Trial · NCT01733407

L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1

NCT01733407, Phase 1, Phase 2, Completed, Massachusetts General Hospital; results first posted 2018-09-12

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1. Trials, record NCT01733407. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01733407

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1
NCT number: NCT01733407
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 18 participants, actual
Lead sponsor: Massachusetts General Hospital
Conditions: Hereditary Sensory and Autonomic Neuropathy Type I
Interventions: L-serine, placebo
First posted: 2012-11-27
Last updated: 2018-09-12
Results first posted: 2018-09-12

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Charcot Marie Tooth Neuropathy Score	48 Weeks	The Charcot Marie Tooth Neuropathy Score (CMTNS) is a 0 to 36 point composite scoring assessment that is used to measure disease severity in Charcot Marie Tooth Neuropathy and other sensory and motor neuropathies. The CMTNS is composed of 9 items that evaluate functions related to disease progression. These 9 parameters include reviewing sensory symptoms, motor symptoms (arms and legs), pinprick sensibility, vibration, leg strength, arm strength, and nerve conduction tests. Each item is scored from 0 to 4, with the lower scores representing less severe symptoms and higher scores representing more severe symptoms.The 9 individual item scores are then totaled to provide a global measure of disease severity. For example the lowest possible total score is 0 which represents an asymptomatic individual and the highest score possible is a 36 which represents an individual with severe disease progression. There are sub scores that can be assessed but sub scores were not utilized in this study
Secondary	Intraepidermal Nerve Fiber Density (IENFD)	48 Weeks	Counts of nerve fibers per unit area in skin biopsies
Secondary	Autonomic Function Testing (AFT) Composite Autonomic Severity Score (CASS)	48 Weeks	Autonomic Function Testing (AFT) tests the effectiveness of your autonomic nervous system which regulates important functions such as blood pressure, heart rate, and respiration. AFT results are quantified using the composite autonomic severity score scale (CASS) which is a scale from 0 to 10 that is the sum of three sub scores (cardiovagal, adrenergic, and sudomotor). Cardiovagal is scored from 0 to 3, sudomotor is scored from 0 to 3, and adrenergic is scored from 0 to 4. The tests include deep breathing, Valsalva maneuver, head-up tilt, and a sweat test. The three subscores are then summed. This total represents the CASS which classifies autonomic function as normal functioning (total score 0), mild (total score 1-3), moderate (total score 4-6), or severe (total score 7-10).
Secondary	Nerve Conduction Testing	48 Weeks	Evaluates the functioning of electrical conduction of the motor and sensory nerves of the human body.
Secondary	1-deoxy-sphinganine	48 Weeks	Plasma levels of the deoxysphingoid lipid 1-deoxy-sphinganine measured by liquid chromatography/mass spectrometry after hydrolyzing the N-acyl and O-linked headgroups
Secondary	1-deoxy-sphingosine	48 weeks	Plasma levels of the deoxysphingoid lipid 1-deoxy-sphingosine measured by liquid chromatography/mass spectrometry after hydrolyzing the N-acyl and O-linked headgroups

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Charcot Marie Tooth Neuropathy Score	48 Weeks	scores on a scale	MEAN
SECONDARY	Intraepidermal Nerve Fiber Density (IENFD)	48 Weeks	nerve fibers per micrometer^2	MEAN
SECONDARY	Autonomic Function Testing (AFT) Composite Autonomic Severity Score (CASS)	48 Weeks	scores on a scale	MEAN
SECONDARY	Nerve Conduction Testing	48 Weeks	microvolts	MEAN
SECONDARY	1-deoxy-sphinganine	48 Weeks	micromole per liter	MEAN
SECONDARY	1-deoxy-sphingosine	48 weeks	micromole per liter	MEAN

Source ClinicalTrials.gov

Results by group30

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Charcot Marie Tooth Neuropathy Score	Sugar Pill		25.67	Standard Deviation 6.69	scores on a scale
Charcot Marie Tooth Neuropathy Score	L-serine		20.22	Standard Deviation 10.10	scores on a scale
Intraepidermal Nerve Fiber Density (IENFD)	Sugar Pill	Upper Thigh	34.67	Standard Deviation 35.12	nerve fibers per micrometer^2
Intraepidermal Nerve Fiber Density (IENFD)	L-serine	Upper Thigh	49.56	Standard Deviation 43.44	nerve fibers per micrometer^2
Intraepidermal Nerve Fiber Density (IENFD)	Sugar Pill	Lower Calf	0.89	Standard Deviation 2.67	nerve fibers per micrometer^2
Intraepidermal Nerve Fiber Density (IENFD)	L-serine	Lower Calf	13.89	Standard Deviation 24.10	nerve fibers per micrometer^2
Autonomic Function Testing (AFT) Composite Autonomic Severity Score (CASS)	Sugar Pill		3.56	Standard Deviation 1.24	scores on a scale
Autonomic Function Testing (AFT) Composite Autonomic Severity Score (CASS)	L-serine		2.22	Standard Deviation 1.64	scores on a scale
Nerve Conduction Testing	Sugar Pill	Sensrory Right Median Amplitude	1.34	Standard Error 4.03	microvolts
Nerve Conduction Testing	L-serine	Sensrory Right Median Amplitude	5.51	Standard Error 9.27	microvolts

1 to 10 of 30Source ClinicalTrials.gov

See all 30 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Sugar Pill	9
Overall Study	STARTED	L-serine	9
Overall Study	COMPLETED	Sugar Pill	7
Overall Study	COMPLETED	L-serine	9
Overall Study	NOT COMPLETED	Sugar Pill	2
Overall Study	NOT COMPLETED	L-serine	0

Source ClinicalTrials.gov

Adverse events64

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	ABDOMINAL PAIN UPPER	Gastrointestinal disorders	Sugar Pill	0	9	0
other	ABDOMINAL PAIN UPPER	Gastrointestinal disorders	L-serine	1	9	1
other	Diarrhea	Gastrointestinal disorders	Sugar Pill	1	9	1
other	Diarrhea	Gastrointestinal disorders	L-serine	0	9	0
other	Dyspepsia	Gastrointestinal disorders	Sugar Pill	0	9	0
other	Dyspepsia	Gastrointestinal disorders	L-serine	1	9	1
other	Cellulitis	Infections and infestations	Sugar Pill	1	9	1
other	Cellulitis	Infections and infestations	L-serine	0	9	0
other	Clostridium Difficile Infection	Infections and infestations	Sugar Pill	1	9	1
other	Clostridium Difficile Infection	Infections and infestations	L-serine	0	9	0

1 to 10 of 64Source ClinicalTrials.gov

See all 64 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sugar Pill	1	9
Total, all-cause mortality	L-serine	0	9
Total, other adverse events	Sugar Pill	9	9
Total, other adverse events	L-serine	9	9
Total, serious adverse events	Sugar Pill	1	9
Total, serious adverse events	L-serine	1	9

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
22045570	BACKGROUND	PubMed
30626650	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Sugar Pill vs L-serine: deaths from any cause	Trial comparisons
Sugar Pill vs L-serine: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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