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Can tracking MADD's natural course unlock better care?

NCT ID NCT07734090

Knowledge-focused Sponsor: Icahn School of Medicine at Mount Sinai Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 29, 2026 · Last updated Sep 17, 2026 · Updated 3 times

Summary

This study follows people with Multiple Acyl-CoA Dehydrogenase Deficiency (MADD) over time to learn how the condition progresses and affects daily life. Researchers will collect health data and patient feedback to identify patterns and potential markers of the disease. The goal is to build a clearer picture of MADD that could guide future treatments and improve quality of life for those affected.

What this could mean

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

What this could lead to
If successful, this study could identify key biomarkers and patient-reported outcomes that help design future treatments and improve care for MADD.
What could go wrong
This is an observational study, not a treatment trial, so it offers no direct benefit to participants. It may also face challenges in recruiting enough participants due to the rarity of MADD.

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.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Oct 2026

An estimate. Start dates often move.

Expected to finish

Jul 2031

An estimate. End dates often move.

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.

Who is studied

Individuals with a biochemical and/or molecular diagnosis of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), as confirmed by a study investigator.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Have a biochemical and/or molecular diagnosis of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), as confirmed by a study investigator * Provision of signed and dated informed consent form (and assent when applicable) from subject or subject's legal representative Exclusion Criteria: \- Presence of a major unrelated condition

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Conditions

The condition(s) this trial relates to.

Multiple Acyl Coenzyme A Dehydrogenase Deficiency multiple acyl-CoA dehydrogenase deficiency

As listed by the trial registrant

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

Multiple acyl-coa dehydrogenase deficiency

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    2 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital Colorado Anschutz Medical Campus

    NOT_YET_RECRUITING

    Aurora, Colorado, 80045, United States

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States