Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01733407 · ClinicalTrials.gov registry record · Phase 1
L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1
A Phase 1 study, sponsored by Massachusetts General Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 18
- Enrollment target
NCT01733407: Completed Phase 1 study, sponsored by Massachusetts General Hospital.
NCT01733407 is a Phase 1 study that has completed, run by Massachusetts General Hospital. The registered enrollment target is 18 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (70% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01733407, a Phase 1 study, has completed, sponsored by Massachusetts General Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 18 participants
- Enrollment target
Study Summary
In hereditary sensory and autonomic neuropathy type 1 (HSAN1) the investigators recently discovered the accumulation of two neurotoxic sphingolipids. It appears that these lipids arise as the mutant enzyme has a reduced affinity for its normal preferred substrate L-serine. The investigators now plan to perform a two year study of L-serine supplementation to correct the biochemistry and neurological disease in humans with HSAN1. In the course the investigators will also establish correlations between an existing neurological rating scale of sensory neuropathy and intraepidermal nerve fiber density. Funding Source - FDA OOPD
Primary Outcome
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 f
Interventions
- DRUG placebo
- DRUG L-serine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 18 participants |
| Start Date | 2013-09 |
| Est. Completion | 2017-07 |
| Phase | Phase 1 |
What the finished NCT01733407 record still lists
NCT01733407 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 18 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (70% lower).
The record links to 0 conditions, and to 2 interventions - of which placebo is the first listed.
NCT01733407 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01733407 about?
NCT01733407 is a clinical study titled "L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1". In hereditary sensory and autonomic neuropathy type 1 (HSAN1) the investigators recently discovered the accumulation of two neurotoxic sphingolipids. It appears that these lipids arise as the mutant enzyme has a reduced affinity for its normal preferred substrate L-serine. The investigators now plan...
What is the current status of trial NCT01733407?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 2013-09. Estimated completion is 2017-07.
What interventions are being tested in trial NCT01733407?
The interventions under investigation include: placebo (DRUG), L-serine (DRUG).
Who is sponsoring clinical trial NCT01733407?
This trial is sponsored by Massachusetts General Hospital, which has 2,032 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01733407's enrollment target sits among peer trials
18 1720th of 2000 higher than 216 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01245179 · 18 participants · Phase 1
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
- NCT01999361 · 18 participants · NA
Prevention of de Novo Allosensitization in Islet Transplant Recipients Following Complete Graft Loss
- NCT02359825 · 18 participants · Phase 1
Nerve Repair Using Hydrophilic Polymers to Promote Immediate Fusion of Severed Axons and Swift Return of Function
- NCT02870244 · 18 participants · Phase 1
Adoptive T Cell Immunotherapy for Advanced Melanoma Using Engineered Lymphocytes
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia