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Scientists use skin and blood to create 'Disease in a Dish' for genetic research

NCT ID NCT03612310

Knowledge-focused Sponsor: Sapna Vyas 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study collects small samples of skin, blood, or urine from people with genetic diseases and healthy volunteers. Researchers will turn these samples into stem cells that can grow into any cell type, like neurons or heart cells. The goal is to develop reliable lab methods to study diseases and test new treatments, without using animals or simple cell models.

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 improve how we study genetic diseases in the lab, potentially speeding up drug discovery and understanding of disease mechanisms.
What could go wrong
This is an early-stage protocol development study, not a treatment trial. It may not lead to direct patient benefits, and results depend on overcoming technical challenges in stem cell differentiation.

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 3,000 people

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

Started

Nov 2018

Expected to finish

Jul 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

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 diagnosed with genetic diseases and/or healthy controls

Ages

1 year to 120 years

Sex

Anyone

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: * Male or female * Individuals diagnosed with a genetic disease - any age between 1-120 years. * Individuals diagnosed with a genetic disease - must be able to communicate well with the investigator and to comply with the requirements of the entire study OR be under the care of an appropriate guardian, if incapacitated or under the age of 16. * Individuals diagnosed with a genetic disease - require provision of written informed consent either by participant or guardian, to participate as shown by a witnessed signature on the Subject Consent Form * Individuals participating as controls - aged between 16-120 years. * Individuals participating as controls -must be able to consent for themselves Exclusion Criteria: * Individuals less than 1 year old. * Infection with any blood borne diseases (e.g. HIV, Hepatitis B or Hepatitis C). * Previous or current intravenous drug abuse. * For donation of blood samples only - has exceeded annual limit for blood donation. * Affected by blood disorders such as anaemia, blood clotting disorders or currently on anticoagulant drug therapy. * Individuals participating as controls - excluded if aged less than 16 years old. * Individuals participating as controls - excluded if unable to consent for themselves. * Individuals diagnosed with a genetic disease - unable to provide informed consent either by themselves or through an appropriate nearest relative, legal guardian or welfare attorney.

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Conditions

The condition(s) this trial relates to.

hereditary disease

As listed by the trial registrant

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

Genetic disease

How to take part

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

  1. The places running it

    1 site. 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

  • Alba Science

    RECRUITING

    Edinburgh, Midlothian, EH1 3RH, United Kingdom

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