Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Gene therapy hope for rare immune disease fails to launch

NCT ID NCT00778882

Disease control Sponsor: Helixmith Co., Ltd. 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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 trial aimed to test a gene therapy for X-linked chronic granulomatous disease, a condition where the immune system cannot fight certain infections. The treatment involved taking the patient's own stem cells, adding a working copy of the defective gene, and returning them to the body. However, the study was withdrawn before any participants were enrolled, so no safety or effectiveness data were collected.

What this could mean

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

Active substance
Gene-modified stem cells (VM106)
What this could lead to
If successful, this approach could provide a way to restore immune function in patients with chronic granulomatous disease, reducing severe infections.
What could go wrong
The trial was withdrawn before enrolling anyone, so no results exist. Gene therapy carries risks like insertional mutagenesis and immune reactions.

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.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Expected to start

Jan 2007

An estimate. Start dates often move.

Expected to finish

Oct 2010

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.

Ages

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

Sex

Male participants only

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: * gp91 defective male patients with chronic granulomatous disease: confirmed by DHR * Weigh greater than or equal to 15 kg * History of severe infections: more than 2 times * Performance status: ECOG 0-2 * Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases. * Heart: a shortening fraction \> 28%; QTc interval \< 0.44 * Liver: total bilirubin \< 2 × upper limit of normal; ALT \< 3 × upper limit of normal; AST \< 3 x upper limit of normal * Kidney: creatine \< 2 x normal * Blood: WBC \> 2,500/uL; platelet \> 100,000/uL; hematocrit \> 26% * Written informed consent obtained from patient (or guardian if patients age \< 19) Exclusion Criteria: * Presence of a HLA-matched sibling for stem cell donation * Evidence or history of malignant tumor * Presence of a severe infection * Presence of an active tuberculosis * Uncorrectable electrolyte, Ca, P * Unable to comply with the protocol or to cooperate fully with the Investigator or site personnel

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Chronic granulomatous disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

chronic granulomatous disease granulomatous disease, chronic, X-linked

As listed by the trial registrant

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

Chronic granulomatous disease

Contacts and locations

Locations

  • Seoul National University Hospital

    Seoul, 110-744, South Korea

More trials for these conditions

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