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Qatar study hunts for hidden genes behind rare diseases

NCT ID NCT02021734

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 2 times

Summary

This study looked at 400 people in Qatar with rare genetic diseases. Because marriages between relatives are more common there, some genetic conditions appear more often. Researchers used advanced DNA sequencing to find the exact gene changes causing these illnesses. The goal was to discover new disease-causing mutations, not to test a treatment.

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 could help identify new genetic causes of rare diseases, potentially leading to better diagnosis and future treatments.
What could go wrong
This is an observational study, not a treatment trial. It may not directly benefit participants, and findings may not apply to other populations.

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 400 people

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

Started

Nov 2012

Finished

Feb 2025

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

physicians and by thorough review of the literature to establish that the disease is indeed Mendelian and with unknown gene.

Ages

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

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

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: * All included individuals must provide informed consent * All genetic disorders are included * All ethnic backgrounds are accepted * Disease must be genetic with no evident environmental cause * Evidence of Mendelian Transmission determined by fulfilling one of the following criteria: * Multiple affected family members (at least first degree relative with disease) * History of consanguinity * Severe disease in newborn in the absence of family history * Sydromic disease in single individuals * Congenital abnormality affecting major organ system(s) * Mendelianized extremes of common disease (eg sever familial diabetes/ obesity/ hypertension) Exclusion Criteria: * Individuals who do no consent to be included * Mendelian disease for which a gene mutation has already been identified * Individuals for which a molecular diagnosis has already been established by alternative method * Disease for which an environmental factor is most likely the cause * Disease for which late age of onset rule out Mendelian transmission * Common diseases for which late age of onset rule out Mendelian transmission

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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

  • Hamad Medical Corporation

    Doha, Qatar

More trials for these conditions

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