CompletedPhase 4

D-Cycloserine for Serine Palmitoyltransferase Inhibition

United States1 participantsStarted 2024-07-10

Plain-language summary

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

Who can participate

Age range

6 Years – 6 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * • 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 Exclusion Criteria: * • 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Frequency of treatment related serious adverse event

Timeframe: From baseline to the end of treatment at 1 year

Concentration of D-Cycloserine

Timeframe: From baseline to the end of treatment at 1 year

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

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes in Brain Magnetic Resonance Imaging

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes in Spine Magnetic Resonance Imaging

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes in Electroencephalogram (EEG)

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes on audiogram.

Timeframe: From baseline to the end of treatment at 1 year

Trial details

NCT IDNCT07542548

SponsorMassachusetts General Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-03-17

View on ClinicalTrials.gov More Hereditary Spastic Paraplegia trials

Who can participate

Age range

6 Years – 6 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Frequency of treatment related serious adverse event

Timeframe: From baseline to the end of treatment at 1 year

Concentration of D-Cycloserine

Timeframe: From baseline to the end of treatment at 1 year

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

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes in Brain Magnetic Resonance Imaging

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes in Spine Magnetic Resonance Imaging

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes in Electroencephalogram (EEG)

Timeframe: From baseline to the end of treatment at 1 year

Presence or Absence of Changes on audiogram.

Timeframe: From baseline to the end of treatment at 1 year