Cure My Disease

Don't miss your cure!

 Log in / Sign up

Can a single gene therapy dose keep working for 14 years?

NCT ID NCT06805695

Knowledge-focused Sponsor: iECURE, Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 31, 2026 · Last updated Sep 01, 2026 · Updated 1 time

Summary

This study follows people who received an investigational gene therapy in an earlier iECURE trial for a urea cycle disorder, a condition that can cause dangerous ammonia buildup. Researchers will track safety, side effects, and whether the therapy's effects last over up to 14.5 years. They will also collect health data from participants who were enrolled but never dosed, to understand the natural course of the disease. The goal is to learn how durable and safe the treatment is over the long term.

What this could mean

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

Active substance
No intervention (observational follow-up study)
What this could lead to
If successful, this follow-up could confirm whether a single gene therapy dose provides lasting benefit and safety over many years for people with urea cycle disorders.
What could go wrong
This is a small, long-term observational study, not a treatment trial. It cannot prove the therapy works, and results depend on the small number of participants who join.

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 13 people

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

Started

Dec 2024

Expected to finish

Jul 2041

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

All participants from an iECURE parent treatment protocol, whether they received IP or not, will be consented to enter this LTFU study. Enrollment into the LTFU will begin upon either premature discontinuation from, or completion of, the parent treatment protocol.

Ages

7 to 15 months

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. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol 2. Participant parent(s)/LAR is willing and able to adhere to the protocol requirements. 3. Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected. Exclusion Criteria: 1\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Ornithine transcarbamylase deficiency are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn brain disorder central nervous system disorder hereditary disease Hyperammonemia inborn disorder of amino acid metabolism inborn errors of metabolism liver disorder metabolic disease nervous system disorder ornithine carbamoyltransferase deficiency urea cycle disorder X-linked disease

As listed by the trial registrant

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

Ornithine transcarbamylase deficiency Ornithine transcarbamylase deficiency disease Urea cycle disorders, inborn

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

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

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Children's Hospital of Colorado, Anshutz Medical Campus

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Great Ormond Street Hospital

    RECRUITING

    London, United Kingdom

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

More trials for these conditions

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