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One-Patient trial hopes to halt rare brain disease

NCT ID NCT05998395

Disease control Sponsor: National Heart, Lung, and Blood Institute (NHLBI) 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called ruxolitinib in one adult with Kohlmeier-Degos disease affecting the brain and spinal cord. The goal was to see if the drug could stop or slow the growth of lesions in the central nervous system. The participant took the drug by mouth twice daily for up to 26 weeks and had regular MRI scans and other tests to monitor effects.

What this could mean

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

Active substance
Ruxolitinib (also known as Jakafi), a tablet taken by mouth
What this could lead to
If it works, this could point toward a treatment for the central nervous system effects of Kohlmeier-Degos disease, which currently has no known effective therapy.
What could go wrong
This is a single-patient study, so results may not apply to others. The drug may not stabilize or improve brain lesions, and side effects like infections or blood count changes are possible.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

1 person

The number who actually took part.

Started

Sep 2023

Finished

Feb 2025

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

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

Ages

18 to 100 years

Sex

Anyone

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: The study design was constructed to treat one participant with CNS Kohlmeier Degos Disease. Therefore, there are no specific inclusion criteria. EXCLUSION CRITERIA: * Active life-threatening infections * Hemoglobin \<7 g/dL * Platelet counts \< 50 K /mcL * Neutropenia (ANC \<0.5 x k/mcL) * Lymphopenia (ALC \<0.2 x k/mcL) * LFTs (liver function test) \> 3 x time upper limit * eGFR/CreatCr \< 30 mL/min

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Conditions

The condition(s) this trial relates to.

malignant atrophic papulosis

As listed by the trial registrant

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

Kohlmeier degos disease with neurologic involvement Kohlmeier-degos disease

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

More trials for these conditions

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