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Gene therapy for rare metabolic disease passes early safety check

NCT ID NCT05506254

Disease control Sponsor: Alexion Pharmaceuticals, Inc. 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 follows 4 people with methylmalonic acidemia who previously received hLB-001 gene therapy. Researchers are checking for long-term side effects. The goal is to see if the treatment remains safe over time.

What this could mean

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

Active substance
hLB-001 gene therapy
What this could lead to
If successful, this follow-up could show that hLB-001 is safe over the long term, supporting its development as a treatment for methylmalonic acidemia.
What could go wrong
This is a very small, non-interventional study with only 4 participants, so results may not apply to others. It only monitors safety, not effectiveness.

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

4 people

The number who actually took part.

Started

Jul 2022

Expected to finish

Jan 2037

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

This is a non-interventional long-term follow-up study of participants who have participated in LogicBio studies for the treatment of MMA and received hLB-001.

Ages

18 years and older

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: * Participant has previously participated in a LogicBio clinical trial and received hLB-001. Exclusion Criteria: * None

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Conditions

The condition(s) this trial relates to.

methylmalonic acidemia

As listed by the trial registrant

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

Methylmalonic acidemia

Contacts and locations

Locations

  • Clinical Trial Site

    Atlanta, Georgia, 30322, United States

  • Clinical Trial Site

    Pittsburgh, Pennsylvania, 15224, United States

  • Clinical Trial Site

    Nashville, Tennessee, 37232, United States

  • Clinical Trial Site

    Seattle, Washington, 98105, United States

More trials for these conditions

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