medicine Sioux Falls
name: Francine Arneson, MD
role: PRINCIPAL_INVESTIGATOR
References: