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Rare disease study tracks pearson syndrome in children

NCT ID NCT02327364

Knowledge-focused Sponsor: Sumit Parikh 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 11 children with Pearson Syndrome, a rare and serious genetic disease, for up to 3 years. Researchers observed how the disease progresses over time and looked for genetic clues that might predict its course. No treatment was given; the goal was simply to learn more about the condition.

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

11 people

The number who actually took part.

Start date

Mar 2014

Finished

Aug 2020

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

Patients with confirmed Pearson Syndrome

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: 1. All individuals of any age with confirmed Pearson Syndrome are eligible to participate. Pearson Syndrome requires the presence of a large-scale mtDNA deletion along with sideroblastic anemia with or without pancreatic insufficiency. 2. All patients must agree to participate in the NAMDC Clinical Registry Exclusion Criteria: 1. Patient does not fulfill criteria for Pearson Syndrome 2. Not willing to participate in the NAMDC clinical Registry

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Conditions

The condition(s) this trial relates to.

Pearson syndrome VLCAD deficiency

As listed by the trial registrant

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

Pearson syndrome

More trials for these conditions

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