D-Cycloserine for Serine Palmitoyltransferase Inhibition

NCT07542548 | Completed Phase 4 FDA Drug

• 3 other identifiers: 2024P0010685T32GM007748-47GR0235874
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The overarching objective of this study is to mitigate the neurological decline associated with SPTSSA related Complex Hereditary Spastic Paraplegia

Conditions

Hereditary Spastic Paraplegia

Keywords

D-Cycloserine; SPTSSA Gene; Complex Hereditary Spastic Paraplegia

Outcome Measures

Primary Outcomes (12)

• Frequency of treatment related serious adverse event

  Frequency of treatment related serious adverse events as assessed by CTCAE Version 5.0

  From baseline to the end of treatment at 1 year

• Concentration of D-Cycloserine

  Concentration of D-Cycloserine in mcg/mL from serum blood samples drawn 6 hours after dosing

  From baseline to the end of treatment at 1 year

• Increases in serum AST and ALT in (U/L)

  From baseline to the end of treatment at 1 year

• Presence or Absence of Changes in Brain Magnetic Resonance Imaging

  Changes in Brain Magnetic Resonance Imaging associated with adverse events

  From baseline to the end of treatment at 1 year

• Presence or Absence of Changes in Spine Magnetic Resonance Imaging

  Change in Spine Magnetic Resonance Imaging associated with adverse events

  From baseline to the end of treatment at 1 year

• Presence or Absence of Changes in Electroencephalogram (EEG)

  Changes in Electroencephalogram (EEG) associated with adverse events

  From baseline to the end of treatment at 1 year

• Presence or Absence of Changes on audiogram.

  Changes in audiogram associated with adverse events related to hearing loss

  From baseline to the end of treatment at 1 year

• Presence of Absence of Changes in nerve conduction studies.

  Changes in nerve conduction studies related to adverse events

  From baseline to the end of treatment at 1 year

• Presence or Absence of Changes in cognitive profile on neuropsychological testing.

  Changes in neuropsychological testing related to adverse events

  From baseline to the end of treatment at 1 year

• Changes in gross motor function as measured by the Gross Motor Function Measure (GMFM-88)

  Higher scores indicate better capacity for gross motor function

  From baseline to the end of treatment at 1 year

• Changes in spasticity as measured by findings on the Tardieu Spasticity Scale

  That Tardieu Spasticity Scale is scored from 0 to 5. Lower scores are given for less spasticity while higher scores are given for more spasticity

  From baseline to the end of treatment at 1 year

• Changes in performance as measured by scores on Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT)

  From baseline to the end of treatment at 1 year

Secondary Outcomes (2)

• Decrease in sphingolipid levels

  From baseline to the end of treatment at 1 year

• Decrease in serum neurofilament light chain level

  From baseline to the end of treatment at 1 year

Study Arms (1)

D-Cycloserine

EXPERIMENTAL

D-Cycloserine Administered for Complex Hereditary Spastic Paraplegia

Drug: D-cycloserine

Interventions

D-cycloserine DRUG

Pyridoxine also prescribed to help prevent neurologic adverse events related to D-Cycloserine.

D-Cycloserine

Eligibility Criteria

• Age: 6 Years - 6 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Informed consent provided by the participant's parents.
• Ability to travel to the study and assessment sites (Massachusetts General Hospital Main Campus, 55 Fruit St, Boston, MA 02114 and MGH IHP Impact Practice Center, 2 Constitution Wharf, Charlestown, MA 02129) and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
• Clinical phenotype, neuroimaging, genetic testing and biochemical results consistent with a diagnosis of SPTSSA-related Complex Hereditary Spastic Paraplegia

You may not qualify if:

• Participant has any known contraindication to or unwillingness to undergo procedures listed in the protocol
• Use of investigational medication within 5 half-lives of the drug at enrollment
• Participant has any condition that, in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Uniformed Services University of the Health Sciences: collaborator
• National Institute of General Medical Sciences (NIGMS): collaborator

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Spastic Paraplegia, Hereditary

Interventions

Cycloserine

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Isoxazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Oxazolidinones; Oxazoles; Serine; Amino Acids, Neutral; Amino Acids; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: The treatment is already FDA approved for other indications (Tuberculosis and UTIs), and is being used off-label.

Study Record Dates

• First Submitted: March 4, 2026
• First Posted: April 21, 2026
• Study Start: July 10, 2024
• Primary Completion: March 17, 2026
• Study Completion: March 17, 2026
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF

Locations

US (1)