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One Patient's hope: can an old TB drug help a rare paralysis condition?

NCT ID NCT07542548

Disease control Sponsor: Massachusetts General Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the drug D-cycloserine in a single patient with a rare genetic form of hereditary spastic paraplegia. The goal was to see if the drug is safe and can slow the disease by lowering certain fats (sphingolipids) in the body. The patient also took pyridoxine to help prevent side effects. Because it's only one person, the results are very early and may not apply to others.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
D-cycloserine (with pyridoxine to reduce side effects)
What this could lead to
If it works, this could point toward a treatment for this specific genetic form of hereditary spastic paraplegia.
What could go wrong
This is a single-patient study, so results may not apply to others. The drug can cause serious side effects like liver problems, hearing loss, and nerve damage.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

1 person

The number who actually took part.

Started

Jul 2024

Finished

Mar 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 6 years

Sex

Female participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

complex hereditary spastic paraplegia hereditary spastic paraplegia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hereditary spastic paraplegia

Contacts and locations

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.