Amyotrophic Lateral Sclerosis (ALS)
Conditions
Brief summary
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
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.
Interventions
DRUGL-Serine
L-Serine is a naturally occurring dietary amino acid. It is abundant in soy products, some edible seaweeds, sweet potatoes, eggs and meat.
Sponsors
Brain Chemistry Labs
Elijah W. Stommel
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Dose Tolerability Based on Subject Reporting | From baseline through when participants withdrew from study or up to 48 weeks | Tolerability was based off of participant self-reported GI symptoms at any time during their participation. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean ALS Functional Rating Scale - Revised (ALSFRS-R) Score | Baseline through 12 weeks | Amyotrophic lateral sclerosis functional rating scale-revised version (ALSFRS-R) is used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS and each is scored between 0 (no function at all) and 4 (normal function). Thus the overall score for this measure can range from 0 to 48, with higher scores indicating more normal function. |
| Efficacy Based on Pulmonary Forced Vital Capacity (FVC) as Measured by Mean Slope Through Time | No data available | — |
Countries
United States
Participant flow
Recruitment details
Routine monitoring revealed major noncompliance that was not adequately addressed by the Sponsor-Investigator. Subsequently, the study was terminated by the Institutional Review Board (IRB). The IRB determined these deviations compromised the integrity of the data. Furthermore, the study data collected have been deemed unreliable due to issues related to the study's conduct.
Participants by arm
| Arm | Count |
|---|---|
| L-Serine Participants determined to be eligible at screening visit | 37 |
| Total | 37 |
Baseline characteristics
| Characteristic | L-Serine |
|---|---|
| Age, Customized 50 - 59 years | 14 Participants |
| Age, Customized 60 - 69 years | 12 Participants |
| Age, Customized 70 - 79 years | 11 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 35 Participants |
| Region of Enrollment United States | 37 participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 25 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 37 |
| other Total, other adverse events | 31 / 37 |
| serious Total, serious adverse events | 12 / 37 |
Outcome results
Primary
Dose Tolerability Based on Subject Reporting
Tolerability was based off of participant self-reported GI symptoms at any time during their participation.
Time frame: From baseline through when participants withdrew from study or up to 48 weeks
Population: Routine monitoring revealed major noncompliance that was not adequately addressed by the Sponsor-Investigator. Subsequently, the study was terminated by the Institutional Review Board (IRB). The IRB determined these deviations compromised the integrity of the data. Furthermore, the study data collected have been deemed unreliable due to issues related to the study's conduct.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| L-Serine | Dose Tolerability Based on Subject Reporting | Diarrhea | 7 Participants |
| L-Serine | Dose Tolerability Based on Subject Reporting | Bowel Obstruction | 1 Participants |
| L-Serine | Dose Tolerability Based on Subject Reporting | Nausea | 8 Participants |
Secondary
Efficacy Based on Pulmonary Forced Vital Capacity (FVC) as Measured by Mean Slope Through Time
Time frame: No data available
Population: Routine monitoring revealed major noncompliance that was not adequately addressed by the Sponsor-Investigator. Subsequently, study was terminated by the Institutional Review Board. The protocol specified that this outcome measure would obtain the slope through time as a measure of efficacy based on FVC. Although raw data for the FVC was collected, these data were not analyzed and slope values were not obtained before study closure. The slope values will never be obtained due to study termination
Secondary
Mean ALS Functional Rating Scale - Revised (ALSFRS-R) Score
Amyotrophic lateral sclerosis functional rating scale-revised version (ALSFRS-R) is used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS and each is scored between 0 (no function at all) and 4 (normal function). Thus the overall score for this measure can range from 0 to 48, with higher scores indicating more normal function.
Time frame: Baseline through 12 weeks
Population: Routine monitoring revealed major noncompliance that was not adequately addressed by the Sponsor-Investigator. Subsequently, the study was terminated by the Institutional Review Board (IRB). The IRB determined these deviations compromised the integrity of the data. Furthermore, the study data collected have been deemed unreliable due to issues related to the study's conduct.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| L-Serine | Mean ALS Functional Rating Scale - Revised (ALSFRS-R) Score | 28.8732 Mean score | Standard Deviation 5.3734 |