Trial Outcomes & Findings for Tolerability and Efficacy of L-Serine in Patients With Amyotrophic Lateral Sclerosis (ALS) (NCT NCT03580616)

NCT ID: NCT03580616

Last Updated: 2025-04-20

Results Overview

Tolerability was based off of participant self-reported GI symptoms at any time during their participation.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

43 participants

Primary outcome timeframe

From baseline through when participants withdrew from study or up to 48 weeks

Results posted on

2025-04-20

Participant Flow

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.

Participant milestones

Participant milestones
Measure	L-Serine Participants who provided consent.
Overall Study STARTED	43
Overall Study Was Determined Eligible at Screening Visit	37
Overall Study Included in Interim Analysis	28
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	43

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	L-Serine n=37 Participants Participants determined to be eligible at screening visit
Age, Customized 50 - 59 years	14 Participants n=99 Participants
Age, Customized 60 - 69 years	12 Participants n=99 Participants
Age, Customized 70 - 79 years	11 Participants n=99 Participants
Sex: Female, Male Female	12 Participants n=99 Participants
Sex: Female, Male Male	25 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	35 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=99 Participants
Region of Enrollment United States	37 participants n=99 Participants

PRIMARY outcome

Timeframe: 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.

Tolerability was based off of participant self-reported GI symptoms at any time during their participation.

Outcome measures

Outcome measures
Measure	L-Serine n=37 Participants Participants determined to be eligible at screening visit
Dose Tolerability Based on Subject Reporting Bowel Obstruction	1 Participants
Dose Tolerability Based on Subject Reporting Nausea	8 Participants
Dose Tolerability Based on Subject Reporting Diarrhea	7 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	L-Serine n=28 Participants Participants determined to be eligible at screening visit
Mean ALS Functional Rating Scale - Revised (ALSFRS-R) Score	28.8732 Mean score Standard Deviation 5.3734

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome data not reported

Adverse Events

L-Serine

Serious events: 12 serious events

Other events: 31 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	L-Serine n=37 participants at risk Participants determined to be eligible at screening visit
Cardiac disorders Respiratory Arrest	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Gastrointestinal disorders Bowel Obstruction	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Musculoskeletal and connective tissue disorders Viral infection and mild rhabdomyolysis	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Psychiatric disorders Suicide Attempt	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Respiratory, thoracic and mediastinal disorders Breathing difficulty/Ventilation Issues	8.1% 3/37 • Number of events 3 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Surgical and medical procedures Gastric tube Placement	5.4% 2/37 • Number of events 2 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Surgical and medical procedures Pre-Planned Surgical Procedure	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Musculoskeletal and connective tissue disorders Worsening weakness and Falls	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Musculoskeletal and connective tissue disorders Bimalleolar Fracture with Lateral Subluxation	2.7% 1/37 • Number of events 1 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.

Other adverse events

Other adverse events
Measure	L-Serine n=37 participants at risk Participants determined to be eligible at screening visit
Musculoskeletal and connective tissue disorders Falls	43.2% 16/37 • Number of events 24 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Gastrointestinal disorders Diarrhea	18.9% 7/37 • Number of events 12 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.
Gastrointestinal disorders Nausea	21.6% 8/37 • Number of events 15 • The adverse event data was collected from baseline through when participants withdrew from study or up to 48 weeks. 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.

Additional Information

Dr. Elijah Stommel

Dartmouth-Hitchcock Medical Center

Phone: 6036505104

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place