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Single-Patient trial tests heart drug for rare genetic syndrome

NCT ID NCT06534671

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 whether a single intravenous dose of diltiazem, a calcium channel blocker, can shorten the QT interval in adults with Jervell and Lange-Nielsen syndrome, a rare genetic heart condition that increases the risk of dangerous heart rhythms. Only one person participated, and the researchers measured heart activity for up to 35 minutes after the drug was given. The goal was to see if diltiazem could quickly affect the heart's electrical activity.

What this could mean

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

Active substance
diltiazem (a calcium channel blocker given intravenously)
What this could lead to
If it works, this could point toward a way to temporarily shorten the QT interval and reduce the risk of dangerous heart rhythms in people with Jervell and Lange-Nielsen syndrome.
What could go wrong
This was a very small study with only one participant, so results may not apply to others. It only looked at immediate effects of a single dose, not long-term safety or benefit.

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

Oct 2024

Finished

Oct 2024

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

18 years and older

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: * Age greater than 18 years * Genetically confirmed diagnosis of Jervell and Lange-Nielsen syndrome * Able to provide written informed consent Exclusion Criteria: * Known hypersensitivity to diltiazem * Pregnancy * Congestive heart failure, angina, preexcitation, or chronic obstructive pulmonary disease * Sick sinus syndrome or atrioventricular block in the absence of a pacemaker/defibrillator * Any clinically significant ongoing medical or surgical condition that might jeopardize the subject's safety or interfere with the conduct of the study in the judgement of the investigator * Baseline systolic blood pressure less than 100 Hg or diastolic blood pressure less than 60 Hg

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States