Status:
UNKNOWN
Hereditary Sensory Neuropathy Serine Trial
Lead Sponsor:
University College, London
Conditions:
Hereditary Sensory Neuropathy Type I
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
This is a randomised double-blind placebo-controlled trial of L-serine in Hereditary Sensory Neuropathy type 1 (HSN1) due to variants in SPTLC1/2 gene. This is a single-centre study being conducted a...
Detailed Description
Hereditary Sensory Neuropathy (HSN) represents a subgroup of disorders called Charcot Marie Tooth (CMT) disease. Hereditary Sensory Neuropathy Type 1 (HSN1) is the most common subtype. It is a rare au...
Eligibility Criteria
Inclusion
- Participants aged ≥ 18 with genetically proven HSN1 due to SPTLC1/2 mutations.
- Participants must be able to undergo an MRI scan without sedation.
- Participants must be able to complete the Charcot Marie Tooth Neuropathy Score (CMTNS)
- Participants must have a CMTES ≤ 26
- Female participants of childbearing potential must agree to use a highly effective method of contraception from the time consent is signed until six days after treatment discontinuation (this is to allow for medication wash out post treatment discontinuation).
- Participants must be willing and able to provide written informed consent.
Exclusion
- Participants have undergone foot surgery in the 6 months prior to trial enrolment or are due to undergo foot surgery during the trial
- Participants have a history of nephrolithiasis
- Participants have another medical condition which precludes them from having an MRI scan or from completing the CMTNSv2
- Participants with known diagnosis of another neuromuscular disease
- Participants with diabetes
- Females who are planning pregnancy or are pregnant or breastfeeding.
- Patient taking regular L-serine supplementation within 6 months of study commencement.
Key Trial Info
Start Date :
August 21 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2025
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT06113055
Start Date
August 21 2023
End Date
August 1 2025
Last Update
November 2 2023
Active Locations (1)
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1
University College London
London, United Kingdom