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A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With ALS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03324399
Recruitment Status : Completed
First Posted : October 27, 2017
Last Update Posted : July 2, 2019
Sponsor:
Information provided by (Responsible Party):
Avera McKennan Hospital & University Health Center

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis ALS Dietary Supplement: probiotic Not Applicable

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With Amyotrophic Lateral Sclerosis (ALS)
Actual Study Start Date : June 1, 2017
Actual Primary Completion Date : June 26, 2019
Actual Study Completion Date : June 26, 2019


Arm Intervention/treatment
Experimental: High Amino Acid levels

Comparison between high amino acid levels and low amino acid levels of clinical global assessments, muscle strength and spasticity and functional assessments.

Patients taking proprietary probiotic

Dietary Supplement: probiotic
a proprietary probiotic formulation

Experimental: Low Amino Acid levels

Comparison between high amino acid levels and low amino acid levels of clinical global assessments, muscle strength and spasticity and functional assessments.

Patients taking proprietary probiotic

Dietary Supplement: probiotic
a proprietary probiotic formulation




Primary Outcome Measures :
  1. Evaluate amino acid levels before and after probiotic use. [ Time Frame: 6 months ]
    Amino acid profiles will be compared before the probiotic use and over a period of treatment for 6 months.


Secondary Outcome Measures :
  1. Measure changes in questionnaires relating to subjective functional assessments. [ Time Frame: 6 months ]

    Changes will be measured with Amyotrophic Lateral Sclerosis -Functional Rating Scale (ALS-FRS).

    The ALS-FRS is a 12 item questionnaire to assess bulbar, limb, and respiratory functions. The scores may range from 0, which is the worst function, to 48 which is the best function. This questionnaire is self-administered, and can be completed with assistance from a family member. The scores range from 0-48. The highest scores reflect no changes, or difficulty with tasks assessed. Low scores indicate poor functioning and need for assistance.


  2. Measure changes in Quality of Life. [ Time Frame: 6 months ]
    Changes will be measured with the Amyotrophic Lateral Sclerosis -Quality of Life questionnaire. The ALS-QOL is a 50-item, self-administered, questionnaire which includes 7 scores. These scores represent six domains and a total average score. The six domains are 1) Negative Emotion, 2) Interaction, 3) Intimacy, 4) Religiosity, 5) Physical and 6) Bulbar.

  3. Evaluate pain levels with the Numbered Pain Scale. [ Time Frame: 6 months ]
    Changes in pain rating of 0 - 10 will be collected during the 6 months of study.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03324399


Locations
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United States, South Dakota
Avera Medical Group Palliative Medicine Sioux Falls
Sioux Falls, South Dakota, United States, 57105
Sponsors and Collaborators
Avera McKennan Hospital & University Health Center
Investigators
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Principal Investigator: Francine Arneson, MD Avera Medical Group Palliative Medicine Sioux Falls

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Responsible Party: Avera McKennan Hospital & University Health Center
ClinicalTrials.gov Identifier: NCT03324399     History of Changes
Other Study ID Numbers: AIHG-2017.004-ALS
First Posted: October 27, 2017    Key Record Dates
Last Update Posted: July 2, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Avera McKennan Hospital & University Health Center:
ALS
Amyotrophic Lateral Sclerosis

Additional relevant MeSH terms:
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Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases